Clinical trials: X-Linked Severe Combined Immune Deficiency (XSCID)
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Severe Combined Immunodeficiency
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat…
- Trial name
- NCT01129544
- Testing
- Gene transfer
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- David Williams
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the…
- Trial name
- NCT03311503
- Testing
- autologous CD34+ cell transduced with G2SCID vector
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- David Williams
Severe Combined Immunodeficiency
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells…
- Trial name
- NCT00028236
- Testing
- Gene-Transduced Autologous CD34+ Stem Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
X-linked Severe Combined Immunodeficiency
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys.
- Trial name
- NCT01175239
- Testing
- Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID)
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
This study will evaluate the safety and effectiveness of insulin-like growth factor-1 (IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID).
- Conditions
- Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
- Trial name
- NCT00490100
- Testing
- Increlex
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency
Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID
The purpose of this study is to learn what factors influence adolescent girls' decisions regarding testing for carrier status of X-Linked Severe Combined Immunodeficiency (XSCID).
- Trial name
- NCT00006335
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Wiskott- Aldrich Syndrome, ADA Deficient SCID
Molecular and Clinical Studies of Primary Immunodeficiency Diseases
This study will try to identify mutations in the genes responsible for primary immunodeficiency disorders (inherited diseases of the immune system) and evaluate the course of these diseases in patients over time to learn more…
- Trial name
- NCT00006319
- Where
- United States
- Volunteers
- 266 people
- Sponsor
- National Human Genome Research Institute (NHGRI)