Severe combined immunodeficiency due to adenosine deaminase deficiency clinical trials
The registry lists 29 studies for Severe combined immunodeficiency due to adenosine deaminase deficiency across 6 countries. 4 are recruiting or about to start. 23 of them are mainly about Severe combined immunodeficiency due to adenosine deaminase deficiency (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-02.
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Status
Who runs them
University of California, Los Angeles (5) · National Human Genome Research Institute (NHGRI) (4) · Fondazione Telethon (4) · TRPHARM (3) · Great Ormond Street Hospital for Children NHS Foundation Trust (3) · National Institute of Allergy and Infectious Diseases (NIAID) (2) · Istituto Giannina Gaslini (1) · Fred Hutchinson Cancer Center (1)
What is being tested here
Drugs under test in Severe combined immunodeficiency due to adenosine deaminase deficiency studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Busulfan | 0 | 0 | 4 |
| Cyclosporine | 0 | 0 | 1 |
| Mycophenolic Acid | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Nov 2027Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector · University of California, Los Angeles
Who these studies take
No Severe combined immunodeficiency due to adenosine deaminase deficiency study is recruiting now.
Across all 29 studies, recruiting or not: 52% stop at 75 or younger and 52% at 65; 90% take children; 0% are for women only and 0% for men only; 7% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 16, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 29 of 29. search them.
- Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)Not yet recruitingNCT03645460Not applicable1 country
- Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)Enrolling by invitationNCT034786701 country
- An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDEnrolling by invitationNCT040490842 countries
- Natural History Study of SCID DisordersEnrolling by invitationNCT011869132 countries
- Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy ApproachCompletedNCT07882680Not applicable1 country
- Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA VectorActive, not recruitingNCT05432310Phase 1/21 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Registry Study of Revcovi Treatment in Patients With ADA-SCIDCompletedNCT038780691 country
- Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene TherapyActive, not recruitingNCT049598901 country
- Retroviral Insertion Site Methodology StudyWithdrawnNCT03311074Phase 4
- Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)CompletedNCT032322031 country
- Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDCompletedNCT03765632Phase 1/21 country
- Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA GeneCompletedNCT01852071Phase 1/21 country
- Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDCompletedNCT02999984Phase 1/21 country
- A Clinical Study to Enable Process Validation of Commercial Grade OTL-101WithdrawnNCT04140539Phase 2/31 country
- Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin EStatus unknownNCT053003731 country
- Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)Status unknownNCT053003471 country
- Investigation of ADA Enzyme DeficiencyStatus unknownNCT053003341 country
- Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyCompletedNCT01380990Phase 1/21 country
- The Role of Bacteria and Genetic Variations in Cystic FibrosisCompletedNCT000432251 country
- MND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDCompletedNCT00794508Phase 21 country
- Patients Treated for SCID (1968-Present)Status unknownNCT013461502 countries
- EZN-2279 in Patients With ADA-SCIDCompletedNCT01420627Phase 31 country
- Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their ParentsWithdrawnNCT01182857
- Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) DeficiencyCompletedNCT00018018Phase 11 country
- Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantCompletedNCT00008450Phase 11 country
- Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral VectorCompletedNCT02022696Phase 11 country
- Gene Therapy ADA DeficiencyCompletedNCT01279720Phase 1/21 country
- Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History StudyCompletedNCT000012551 country