Trial Outcomes & Findings for Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy (NCT NCT04784455)
NCT ID: NCT04784455
Last Updated: 2025-06-05
Results Overview
Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24 Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
10 participants
Primary outcome timeframe
24 weeks
Results posted on
2025-06-05
Participant Flow
The study population consists of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and developed haematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT
Participant milestones
| Measure |
Nomacopan
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Nomacopan
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Overall Study
Death
|
4
|
Baseline Characteristics
Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy
Baseline characteristics by cohort
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Age, Customized
≥ 0.5 to < 2 years
|
1 Participants
n=5 Participants
|
|
Age, Customized
≥ 2 to < 9 years
|
3 Participants
n=5 Participants
|
|
Age, Customized
≥ 9 to < 18 years
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksRed blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24 Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Outcome measures
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
RBC transfusion independent for ≥ 28 days
|
2 Participants
|
|
Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.
Urine protein creatinine ratio (UPCR) ≤ 2 mg/mg for ≥ 28 days
|
4 Participants
|
SECONDARY outcome
Timeframe: 24 weeksPlasma sC5b-9 ≤ upper limit of normal (ULN)
Outcome measures
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)
|
10 Participants
|
SECONDARY outcome
Timeframe: 24 weeksLactate dehydrogenase (LDH) ≤ULN
Outcome measures
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)
|
2 Participants
|
SECONDARY outcome
Timeframe: 24 weeksNumber of participants with a normalised haptoglobin value (where haptoglobin is within the normal ranges)
Outcome measures
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Normalisation of Lab Parameters
|
4 Participants
|
SECONDARY outcome
Timeframe: 24 weeksTransfusion independence is defined as no platelet transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.
Outcome measures
| Measure |
Nomacopan
n=10 Participants
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
|---|---|
|
Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.
|
0 Participants
|
Adverse Events
Nomacopan - 1.7 mg/kg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Nomacopan - 1.3 mg/kg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Nomacopan - 1.0 mg/kg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Nomacopan - 0.30 mg/kg
Serious events: 6 serious events
Other events: 7 other events
Deaths: 4 deaths
Nomacopan - 0.45 mg/kg
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Nomacopan - 0.60 mg/kg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Nomacopan - 1.7 mg/kg
n=1 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
One patient received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 1.3 mg/kg
n=3 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
Three patients received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 1.0 mg/kg
n=6 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
Six patients received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 0.30 mg/kg
n=10 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
10 patient received the starting dose of 0.30 mg/kg
|
Nomacopan - 0.45 mg/kg
n=3 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
Of the 10 enrolled patients, 3 patients had a dose increase to 0.45 mg/kg
|
Nomacopan - 0.60 mg/kg
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
No patients had a dose increase to 0.60 mg/kg
|
|---|---|---|---|---|---|---|
|
Cardiac disorders
Cardiac arrest
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Cytopenia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Infections and infestations
Device related infection
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/10 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
General disorders
Generalised oedema
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Vascular disorders
Hypertension
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/10 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
General disorders
Multiple organ dysfunction syndrome
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Infections and infestations
Pneumonia parainfluenzae viral
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Nervous system disorders
Posterior reversible encephalopathy syndrome
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/10 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
Other adverse events
| Measure |
Nomacopan - 1.7 mg/kg
n=1 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
One patient received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 1.3 mg/kg
n=3 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
Three patients received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 1.0 mg/kg
n=6 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
Six patients received the ablating dose of 1.7mg/kg.
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
|
Nomacopan - 0.30 mg/kg
n=10 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
10 patient received the starting dose of 0.30 mg/kg
|
Nomacopan - 0.45 mg/kg
n=3 participants at risk
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
Of the 10 enrolled patients, 3 patients had a dose increase to 0.45 mg/kg
|
Nomacopan - 0.60 mg/kg
The trial was a 24-week open-label, non-comparative study in paediatric patients with HSCT-TMA.
Patients will be given an ablating dose on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1:
Ablating Dose:
* 0.5 to \< 2 years =1.7 mg/kg
* 2 to \< 9 years =1.3 mg/kg
* 9 to \< 18 years =1.0 mg/kg
For children in the youngest two cohorts (≥ 0.5 to \< 2 years, and ≥ 2 to \< 9 years), the dose was re-calculated at the week 8 and week 16 visits using the child's new weight measured at that visit.
The starting maintenance dose, administered 12 hours apart, from Day 2 until the end of treatment was 0.30 mg/kg for all 3 age groups.
If required, from pre-dose Day 7 onwards, a dose escalation was permitted if the CH50 results were \> 10 U Eq/mL and/or the unbound nomacopan in serum was \< 55 ng/mL.
Two dose escalations were permitted - an increase from the maintenance dose of 0.30 mg/kg to 0.45 mg/kg with a further increase to 0.60 mg/kg if required. Dose increases were preceded by an ablating dose as determined by age as per Day 1.
No patients had a dose increase to 0.60 mg/kg
|
|---|---|---|---|---|---|---|
|
Investigations
Blood Creatinine Increased
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Vascular disorders
Hypotension
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 4 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
16.7%
1/6 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 24 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
50.0%
5/10 • Number of events 16 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 4 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
General disorders
Pyrexia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
50.0%
5/10 • Number of events 17 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
40.0%
4/10 • Number of events 16 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
40.0%
4/10 • Number of events 10 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypermagnesaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
40.0%
4/10 • Number of events 7 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 5 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 7 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 8 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 7 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 5 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
30.0%
3/10 • Number of events 5 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 2 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
20.0%
2/10 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
0.00%
0/1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
0.00%
0/6 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
10.0%
1/10 • Number of events 3 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
33.3%
1/3 • Number of events 1 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
|
—
0/0 • Duration of the study (Maximum of 2 year follow-up)
An ablating dose was given on Day 1, 12 hours apart which was determined by the child's age and weight on Day 1. One patient was administered the 1.7 mg/kg ablation dose, 3 patients received the 1.3 mg/kg ablation dose and 6 patients received the 1.0 mg/kg ablation dose. There were three maintenance dose levels - 0.3 mg/kg, 0.45 mg/kg and 0.60 mg/kg. All ten patients started on 0.3 mg/kg. Three patients had a dose escalation to 0.45mg/kg. No patient were dose escalated to 0.60 mg/kg.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place