Severe Combined Immunodeficiency clinical trials
The registry lists 59 studies for Severe Combined Immunodeficiency across more than 10 countries. 14 are recruiting or about to start. 29 of them are mainly about Severe Combined Immunodeficiency (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10. Counting its 2 narrower conditions too, such as Severe combined immunodeficiency due to adenosine deaminase deficiency: 96 studies, 23 open.
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Who runs them
National Institute of Allergy and Infectious Diseases (NIAID) (5) · National Human Genome Research Institute (NHGRI) (5) · Paul Szabolcs (4) · University of California, San Francisco (4) · Assistance Publique - Hôpitaux de Paris (3) · St. Jude Children's Research Hospital (3) · National Heart, Lung, and Blood Institute (NHLBI) (3) · Centre Leon Berard (2)
What is being tested here
Drugs under test in Severe Combined Immunodeficiency 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 | 1 | 0 | 4 |
| Fludarabine | 1 | 0 | 4 |
| Alemtuzumab | 1 | 0 | 3 |
| Melphalan | 1 | 0 | 3 |
| Hydroxyurea | 1 | 0 | 1 |
| Thiotepa | 1 | 0 | 1 |
| Cyclophosphamide | 0 | 1 | 2 |
| Fludarabine phosphate | 0 | 0 | 2 |
| Antilymphocyte Serum | 0 | 1 | 2 |
| Mesna | 0 | 0 | 1 |
Drug classes tested here Alkylating agents 2 open · Antimetabolites 1 open
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 3
- Nov 2026Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases · Paul Szabolcs
- Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
- Jul 2027Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) · St. Jude Children's Research Hospital
Who these studies take
Of the 7 Severe Combined Immunodeficiency studies recruiting now, 2 (29%) set no upper age limit and 4 (57%) stop at 75 or younger. 6 (86%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (71%) | 5 (71%) |
| 18 | 6 (86%) | 6 (86%) |
| 30 | 6 (86%) | 6 (86%) |
| 40 | 6 (86%) | 6 (86%) |
| 50 | 5 (71%) | 5 (71%) |
| 65 | 3 (43%) | 3 (43%) |
| 75 | 3 (43%) | 3 (43%) |
| 85 | 3 (43%) | 3 (43%) |
Across all 59 studies, recruiting or not: 63% stop at 75 or younger and 59% at 65; 95% take children; 2% are for women only and 2% for men only; 12% 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 13, 2026.
Results posted1
- Immune Disorder HSCT ProtocolOct 27, 2026NCT01821781
Newly registered1
- 25BT056: Basket Study of Viral Cytotoxic T LymphocytesOct 14, 2026NCT07873970
Studies
Recruiting first, then most recently updated. Showing 50 of 59. Browse all 59 → or search them.
- Genetic Basis of ImmunodeficiencyRecruitingNCT000551721 country
- Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation DisordersEnrolling by invitationNCT008952711 country
- 25BT056: Basket Study of Viral Cytotoxic T LymphocytesNot yet recruitingNCT07873970Early Phase 1
- Follow-up of the Cohort of Newborns Screened at Birth Using TREC AnalysisNot yet recruitingNCT077042811 country
- Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)RecruitingNCT07284641Phase 21 country
- Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)RecruitingNCT05071222Phase 1/21 country
- Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell BoostsAvailableNCT06731036
- Autologous Gene Therapy for Artemis-Deficient SCIDRecruitingNCT03538899Phase 1/21 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDEnrolling by invitationNCT040490842 countries
- Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency DiseasesEnrolling by invitationNCT01852370Phase 1/21 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- The Experience of Screening for SCIDRecruitingNCT056511131 country
- Natural History Study of SCID DisordersEnrolling by invitationNCT011869132 countries
- Immune Disorder HSCT ProtocolCompletedNCT01821781Phase 21 country
- Study of Populations at Risk of Developing Chronic Hepatitis Linked to Chronic Enteric Virus Infection in Patients With Primary Immunodeficiency and Secondary Humoral DeficiencyCompletedNCT066595881 country
- Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Active, not recruitingNCT03597594Phase 1/21 country
- Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCIDSuspendedNCT04797260Phase 1/24 countries
- Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesActive, not recruitingNCT01652092Not applicable1 country
- Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID PatientsCompletedNCT03879876Phase 1/21 country
- Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral VectorCompletedNCT01129544Phase 1/21 country
- Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusWithdrawnNCT04370795Phase 1/21 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesCompletedNCT01019876Phase 2/31 country
- Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked ProductsCompletedNCT006952791 country
- Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell TransplantationCompletedNCT05298930Not applicable1 country
- Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesTerminatedNCT02231710Phase 11 country
- Retroviral Insertion Site Methodology StudyWithdrawnNCT03311074Phase 4
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationCompletedNCT03513328Phase 1/21 country
- A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell AllograftsWithdrawnNCT04331483Not applicable1 country
- Neonatal Screening of Severe Combined ImmunodeficienciesCompletedNCT025903281 country
- Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyCompletedNCT01380990Phase 1/21 country
- Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow AllograftCompletedNCT042468401 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
- Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined ImmunodeficiencyStatus unknownNCT02860559Phase 11 country
- 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
- Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesCompletedNCT01529827Phase 21 country
- Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantCompletedNCT00008450Phase 11 country
- Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & FilgrastimTerminatedNCT01182675Phase 21 country
- Generalized Neonatal Screening of Severe Combined ImmunodeficienciesCompletedNCT02244450Not applicable1 country
- SCID Bu/Flu/ATG Study With T Cell DepletionTerminatedNCT02127892Phase 1/21 country
- Developing Newborn Screening for Infants With Primary ImmunodeficiencyCompletedNCT001134641 country
- Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)CompletedNCT00028236Phase 11 country
- Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCIDCompletedNCT000063351 country
- Cord Blood Stem Cell Transplantation Study (COBLT)CompletedNCT00000603Phase 2
- Sirolimus Prophylaxis for aGVHD in TME SCIDWithdrawnNCT02177760Phase 21 country