10 active not recruiting. 10 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 10 | Active not recruiting 10 |
Follow Primary Immunodeficiency Diseases: new trials by email Literature Review of Primary Immunodeficiency Diseases trials Research Report on Primary Immunodeficiency Diseases
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 |
|---|---|---|---|---|---|---|
| A Study of Navenibart in Participants With Hereditary Angioedema navenibart, Placebo - Hereditary Angioedema (HAE) | Phase 3 | Active not recruiting | Astria Therapeutics, Inc. | 157 | 2025-03-20 | 2026-06-30 |
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) N-Acetyl-L-Leucine, Placebo - Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2025-03-18 | 2026-11-04 |
| HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) NTLA-2002, Normal Saline IV Administration - Hereditary Angioedema | Phase 3 | Active not recruiting | Intellia Therapeutics | 80 | 2025-01-15 | 2026-10-08 |
| A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases TAK-881, Koru 24 G HIgH Flo Subcutaneous Safety Needle Set (investigational in the US) - Primary Immunodeficiency Diseases (PID) | Phase 3 | Active not recruiting | Takeda | 53 | 2024-11-04 | 2026-04-08 |
| Berotralstat Treatment in Children With Hereditary Angioedema Berotralstat - Hereditary Angioedema, Pediatric | Phase 3 | Active not recruiting | BioCryst Pharmaceuticals | 29 | 2022-10-25 | 2026-02-25 |
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) Donidalorsen - Hereditary Angioedema | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 154 | 2022-07-13 | 2025-12-12 |
| Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases Newnorm - Primary Immune Deficiency | Phase 3 | Active not recruiting | Octapharma | 50 | 2021-08-04 | 2026-11-09 |
| Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10) Kedrion IVIG 10% - Primary Immunodeficiency Disease | Phase 3 | Active not recruiting | Kedrion S.p.A. | 30 | 2021-03-31 | 2026-05-29 |
| Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency GC5107 - Primary Immune Deficiency | Phase 3 | Active not recruiting | GC Biopharma Corp | 24 | 2020-12-21 | 2025-07-25 |
| A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome OTL-103 - Wiskott-Aldrich Syndrome | Phase 3 | Active not recruiting | Fondazione Telethon | 10 | 2019-01-21 | 2025-09-29 |
Angioedemas, Hereditary (4) · Agammaglobulinemia (1) · Angioedema (1) · Ataxia Telangiectasia (1) · Wiskott-Aldrich Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Immune System Diseases (10) · Immunologic Deficiency Syndromes (10) · Cardiovascular Diseases (5) · Vascular Diseases (5) · Hereditary Complement Deficiency Diseases (4) · Hypersensitivity (4) · Hypersensitivity, Immediate (4) · Skin Diseases (4) · Skin Diseases, Vascular (4) · Skin and Connective Tissue Diseases (4) · Urticaria (4) · Angioedema (3)
gamma-Globulins (2) · Fluid Therapy (1) · IB1001 (1) · IONIS-PKK-LRx (1) · Solutions (1) · acetylleucine (1) · berotralstat (1) · donidalorsen (1)
Astria Therapeutics, Inc. (1) · BioCryst Pharmaceuticals (1) · Fondazione Telethon (1) · GC Biopharma Corp (1) · Intellia Therapeutics (1) · IntraBio Inc (1) · Ionis Pharmaceuticals, Inc. (1) · Kedrion S.p.A. (1) · Octapharma (1) · Takeda (1)
United States (9) · Germany (6) · Italy (6) · Poland (5) · Spain (5) · United Kingdom (5) · Canada (4) · France (4) · Slovakia (4) · Hungary (3) · Israel (3) · Netherlands (3) · Australia (2) · Austria (2) · Bulgaria (2)
Chiara Azzari (1) · Jolanta Bernatoniene, MD (1)
PMID 38446574, PMID 40673861, PMID 40716738, PMID 41767175, PMID 42294842, PMID 42309084, PMID 42777242
2018 (1), 2023 (1)