Wiskott-Aldrich Syndrome Clinical Trial
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency
disorders.
II. Explore the mutations within each syndrome to better understand the genetics of these
disorders.
III. Study the function of the Wiskott-Aldrich syndrome proteins (WASP). IV. Design methods
to identify carriers and for prenatal diagnosis. V. Explore new avenues for therapy.
PROTOCOL OUTLINE: Patients are studied systematically to determine the extent of their
immune deficiency and to confirm a specific diagnosis. Patients with a known
immunodeficiency syndrome are studied in detail to identify the gene mutation, to assess the
effect of the mutation on the gene product, and to establish cell lines for further in vitro
assessment of the genetic defect. The function of Wiskott-Aldrich syndrome proteins (WASP)
in hematopoietic cells is studied.
Family members of patients with X-linked disorders are studied to identify carrier females.
;
Primary Purpose: Screening
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
|
||
Recruiting |
NCT04371939 -
Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
|
Phase 2 | |
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 |
NCT01347242 -
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
|
Phase 1/Phase 2 | |
Completed |
NCT01289847 -
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
|
Phase 4 | |
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 | |
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 |