Clinical trials: ADA SCID
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 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
Severe Combined Immunodeficiency Due to ADA Deficiency
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic…
- Trial name
- NCT02999984
- Testing
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
- Where
- United States
- Volunteers
- 10 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
Adenosine Deaminase Deficiency
Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E
This study was designed as an observational, prospective, epidemiological screening study. Patients who have been admitted to the center and whose lymphopenia and/or Immunoglobulin E elevation has been detected in at least one…
- Trial name
- NCT05300373
- Testing
- ADENOSINE DEAMINASE TEST
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- TRPHARM
ADA-SCID
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Background: Severe combined immunodeficiency (SCID) is a rare inherited disorder in which certain white blood cells have impaired function and are unable to properly fight infections.
- Trial name
- NCT01182857
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Severe Combined Immunodeficiency
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA).
- Trial name
- NCT00001255
- Testing
- ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.
Immune System Diseases
Retroviral Insertion Site Methodology Study
Adenosine deaminase (ADA) is an enzyme involved in the development and functioning of the immune system. Deficiency of ADA results in severe combined immunodeficiency (SCID), a fatal inherited immune disorder.
- Trial name
- NCT03311074
- Testing
- Strimvelis
- Sponsor
- Fondazione Telethon
Adenosine Deaminase Deficiency
Gene Therapy ADA Deficiency
Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection.
- Trial name
- NCT01279720
- Testing
- Intravenous infusion of transduced cells
- Where
- United Kingdom
- Volunteers
- 8 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust