Clinical trials: Adenosine Deaminase Deficiency, open to volunteers
Severe Combined Immunodeficiency (SCID), Leaky SCID
Natural History Study of SCID Disorders
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions.
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Trial name
- NCT01186913
- Where
- 44 sites in United States, Canada
- Volunteers
- 690 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the…
- Trial name
- NCT03645460
- Testing
- Direct intravenous injection of ivlv-ADA lentiviral vector
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human…
- Trial name
- NCT04049084
- Testing
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 70 people
- Sponsor
- University of California, Los Angeles
Immunologic Deficiency Syndromes
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder.
- Trial name
- NCT03478670
- Testing
- Strimvelis
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Fondazione Telethon