Wiskott-Aldrich Syndrome Clinical Trial
Official title:
Randomized, Two-arm Single-center Phase II Clinical Trial Comparing the Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
This is a prospective, open-label, randomized, two-arm clinical trial conducted to evaluate the safety and efficacy of romiplostim in comparison with eltrombopag in the treatment of thrombocytopenia in patients with Wiskott-Aldrich syndrome
Status | Recruiting |
Enrollment | 30 |
Est. completion date | July 30, 2023 |
Est. primary completion date | December 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 18 Years |
Eligibility |
Inclusion Criteria: - Genetically verified Wiskott-Aldrich syndrome - Thrombocytopenia (platelet count < 70 x 109/L) - Age: under 18 years - Subject/legal representative has signed written informed consent. Exclusion Criteria: - Patients, who do not meet the inclusion criteria. - Any prior history of arterial or venous thrombosis within the past year. - Arm II (eltrombopag): 1. abnormal hepatic function -elevated AST/ALT > 1.5 times upper limit of normal within 4 weeks prior to enrollment 2. Active colitis |
Country | Name | City | State |
---|---|---|---|
Russian Federation | Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology | Moscow |
Lead Sponsor | Collaborator |
---|---|
Federal Research Institute of Pediatric Hematology, Oncology and Immunology |
Russian Federation,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of patients with overall platelet response (complete response + partial response) for romiplostim and eltrombopag group | A complete response will be defined as a platelet count >100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. | 1 month (30 day +/- 7 days) | |
Secondary | Number of participants with bleeding events and severity of bleeding in romiplostim and eltrombopag group | The incidence and severity of bleeding events is evaluated with a modified World Health Organization (WHO) Bleeding Scale. (G1=Petechiae, epistaxis <30 min, G2=Mild blood loss, hematomas, epistaxis >30 min, melanotic stool G3=Gross blood loss, requiring blood transfusions, G4=Fatal bleeding). |
until discontinuation, from at least one month to one year | |
Secondary | Number of participants with drug related adverse events in each treatment group | Adverse events are graded according to Common Terminology Criteria for Adverse Events (CTCAE). | until discontinuation, from at least one month to one year | |
Secondary | Percentage of patients with overall platelet response (complete response + partial response) after switching from one TPO-RA to another one. | until discontinuation, from at least one month to one year |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT01652092 -
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
|
N/A | |
Completed |
NCT01953016 -
Participation in a Research Registry for Immune Disorders
|
||
Active, not recruiting |
NCT02333760 -
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
|
Phase 1/Phase 2 | |
Terminated |
NCT01319851 -
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
|
N/A | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Completed |
NCT01347346 -
Gene Therapy for WAS
|
Phase 1/Phase 2 | |
Completed |
NCT00160355 -
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
|
Phase 1 | |
Recruiting |
NCT01821781 -
Immune Disorder HSCT Protocol
|
Phase 2 | |
Completed |
NCT01289847 -
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
|
Phase 4 | |
Completed |
NCT01347242 -
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
|
Phase 1/Phase 2 | |
Terminated |
NCT00006054 -
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
|
N/A | |
Enrolling by invitation |
NCT03198195 -
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
|
N/A | |
Recruiting |
NCT03019809 -
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
|
Phase 2 | |
Terminated |
NCT00909363 -
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
|
Phase 2 | |
Enrolling by invitation |
NCT01852370 -
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
|
Phase 1/Phase 2 | |
Completed |
NCT03513328 -
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT01410825 -
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT00004341 -
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
|
N/A | |
Completed |
NCT03399461 -
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
|
||
Active, not recruiting |
NCT03837483 -
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
|
Phase 3 |