Trial Outcomes & Findings for Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) (NCT NCT02591862)
NCT ID: NCT02591862
Last Updated: 2023-07-12
Results Overview
LDH is an indicator of disease progression in patients with PNH and is expected to fall to within 2x upper limit of normal (ULN) within 28 days in successfully treated patients. Units are measured in ratio of LDH:ULN, where the ULN is 250 U/L. The primary efficacy endpoint was the LDH AUC from Day 0 to 28 compared with 28 days pretreatment. Data for the 28 days pre-treatment was not collected, and as only one patient was recruited, the LDH values compared with baseline and the ratio of LDH to the Upper Limit of Normal (ULN) are presented.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
Day 0 and Day 28
Results posted on
2023-07-12
Participant Flow
Participant milestones
| Measure |
Coversin
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin: Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Overall Study
STARTED
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1
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Overall Study
COMPLETED
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1
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)
Baseline characteristics by cohort
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin: Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Age, Categorical
<=18 years
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0 Participants
n=5 Participants
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Age, Categorical
Between 18 and 65 years
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1 Participants
n=5 Participants
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
|
Age, Continuous
|
32 years
STANDARD_DEVIATION 0 • n=5 Participants
|
|
Sex: Female, Male
Female
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 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
Netherlands
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1 participants
n=5 Participants
|
|
Weight
|
67.8 kg
n=5 Participants
|
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Height
|
172 cm
n=5 Participants
|
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Serum Lactate Dehydrogenase
|
1391 U/L
n=5 Participants
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|
Haemoglobin concentration (Hb)
|
8.2 Millimole(s)/litre
n=5 Participants
|
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Haptoglobin (Hp) concentration
|
0.0 Gram(s)/litre
n=5 Participants
|
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Dependency on blood transfusion
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0 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 0 and Day 28Population: Single arm trial design, results from 1 enrolled participant.
LDH is an indicator of disease progression in patients with PNH and is expected to fall to within 2x upper limit of normal (ULN) within 28 days in successfully treated patients. Units are measured in ratio of LDH:ULN, where the ULN is 250 U/L. The primary efficacy endpoint was the LDH AUC from Day 0 to 28 compared with 28 days pretreatment. Data for the 28 days pre-treatment was not collected, and as only one patient was recruited, the LDH values compared with baseline and the ratio of LDH to the Upper Limit of Normal (ULN) are presented.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Measurement of Ratio of LDH to the Upper Limit of Normal (ULN)
Day 0
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5.6 Ratio of LDH:ULN (250 U/L)
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Measurement of Ratio of LDH to the Upper Limit of Normal (ULN)
Day 28
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1.9 Ratio of LDH:ULN (250 U/L)
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PRIMARY outcome
Timeframe: 2 yearsPopulation: Results from 1 enrolled participant.
The number and type of reported AEs will be recorded as well as the opinion of the Principle Investigator (PI) as to their possible relationship to the study drug.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Number and Type of Adverse Events (AE)
Serious Adverse Events (SAE)
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2 Events
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Number and Type of Adverse Events (AE)
Adverse Event (AE) (total)
|
20 Events
|
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Number and Type of Adverse Events (AE)
Treatment Emergent Adverse Event (TAEA)
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13 Events
|
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Number and Type of Adverse Events (AE)
AE - moderate
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1 Events
|
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Number and Type of Adverse Events (AE)
AE - severe
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1 Events
|
|
Number and Type of Adverse Events (AE)
AE- mild
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18 Events
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SECONDARY outcome
Timeframe: Baseline, Day 28, Day 90 and Day 180Population: Results from 1 enrolled participant.
Measuring change in mean Hb from Day 28, Day 90 and Day 180 (absolute and change from baseline)
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Baseline
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8.2 millimole(s)/litre
|
|
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 28 (absolute)
|
7.8 millimole(s)/litre
|
|
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 28 (change from baseline)
|
-0.7 millimole(s)/litre
|
|
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 90 (absolute)
|
7.8 millimole(s)/litre
|
|
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 90 (change from baseline)
|
-0.4 millimole(s)/litre
|
|
Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 180 (absolute)
|
8.0 millimole(s)/litre
|
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Measurement of Haemoglobin (Hb) at Days 28, 90, and 180, Absolute and Change From Baseline
Day 180 (change from baseline)
|
-0.2 millimole(s)/litre
|
SECONDARY outcome
Timeframe: Baseline, Day 28, Day 90 and Day 180Population: Results from 1 enrolled participant.
Measuring change in mean Hp from Day 28, Day 90 and Day 180 (absolute and change from baseline)
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Measurement of Haptoglobin (Hp) at Days 28, 90 and 180, Absolute and Change From Baseline
Day 28
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NA gram(s)/litre
Haptoglobin was undetectable
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Measurement of Haptoglobin (Hp) at Days 28, 90 and 180, Absolute and Change From Baseline
Day 90
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NA gram(s)/litre
Haptoglobin was undetectable
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Measurement of Haptoglobin (Hp) at Days 28, 90 and 180, Absolute and Change From Baseline
Day 180
|
NA gram(s)/litre
The assay failed on day 180
|
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Measurement of Haptoglobin (Hp) at Days 28, 90 and 180, Absolute and Change From Baseline
Baseline
|
NA gram(s)/litre
Haptoglobin was undetectable
|
SECONDARY outcome
Timeframe: Baseline, Day 90 and Day 180Population: Single arm trial design, results from 1 enrolled participant.
Measuring the change in LDH at Baseline, Day 90 and Day 180. LDH is an indicator of disease progression in patients with PNH and is expected to fall to within 2x upper limit of normal (ULN) within 28 days in successfully treated patients. Units are measured in ratio of LDH:ULN, where the ULN is 250 U/L.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Measurement of Lactate Dehydrogenase (LDH) at Baseline, Day 90 and Day 180
Day 0
|
5.6 Ratio of LDH:ULN (250 U/L)
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Measurement of Lactate Dehydrogenase (LDH) at Baseline, Day 90 and Day 180
Day 90
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1.6 Ratio of LDH:ULN (250 U/L)
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Measurement of Lactate Dehydrogenase (LDH) at Baseline, Day 90 and Day 180
Day 180
|
1.5 Ratio of LDH:ULN (250 U/L)
|
SECONDARY outcome
Timeframe: Day 28, 90 and 180Functional Assessment of Chronic Illness Therapy - fatigue (FACIT-F) is a 13 item instrument designed to assess fatigue/tiredness and it's impact on daily activities and functioning in a chronic disease setting. The scale for each question in relation to quality of life ranges from 0-4 where 0= not at all, 1= a little bit, 2= somewhat, 3 = quite a bit and 4= very much. An increase in the scale score indicates an improvement in quality of life (less fatigue). A maximum score of 52 is interpreted as no fatigue . Baseline is assumed as 0.0 to demonstrate the change in units. The subscale score (as presented) is determined as per FACIT-f guidance, by multiplying the sum of the item scores by the number of items in the subscale, then divide by the number of items answered.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Change in Functional Assessment of Chronic Illness Therapy (FACIT) Score at Days 0, 28, 90 and 180
Day 28
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13.0 FACIT-f scale (Change from Baseline)
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Change in Functional Assessment of Chronic Illness Therapy (FACIT) Score at Days 0, 28, 90 and 180
Day 90
|
13.3 FACIT-f scale (Change from Baseline)
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Change in Functional Assessment of Chronic Illness Therapy (FACIT) Score at Days 0, 28, 90 and 180
Day 180
|
11.0 FACIT-f scale (Change from Baseline)
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SECONDARY outcome
Timeframe: Day 28, 90 and 180Population: Single arm trial design, results from 1 enrolled participant.
The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 instrument measures the change in the quality of of life of patients in the trial. It comprises 30 questions on daily QOL and incorporates a global health status, five functional scales (physical, role, cognitive, emotional and social), three symptom scales (fatigue, nausea/vomiting, pain), and six single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, financial difficulties). All of the scales range in score from 0 to 100, A high scale score represents a higher response level. Thus a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems. Scale scores were calculated by averaging items within scales and transforming average scores linearly.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
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|---|---|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Global Health Status/ QOL (day 28)
|
16.7 Scores on a scale (Change from baseline)
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Global Health Status/ QOL (day 90)
|
16.7 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Global Health Status/ QOL (day 180)
|
16.7 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Physical Functioning (day 28)
|
6.6 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Physical Functioning (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Physical Functioning (day 180)
|
6.6 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Role functioning (day 28)
|
33.4 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Role functioning (day 90)
|
33.4 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Role functioning (day 180)
|
16.7 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Emotional functioning (day 28)
|
16.6 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Emotional functioning (day 90)
|
8.3 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Emotional functioning (day 180)
|
8.3 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Cognitive functioning (day 28)
|
33.3 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Cognitive functioning (day 90)
|
16.7 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Cognitive functioning (day 180)
|
16.7 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Social functioning (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Social functioning (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Social functioning (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Fatigue symptom scale (day 28)
|
-11.1 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Fatigue symptom scale (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Fatigue symptom scale (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
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Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Nausea and vomiting symptom scale (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Nausea and vomiting symptom scale (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Nausea and vomiting symptom scale (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Pain symptom scale (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Pain symptom scale (day 90)
|
16.7 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Pain symptom scale (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Dyspnoea (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Dyspnoea (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Dyspnoea (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Insomnia (day 28)
|
-33.3 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Insomnia (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Insomnia (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Appetite loss (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Appetite loss (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: Appetite loss (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: constipation (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: constipation (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: constipation (day 180)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: diarrhoea (day 28)
|
-33.3 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: diarrhoea (day 90)
|
-33.3 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: diarrhoea (day 180)
|
-33.3 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: financial difficulties (day 28)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: financial difficulties (day 90)
|
0.0 Scores on a scale (Change from baseline)
|
|
Change in Quality Of Life Questionnaire (QOQ) Score at Days 0, 28, 90 and 180
Symptom: financial difficulties (day 190)
|
0.0 Scores on a scale (Change from baseline)
|
SECONDARY outcome
Timeframe: Day 0 through to study completion (2 years)Population: Results from 1 enrolled participant.
Number of participants depending on blood transfusion prior to starting the study compared to during the study.
Outcome measures
| Measure |
Coversin
n=1 Participants
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin : Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
|
|---|---|
|
Dependency on Blood Transfusion
Transfusion dependent during trial
|
0 participants
|
|
Dependency on Blood Transfusion
Transfusion dependent prior to starting trial
|
0 participants
|
Adverse Events
Coversin
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Coversin
n=1 participants at risk
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin: Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
|
|---|---|
|
Blood and lymphatic system disorders
Breakthrough Hemolysis
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Infections and infestations
Lung infection
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
Other adverse events
| Measure |
Coversin
n=1 participants at risk
This is an open label, non-comparator study.
Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given.
Coversin: Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
100.0%
1/1 • Number of events 2 • Duration of the study (2 years)
|
|
Gastrointestinal disorders
Nausea
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
General disorders
Cough
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
General disorders
Fatigue
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
General disorders
Flu like symptoms
|
100.0%
1/1 • Number of events 5 • Duration of the study (2 years)
|
|
General disorders
Injection site Reaction
|
100.0%
1/1 • Number of events 13 • Duration of the study (2 years)
|
|
General disorders
Malaise
|
100.0%
1/1 • Number of events 2 • Duration of the study (2 years)
|
|
General disorders
Non-cardiac chest pain
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Infections and infestations
Papulopustular rash
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Nervous system disorders
Headache
|
100.0%
1/1 • Number of events 6 • Duration of the study (2 years)
|
|
Nervous system disorders
Insomnia
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Renal and urinary disorders
Haematuria
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Renal and urinary disorders
Haemoglobinurea
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Coughing
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
100.0%
1/1 • Number of events 2 • Duration of the study (2 years)
|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
|
Vascular disorders
Cold fingers and toes
|
100.0%
1/1 • Number of events 1 • Duration of the study (2 years)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The PI of the clinical units has independent rights of publication but will agree to discuss any intended publications or presentations with the Sponsor and to allow the Sponsor reasonable time to make comments, file or add to patent applications or request changes to the manuscript
- Publication restrictions are in place
Restriction type: OTHER