Trial · NCT00909363
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
NCT00909363, Phase 2, Terminated, Weill Medical College of Cornell University; results first posted 2019-03-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients. Trials, record NCT00909363. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00909363
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
- NCT number
- NCT00909363
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Weill Medical College of Cornell University
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Interventions
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- First posted
- 2009-05-28
- Last updated
- 2019-03-18
- Results first posted
- 2019-03-18
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul. | 12 weeks | number of WAS patients achieving this increase to \> 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment |
| Secondary | Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) | 12 Weeks | 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 |
| Secondary | How Many Patients With WAS Had Abnormal Platelet Function Including Activation | 12 weeks | 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 |
| Secondary | How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag | 12 weeks | 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 |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul. | 12 weeks | 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. | participants | NUMBER |
| SECONDARY | Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) | 12 Weeks | 1 subject withdrew prior to starting treatment and was not included in the analysis | participants | NUMBER |
| SECONDARY | How Many Patients With WAS Had Abnormal Platelet Function Including Activation | 12 weeks | 1 subject withdrew prior to starting treatment and was not included in the analysis | participants | NUMBER |
| SECONDARY | How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag | 12 weeks | 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 | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul. | Promacta | 8 | participants | ||
| Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) | Promacta | 1 | participants | ||
| Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) | Healthy Volunteers | 0 | participants | ||
| Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) | WAS Pts: Blood Drawing Only | 0 | participants | ||
| How Many Patients With WAS Had Abnormal Platelet Function Including Activation | Promacta | 0 | participants | ||
| How Many Patients With WAS Had Abnormal Platelet Function Including Activation | Healthy Volunteers | 0 | participants | ||
| How Many Patients With WAS Had Abnormal Platelet Function Including Activation | WAS Pts: Blood Drawing Only | 0 | participants | ||
| How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag | Promacta | 3 | Participants | ||
| How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag | Healthy Volunteers | 0 | Participants | ||
| How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag | Was Patients Blood Drawing Only | 0 | Participants |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | WAS Patients Treated With Promacta | 11 | |
| Overall Study | STARTED | WAS Patients for Blood Drawing Only | 8 | |
| Overall Study | STARTED | Healthy Children | 5 | |
| Overall Study | COMPLETED | WAS Patients Treated With Promacta | 9 | |
| Overall Study | COMPLETED | WAS Patients for Blood Drawing Only | 8 | |
| Overall Study | COMPLETED | Healthy Children | 5 | |
| Overall Study | NOT COMPLETED | WAS Patients Treated With Promacta | 2 | |
| Overall Study | NOT COMPLETED | WAS Patients for Blood Drawing Only | 0 | |
| Overall Study | NOT COMPLETED | Healthy Children | 0 |
Source ClinicalTrials.gov
Adverse events9
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | increased liver tests | Hepatobiliary disorders | Promacta | 2 | 10 | 4 |
| other | increased liver tests | Hepatobiliary disorders | Healthy Volunteers | 0 | 5 | 0 |
| other | increased liver tests | Hepatobiliary disorders | WAS Patients for Blood Drawing Only | 0 | 8 | 0 |
| serious | bleeding SAE | Blood and lymphatic system disorders | Promacta | 1 | 10 | 1 |
| serious | bleeding SAE | Blood and lymphatic system disorders | Healthy Volunteers | 0 | 5 | 0 |
| serious | bleeding SAE | Blood and lymphatic system disorders | WAS Patients for Blood Drawing Only | 0 | 8 | 0 |
| serious | increased liver tests | Hepatobiliary disorders | Promacta | 2 | 10 | 2 |
| serious | increased liver tests | Hepatobiliary disorders | Healthy Volunteers | 0 | 5 | 0 |
| serious | increased liver tests | Hepatobiliary disorders | WAS Patients for Blood Drawing Only | 0 | 8 | 0 |
Source ClinicalTrials.gov
Adverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Promacta | 0 | 10 |
| Total, all-cause mortality | Healthy Volunteers | 0 | 5 |
| Total, all-cause mortality | WAS Patients for Blood Drawing Only | 0 | 8 |
| Total, other adverse events | Promacta | 2 | 10 |
| Total, other adverse events | Healthy Volunteers | 0 | 5 |
| Total, other adverse events | WAS Patients for Blood Drawing Only | 0 | 8 |
| Total, serious adverse events | Promacta | 3 | 10 |
| Total, serious adverse events | Healthy Volunteers | 0 | 5 |
| Total, serious adverse events | WAS Patients for Blood Drawing Only | 0 | 8 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26224646 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00909363
GET https://conductscience.org/api/trials/v1/NCT00909363/registered