Wiskott-Aldrich Syndrome Clinical Trial
Official title:
NIH Participation to USIDNET Registry
Verified date | July 2023 |
Source | National Institutes of Health Clinical Center (CC) |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
Background: - People with primary immune deficiency diseases (PIDD) have weak immune systems. This makes it hard for their bodies to fight infection. The Immune Deficiency Foundation has a network to collect data about people with PIDD. It is called the United States Immunodeficiency Network. It will help doctors and scientists better understand these disorders. The goal is to get medical data for everyone with these disorders in the U.S. and Canada. Data will be stored in a registry. Researchers can use it to study if these disorders are increasing. They can also learn how the disorders are diagnosed and treated. Objectives: - To collect data on people with primary immune deficiency disorders. Eligibility: - People who have a PIDD. Design: - Data can be added with no record of personal identity. - Data can be added with identity kept separate. This data will be linked to the registry by a code number. - Data for the registry includes: - Family history - Disease treatment - Disease characteristics - Medical history - Laboratory data
Status | Completed |
Enrollment | 716 |
Est. completion date | July 19, 2023 |
Est. primary completion date | May 1, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month and older |
Eligibility | - INCLUSION CRITERIA Individuals of all ages, gender, and races with an immunodeficiency disorder from NIH studies will be accepted for registration. No healthy volunteers will be enrolled. EXCLUSION CRITERIA Individuals with immunodeficiency associated with HIV infection, chemotherapy or other immunosuppressive therapies will not be accepted for registration unless there is clear evidence that these individuals also have a genetically determined immunodeficiency disease as well. Adult individuals who do not give informed consent will also be excluded. |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Human Genome Research Institute (NHGRI) | National Institute of Allergy and Infectious Diseases (NIAID), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Prevalence | The Registry will provide a minimum estimate of the prevalence of each disorder in the US, a comprehensive clinical picture of each disorder, and a resource for clinical and laboratory research. | Ongoing |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT01652092 -
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
|
N/A | |
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 | |
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 |