Clinical trials: ADA SCID
ADA-SCID
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA…
- Trial name
- NCT01852071
- Testing
- Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- University of California, Los Angeles
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)
Adenosine Deaminase Deficiency
Investigation of ADA Enzyme Deficiency
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study and were found to be lymphopenic in at least one examination will be included…
- Trial name
- NCT05300334
- Testing
- adenosine deaminase test
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people
- Sponsor
- TRPHARM
SCID, ADA-SCID
Patients Treated for SCID (1968-Present)
Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme…
- Conditions
- SCID, ADA-SCID, XSCID, Leaky SCID
- Trial name
- NCT01346150
- Where
- 36 sites in United States, Canada
- Volunteers
- 1,007 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency Due to ADA Deficiency
Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects…
- Trial name
- NCT04959890
- Testing
- Strimvelis
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Fondazione Telethon
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
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
Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach
Adenosine deaminase 2 deficiency (DADA2), caused by autosomal recessive mutations in the ADA2 gene (OMIM: 607575), is an autoinflammatory disease characterized by early-onset inflammatory vasculopathy, stroke, and…
- Trial name
- DADA2 (NCT07882680)
- Testing
- bone marrow aspiration and biopsy
- Where
- Italy
- Volunteers
- 29 people
- Sponsor
- Istituto Giannina Gaslini
Adenosine Deaminase Severe Combined Immune Deficiency
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human…
- Trial name
- NCT05432310
- Testing
- A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, Los Angeles