Wiskott-Aldrich SyndromePhase 2WASNCT00909363
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
A Phase 2 study testing Promacta and blood drawing in patients with WAS in people with Wiskott-Aldrich Syndrome and Thrombocytopenia, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo. Read more about this study
- Who is it for?
- Ages 3 months to 80 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- What is being tested?
- Promacta and blood drawing in patients with WAS
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to 30 Jun 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 80 years
- Men only
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- In order to be eligible for study entry, subjects must comply with the following:
- Males from 3 months old to 80 years old
- Signed written informed consent obtained prior to study entry
- Clinical diagnosis of WAS or XLT
- Platelet levels less than 100 x 109/L
- Adequate renal and hepatic function (creatinine and bilirubin less than or equal to 1.5 x IULN, AST and ALT less than or equal to 2.5 x IULN)
Who cannot join
- Any patient is ineligible for study entry if he/she:
- Over the age of 80
- Women (only males are eligible)
- fertile men who are not practicing or who are unwilling to practice birth control while enrolled in the study or until at least 6 months after treatment
- Aspirin, aspirin-containing compounds, salicylates, non-steroidal anti-inflammatory medications (NSAIDS), clopidogrel or ticlopidine, warfarin or other vitamin K antagonists, unfractionated or low molecular heparin within 7 days of first infusion
- Red blood cell transfusion in the past four weeks
Show all 12Show fewer
- Elevated (> 1.5 x ULN) prothrombin time (PT) or partial thromboplastin time (PTT)
- New York Heart Classification III or IV heart disease. Other severe cardiovascular or cardiopulmonary disease, including COPD.
- Known HIV infection, hepatitis B or C infection
- Any infection requiring antibiotic treatment within 3 days
- Other concurrent medical or psychiatric conditions that, in the Investigator's opinion, may be likely to confound study interpretation or prevent completion of study procedures and follow-up examinations.
- Prior malignancy with less than a 5-year disease-free interval, excluding nonmelanoma skin cancers and carcinoma in situ of the cervix
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medical College
New York, New York, 10065
About this study
Official title: Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
Read the detailed description
Wiskott Aldrich Syndrome is an X-linked disease characterized by immunodeficiency, eczema and thrombocytopenia; a milder form of the disease known as X-Linked thrombocytopenia also exists. The thrombocytopenia in both WAS and XLT is characterized by: severe thrombocytopenia with platelet counts frequently less than 10-30,000/ul; small platelets which may be dysfunctional; and, as a result, a high rate of serious bleeding including intracranial hemorrhage.
Because eltrombopag has been shown to be remarkably efficacious in substantially increasing platelet counts in a high percentage of ITP patients, this study seeks to effectively treat patients who exhibit similar pathologies, as well as evaluate the state of platelets in patients with WAS and relate it to clinical bleeding. It also aims to demonstrate whether eltrombopag administered daily will enhance stem cell function, increase platelet production and platelet count, and reduce bleeding in patients with WAS.
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Being tested
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- Medical subject headings
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PromactaDrug
Also called eltrombopag, revolade
WAS Patients receiving treatment 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 as well as on liver tests.
they will also have diagnostic blood testing prior to initiating treatment
blood drawing in patients with WASDiagnostic test
blood will be drawn for platelet parameters in WAS patients not receiving treatment either because they declined or because they were ineligible
blood drawing in healthy controlsDiagnostic test
blood will be drawn once in healthy children as controls for platelet parameters
| Study group | Role | What they receive |
|---|---|---|
| WAS patients receiving Promacta | Experimental | 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. Receives: Drug: Promacta |
| WAS patients for blood drawing only | Experimental | WAS patients not receiving treatment to serve as subjects for platelet parameter studies blood drawing once only Receives: Diagnostic Test: blood drawing in patients with WAS |
| healthy children for blood drawing only | Placebo comparator | healthy children having blood obtained once as controls for platelet parameters study Receives: Diagnostic Test: blood drawing in healthy controls |
What is being measured
The primary outcome is the main question the study is designed to answer.
How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.
number of WAS patients achieving this increase to > 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment
Measured: 12 weeks
Secondary outcomes (3)
Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)
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
Measured: 12 Weeks
How Many Patients With WAS Had Abnormal Platelet Function Including Activation
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
Measured: 12 weeks
How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag
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
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2009 (estimated)
- Study startJun 2009
- Primary completion15 May 2017
- Study completion30 Jun 2017
Why it stopped: retirement of PI
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Wiskott-Aldrich SyndromeStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- 1. group of 11 WAS patients treated with eltrombopag 2. WAS patients who had their blood drawn once but did not receive eltrombopag treatment 3. healthy children as controls for testing only 3\) healthy volunteers who had their blood drawn once
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- James B Bussel, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00909363
- Sponsor's protocol ID
- 0801009600
- Acronym
- WAS
- Status last verified
- Mar 2019
- First submitted
- 27 May 2009
- Last updated
- 18 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Gerrits AJ, Leven EA, Frelinger AL 3rd, Brigstocke SL, Berny-Lang MA, Mitchell WB, Revel-Vilk S, Tamary H, Carmichael SL, Barnard MR, Michelson AD, Bussel JB. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015 Sep 10;126(11):1367-78. doi: 10.1182/blood-2014-09-602573. Epub 2015 Jul 29.
PubMed 26224646
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.
- ClinicalTrials.gov ID
- NCT00909363
- Acronym / sponsor ID
- WAS / 0801009600
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: retirement of PI
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- Jun 2009
- Primary completion
- 15 May 2017 (actual)
- Study completion
- 30 Jun 2017 (actual)
- First posted
- 28 May 2009 (estimated)
- Results
- Posted 18 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
Detailed description
Wiskott Aldrich Syndrome is an X-linked disease characterized by immunodeficiency, eczema and thrombocytopenia; a milder form of the disease known as X-Linked thrombocytopenia also exists. The thrombocytopenia in both WAS and XLT is characterized by: severe thrombocytopenia with platelet counts frequently less than 10-30,000/ul; small platelets which may be dysfunctional; and, as a result, a high rate of serious bleeding including intracranial hemorrhage.
Because eltrombopag has been shown to be remarkably efficacious in substantially increasing platelet counts in a high percentage of ITP patients, this study seeks to effectively treat patients who exhibit similar pathologies, as well as evaluate the state of platelets in patients with WAS and relate it to clinical bleeding. It also aims to demonstrate whether eltrombopag administered daily will enhance stem cell function, increase platelet production and platelet count, and reduce bleeding in patients with WAS.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- 1. group of 11 WAS patients treated with eltrombopag 2. WAS patients who had their blood drawn once but did not receive eltrombopag treatment 3. healthy children as controls for testing only 3) healthy volunteers who had their blood drawn once
| Arm | Type | Receives |
|---|---|---|
| WAS patients receiving Promacta | Experimental | Drug: PromactaPromacta® 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. |
| WAS patients for blood drawing only | Experimental | Diagnostic Test: blood drawing in patients with WASWAS patients not receiving treatment to serve as subjects for platelet parameter studies blood drawing once only |
| healthy children for blood drawing only | Placebo comparator | Diagnostic Test: blood drawing in healthy controlshealthy children having blood obtained once as controls for platelet parameters study |
Who may qualify
- Ages
- 3 months to 80 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Males from 3 months old to 80 years old
- Signed written informed consent obtained prior to study entry
- Clinical diagnosis of WAS or XLT
- Platelet levels less than 100 x 109/L
- Adequate renal and hepatic function (creatinine and bilirubin less than or equal to 1.5 x IULN, AST and ALT less than or equal to 2.5 x IULN)
Key exclusion criteria (12 listed)
- Over the age of 80
- Women (only males are eligible)
- fertile men who are not practicing or who are unwilling to practice birth control while enrolled in the study or until at least 6 months after treatment
- Aspirin, aspirin-containing compounds, salicylates, non-steroidal anti-inflammatory medications (NSAIDS), clopidogrel or ticlopidine, warfarin or other vitamin K antagonists, unfractionated or low molecular heparin within 7 days of first infusion
- Red blood cell transfusion in the past four weeks
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PromactaAlso: eltrombopag, revoladeWAS Patients receiving treatment 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 as well as on liver tests. they will also have diagnostic blood testing prior to initiating treatment | Drug | WAS patients receiving Promacta |
| blood drawing in patients with WASblood will be drawn for platelet parameters in WAS patients not receiving treatment either because they declined or because they were ineligible | Diagnostic test | WAS patients for blood drawing only |
| blood drawing in healthy controlsblood will be drawn once in healthy children as controls for platelet parameters | Diagnostic test | healthy children for blood drawing only |
Primary outcome
How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.
Time frame: 12 weeks
number of WAS patients achieving this increase to > 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment
3 secondary outcomes
Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)
Time frame: 12 Weeks
How Many Patients With WAS Had Abnormal Platelet Function Including Activation
Time frame: 12 weeks
How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag
Time frame: 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26224646Gerrits AJ, Leven EA, Frelinger AL 3rd, Brigstocke SL, Berny-Lang MA, Mitchell WB, Revel-Vilk S, Tamary H, Carmichael SL, Barnard MR, Michelson AD, Bussel JB. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015 Sep 10;126(11):1367-78. doi: 10.1182/blood-2014-09-602573. Epub 2015 Jul 29.
- Individual participant dataSharing planned: Undecided
Related trials for Wiskott-Aldrich Syndrome
Active, not recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
SCID, Omenn's Syndrome
Enrolling by invitationObservational study
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study
Chronic Pain, Depression Disorders
RecruitingObservational study
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Enrolling by invitationPhase 1/2
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James B Bussel, MD principal investigator
Weill Medical College of Cornell University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Promacta and blood drawing in patients with WAS in people with Wiskott-Aldrich Syndrome and Thrombocytopenia, run by Weill Medical College of Cornell University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul. (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00909363, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2019.