50 completed, 10 recruiting, 8 active not recruiting, 7 unknown. 86 Phase 1, 48 Phase 2.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection Direct intravenous injection of ivlv-X1 lentiviral vector - SCID, X-Linked | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-30 | 2026-08-27 |
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy Plerixafor, Filgrastim, Palifermin - X-linked Severe Combined Immunodeficiency, X-SCID | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 18 | 2025-05-09 | 2026-10-13 |
| Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients Deucrictibant XR tablet, Placebo comparator to XR tablet, Deucrictibant 20 mg capsule - Hereditary Angioedema (HAE), Angioedema | Phase 1 / Phase 2 | Recruiting | Institute for Asthma and Allergy | 10 | 2025-03-10 | 2025-07-02 |
| 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) ARTEGENE drug product - Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 7 | 2023-07-19 | 2026-03-27 |
| A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema Navenibart - Hereditary Angioedema | Phase 1 / Phase 2 | Completed | Astria Therapeutics, Inc. | 29 | 2023-02-21 | 2026-05-08 |
| Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients ADX-324, Placebo - Hereditary Angioedema | Phase 1 / Phase 2 | Active not recruiting | ADARx Pharmaceuticals, Inc. | 53 | 2022-12-14 | 2026-06-16 |
| Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2 - Ataxia Telangiectasia | Phase 1 | Unknown | Grace Therapeutics Inc. | 48 | 2022-09-13 | 2023-01-23 |
| A Study of STAR-0215 in Healthy Adult Participants STAR-0215 (SC), Placebo (SC), STAR-0215 (IV) - Hereditary Angioedema | Phase 1 | Completed | Astria Therapeutics, Inc. | 41 | 2022-07-27 | 2024-01-11 |
| NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) gp91 Grans - Chronic Granulomatous Disease, Infection | Phase 1 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 25 | 2022-07-22 | 2026-10-29 |
| Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts Norovirus -specific T-cell (NST) therapy - Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT) | Phase 1 | Recruiting | Children's National Research Institute | 48 | 2022-03-17 | 2026-01-26 |
| A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331 - Hereditary Angioedema, HAE | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 44 | 2022-02-15 | 2024-05-16 |
| NTLA-2002 in Adults With Hereditary Angioedema (HAE) Biological NTLA-2002, Normal Saline IV Administration - Hereditary Angioedema | Phase 1 / Phase 2 | Active not recruiting | Intellia Therapeutics | 37 | 2021-12-10 | 2026-09-25 |
| Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Active not recruiting | St. Jude Children's Research Hospital | 4 | 2021-09-02 | 2026-09-16 |
| Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency TBX-1400 - Severe Combined Immunodeficiency | Phase 1 | Unknown | Taiga Biotechnologies, Inc. | 8 | 2021-08-01 | 2020-10-08 |
| Evaluation of Safety, Tolerability and Immune Responses of Ebola-S and Marburg Vaccines in Healthy Adults cAd3-Marburg, cAd3-EBO-S - Ebola Virus Disease, Marburg Virus Disease | Phase 1 | Completed | Albert B. Sabin Vaccine Institute | 32 | 2021-01-06 | 2022-08-03 |
| Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs) BK CTL - Viral Infection, Primary Immune Deficiency Disorder | Phase 1 / Phase 2 | Recruiting | New York Medical College | 40 | 2020-07-01 | 2026-04-15 |
| Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations KVD824 Prototype 1 modified-release tablet, KVD824 Prototype 2 modified-release tablet, KVD824 Immediate-Release Capsule - Hereditary Angioedema | Phase 1 | Completed | KalVista Pharmaceuticals, Ltd. | 37 | 2020-05-19 | 2021-11-12 |
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Angioedemas, Hereditary (22) · Severe Combined Immunodeficiency (18) · X-Linked Combined Immunodeficiency Diseases (14) · Wiskott-Aldrich Syndrome (12) · Anemia, Aplastic (9) · Anemia, Diamond-Blackfan (9) · Anemia, Sickle Cell (8) · Granulomatous Disease, Chronic (7) · Hemoglobinopathies (6) · Immunologic Deficiency Syndromes (6) · Mucopolysaccharidosis I (6) · Myelodysplastic Syndromes (6) · Thalassemia (6) · Angioedema (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (86) · Genetic Diseases, Inborn (83) · Immune System Diseases (83) · Immunologic Deficiency Syndromes (80) · Skin and Connective Tissue Diseases (33) · Genetic Diseases, X-Linked (31) · Nutritional and Metabolic Diseases (30) · Infant, Newborn, Diseases (29) · Metabolic Diseases (29) · Hematologic Diseases (28) · Hemic and Lymphatic Diseases (28) · Skin Diseases (26) · Cardiovascular Diseases (25) · DNA Repair-Deficiency Disorders (25)
Busulfan (11) · fludarabine (7) · Alemtuzumab (5) · Thiotepa (4) · berotralstat (4) · AP 1903 reagent (3) · Fibroblast Growth Factor 7 (3) · Melphalan (3) · fludarabine phosphate (3) · Cyclosporine (2) · Filgrastim (2) · Genetic Engineering (2) · Hematopoietic Stem Cell Transplantation (2) · Sirolimus (2) · Whole-Body Irradiation (2)
National Institute of Allergy and Infectious Diseases (NIAID) (10) · BioCryst Pharmaceuticals (6) · Shire (5) · Bellicum Pharmaceuticals (4) · St. Jude Children's Research Hospital (4) · David Williams (3) · Genethon (3) · KalVista Pharmaceuticals, Ltd. (3) · Assistance Publique - Hôpitaux de Paris (2) · Astria Therapeutics, Inc. (2) · Diane George (2) · Emory University (2) · Great Ormond Street Hospital for Children NHS Foundation Trust (2) · New York Medical College (2) · ADARx Pharmaceuticals, Inc. (1)
United States (54) · United Kingdom (18) · France (6) · Italy (4) · Australia (3) · China (3) · Germany (3) · Canada (2) · Spain (2) · Bulgaria (1) · Czechia (1) · Israel (1) · Jordan (1) · Netherlands (1) · New Zealand (1)
Suk S De Ravin, M.D. (4) · Claire Booth, Dr (2) · Diane George, MD (2) · Joanne Collier, MBChB, FFPM, Dip Stats (OU) (2) · Kimberly Kasow, DO (2) · Michael Keller, MD (2) · Mitchell S Cairo, MD (2) · Paul Szabolcs, MD (2) · Adrian Thrasher, Prof (1) · Alain Fischer, MD, PhD (1) · Biljana Horn, MD (1)
PMID 35075289, PMID 25295500, PMID 30261899, PMID 1538192, PMID 15577929, PMID 16375711, PMID 18218852, PMID 19179314, PMID 19633200, PMID 19652199, PMID 21725047, PMID 22184407, PMID 24296492, PMID 24980392, PMID 25426834, PMID 26319134, PMID 26419363, PMID 28225674, PMID 28716862, PMID 29635077, PMID 30981783, PMID 30995372, PMID 33152483, PMID 33974366, PMID 34655400, PMID 35086692, PMID 35764638, PMID 37244942, PMID 37478401, PMID 38294975, PMID 38446574, PMID 39445704, PMID 40158724, PMID 41794053, PMID 42777242, PMID 8237108
2009 (2), 2008 (1), 2014 (1), 2018 (1), 2019 (1), P01HL036444 (1), P01HL053749 (1), P30CA015704 (1), U01AI125050 (1)