Trial Outcomes & Findings for Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients (NCT NCT00909363)
NCT ID: NCT00909363
Last Updated: 2019-03-18
Results Overview
number of WAS patients achieving this increase to \> 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
12 weeks
Results posted on
2019-03-18
Participant Flow
24 patients' families signed consents 11 WAS patients for treatment: 1 mother signed consent but never consented to have her very young baby start study treatment (dietary issues) + 1 withdrew 8 WAS patients' parents only were willing to have study bloods drawn 5 normal healthy children had their parents give consent for a blood draw
Participant milestones
| Measure |
WAS Patients Treated With Promacta
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
WAS Patients for Blood Drawing Only
patients with WAS whose parents did not want them to receive treatment but were willing to let them have their blood drawn to increase the number of patients studied for platelet parameters with WAS
|
Healthy Children
platelet parameters (primarily function) needed normal controls in children-----three were pre-op and two siblings were being tested as potential bone marrow donors
|
|---|---|---|---|
|
Overall Study
STARTED
|
11
|
8
|
5
|
|
Overall Study
COMPLETED
|
9
|
8
|
5
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
0
|
Reasons for withdrawal
| Measure |
WAS Patients Treated With Promacta
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
WAS Patients for Blood Drawing Only
patients with WAS whose parents did not want them to receive treatment but were willing to let them have their blood drawn to increase the number of patients studied for platelet parameters with WAS
|
Healthy Children
platelet parameters (primarily function) needed normal controls in children-----three were pre-op and two siblings were being tested as potential bone marrow donors
|
|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
0
|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
0
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Promacta
n=11 Participants
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
n=5 Participants
5 well children having blood drawn for another reason: 3 pre-op and 2 for HLAA-typing
|
WAS Patients for Blood Drawing Only
n=8 Participants
patients with WAS either not eligible or not interested in eltrombpopag treatment who are willing to have their blood drawn once
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=11 Participants
|
5 Participants
n=5 Participants
|
8 Participants
n=8 Participants
|
23 Participants
n=24 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=11 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=8 Participants
|
1 Participants
n=24 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=11 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=8 Participants
|
0 Participants
n=24 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=11 Participants
|
2 Participants
n=5 Participants
|
0 Participants
n=8 Participants
|
2 Participants
n=24 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=11 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=8 Participants
|
22 Participants
n=24 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
United States
|
11 participants
n=11 Participants
|
5 participants
n=5 Participants
|
8 participants
n=8 Participants
|
24 participants
n=24 Participants
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: 1 WAS subject withdrew prior to starting treatment and was not included in the analysis. Healthy volunteers were neither WAS patients nor received eltrombopag (both required for achievement of primary outcome #1). The WAS patients who were blood draw only also were 0 since they did not receive eltrombopag.
number of WAS patients achieving this increase to \> 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment
Outcome measures
| Measure |
Promacta
n=10 Participants
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
Was Patients Blood Drawing Only
WAS pts who were ineligible for or did not want eltrombopag treatment
|
|---|---|---|---|
|
How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.
|
8 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 WeeksPopulation: 1 subject withdrew prior to starting treatment and was not included in the analysis
number of patients with bleeding SAEs while on treatment and/or number of patients with grade 3 or higher bleeding on WHO (World Health Organization) scale: the scale is from 1 to 5 with 5 = fatality and 1=very little bleeding
Outcome measures
| Measure |
Promacta
n=10 Participants
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
n=5 Participants
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
Was Patients Blood Drawing Only
n=8 Participants
WAS pts who were ineligible for or did not want eltrombopag treatment
|
|---|---|---|---|
|
Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)
|
1 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: 1 subject withdrew prior to starting treatment and was not included in the analysis
in how many patients with WAS were platelets dysfunctional or activated before treatment as measured by flow cytometry to a substantial degree and the same after treatment with eltrombopag
Outcome measures
| Measure |
Promacta
n=10 Participants
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
n=5 Participants
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
Was Patients Blood Drawing Only
n=8 Participants
WAS pts who were ineligible for or did not want eltrombopag treatment
|
|---|---|---|---|
|
How Many Patients With WAS Had Abnormal Platelet Function Including Activation
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: 1 subject withdrew prior to starting treatment and was not included in the analysis. no treatment with eltrombopag was given to healthy volunteers and WAS patients who were blood drawing only so the effect of treatment in these 2 groups could not be assessed
in how many patients with WAS did eltrombopag increase platelet production as measured by the immature platelet fraction (IPF), a variable derived from the Sysmex auto analyzer, which is considered to be a measure of newly formed platelets ie reticulated platelets
Outcome measures
| Measure |
Promacta
n=10 Participants
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
Was Patients Blood Drawing Only
WAS pts who were ineligible for or did not want eltrombopag treatment
|
|---|---|---|---|
|
How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag
|
3 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Promacta
Serious events: 3 serious events
Other events: 2 other events
Deaths: 0 deaths
Healthy Volunteers
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
WAS Patients for Blood Drawing Only
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Promacta
n=10 participants at risk
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
n=5 participants at risk
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
WAS Patients for Blood Drawing Only
n=8 participants at risk
WAS patients who are either ineligible or do not want treatment but are willing to have their blood drawn once for testing
|
|---|---|---|---|
|
Blood and lymphatic system disorders
bleeding SAE
|
10.0%
1/10 • Number of events 1 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/5 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/8 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
|
Hepatobiliary disorders
increased liver tests
|
20.0%
2/10 • Number of events 2 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/5 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/8 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
Other adverse events
| Measure |
Promacta
n=10 participants at risk
Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.
Promacta (eltrombopag): WAS Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
Eltrombopag/promacta: Patients will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP.
|
Healthy Volunteers
n=5 participants at risk
8 normal subjects will be studied for degree of platelet activation from one blood draw by collaborator Alan Michelson at Boston Childrens Hospital
|
WAS Patients for Blood Drawing Only
n=8 participants at risk
WAS patients who are either ineligible or do not want treatment but are willing to have their blood drawn once for testing
|
|---|---|---|---|
|
Hepatobiliary disorders
increased liver tests
|
20.0%
2/10 • Number of events 4 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/5 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
0.00%
0/8 • 12 weeks of treatment was the focus-----subsequent information (after 12 weeks) did not demonstrate any problems
in how many patients and in how many events was there an increase in transaminases to \> 2 times the upper limit of normal. Note, one patient was never treated and thus 10, not 11, patients were evaluable for adverse events
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place