96 completed, 11 active not recruiting, 5 recruiting, 5 terminated. 128 Phase 3, 15 Phase 2.
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 |
|---|---|---|---|---|---|---|
| IVIG Boya: Safety, Efficacy, and Pharmacokinetics Boya IVIG - Primary Immunodeficiency Disease | Phase 3 | Not yet recruiting | Azidus Brasil | 50 | 2026-09-01 | 2026-06-03 |
| Donidalorsen Treatment in Children With Hereditary Angioedema Donidalorsen - Hereditary Angioedema (HAE) | Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc. | 20 | 2026-04-21 | 2026-09-02 |
| Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) ADX-324 Dose Level 1, ADX-324 Dose Level 2 - Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1 | Phase 3 | Recruiting | ADARx Pharmaceuticals, Inc. | 90 | 2026-04-02 | 2026-09-04 |
| Study of BP-SCIG 20% in Patients With Primary Immunodeficiency (PID) BP-SCIG 20% - Primary Immunodeficiency Diseases | Phase 3 | Recruiting | Biopharma Plasma LLC | 56 | 2025-12-17 | 2026-01-16 |
| Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency Deucrictibant, Placebo - Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH) | Phase 3 | Recruiting | Pharvaris Netherlands B.V. | 48 | 2025-10-16 | 2026-09-21 |
| A Long-Term Study of Navenibart in Participants With Hereditary Angioedema navenibart - Hereditary Angioedema (HAE) | Phase 3 | Enrolling by invitation | Astria Therapeutics, Inc. | 145 | 2025-10-01 | 2026-07-30 |
| STOP-HAE: A Phase 3 Study of ADX-324 in HAE ADX-324, Placebo - Hereditary Angioedema, HAE | Phase 3 | Recruiting | ADARx Pharmaceuticals, Inc. | 90 | 2025-08-28 | 2026-09-10 |
| Efficacy, Safety and Pharmacokinetic of Virchow IVIG in PID Patients IVIG-VImmune, IVIG-Immuglo - Primary Immunodeficiency Diseases (PID) | Phase 3 | Not yet recruiting | Virchow Group | 44 | 2025-07-01 | 2025-06-12 |
| 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 | 2025-07-03 |
| Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE Deucrictibant - Hereditary Angioedema (HAE) | Phase 3 | Enrolling by invitation | Pharvaris Netherlands B.V. | 170 | 2025-02-01 | 2026-09-22 |
| 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 |
| Open-Label Extension of EryDex Study IEDAT-04-2022 Dexamethasone sodium phosphate - Ataxia Telangiectasia | Phase 3 | Terminated | Quince Therapeutics S.p.A. | 101 | 2024-12-11 | 2026-05-05 |
| Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE Deucrictibant, Placebo - Hereditary Angioedema (HAE) | Phase 3 | Active not recruiting | Pharvaris Netherlands B.V. | 81 | 2024-12-01 | 2026-02-13 |
| 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 |
| Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II KVD900 150 mg, KVD900 300 mg, KVD900 600 mg - Hereditary Angioedema | Phase 3 | Completed | KalVista Pharmaceuticals, Ltd. | 36 | 2024-08-01 | 2026-08-05 |
| Evaluate the Neurological Effects of EryDex on Subjects With A-T Dexamethasone sodium phosphate, Placebo - Ataxia Telangiectasia | Phase 3 | Completed | Quince Therapeutics S.p.A. | 105 | 2024-06-24 | 2026-04-30 |
| Efficacy, Safety, and Pharmacokinetics of Shu Yang IVIG IVIG - Primary Immunodeficiency Disease | Phase 3 | Unknown | Azidus Brasil | 50 | 2024-04-01 | 2024-03-12 |
| Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema Deucrictibant, Placebo - Hereditary Angioedema, Hereditary Angioedema Type I | Phase 3 | Completed | Pharvaris Netherlands B.V. | 134 | 2024-02-26 | 2025-11-21 |
| A Study About How TAK-881 is Processed by the Body and Side Effects in People With Primary Immunodeficiency Diseases TAK-881, HYQVIA - Primary Immunodeficiency Diseases (PID) | Phase 2 / Phase 3 | Completed | Takeda | 65 | 2023-10-24 | 2026-09-30 |
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Angioedemas, Hereditary (60) · Angioedema (8) · Common Variable Immunodeficiency (6) · Agammaglobulinemia (5) · Ataxia Telangiectasia (5) · Immunologic Deficiency Syndromes (5) · Bruton type agammaglobulinemia (4) · Hyper-IgM Immunodeficiency Syndrome (4) · Genetic Diseases, Inborn (3) · Hereditary Angioedema Types I and II (3) · Severe Combined Immunodeficiency (3) · Wiskott-Aldrich Syndrome (3) · Hereditary Angioedema Type III (2) · Immune System Diseases (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (128) · Immune System Diseases (126) · Genetic Diseases, Inborn (125) · Immunologic Deficiency Syndromes (123) · Cardiovascular Diseases (66) · Vascular Diseases (66) · Skin Diseases (61) · Skin Diseases, Vascular (61) · Skin and Connective Tissue Diseases (61) · Hereditary Complement Deficiency Diseases (60) · Hypersensitivity, Immediate (60) · Urticaria (60) · Hypersensitivity (59) · Angioedema (52)
gamma-Globulins (24) · Complement C1 Inhibitor Protein (12) · Hizentra (7) · Sodium Chloride (7) · icatibant (7) · lanadelumab (6) · Immunoglobulins, Intravenous (5) · berotralstat (5) · Fluid Therapy (4) · Solutions (4) · ecallantide (4) · sebetralstat (4) · Caprylates (3) · Immunoglobulin G (3) · donidalorsen (3)
Shire (21) · CSL Behring (12) · Baxalta now part of Shire (9) · Takeda (8) · BioCryst Pharmaceuticals (7) · Octapharma (7) · Grifols Therapeutics LLC (5) · Pharvaris Netherlands B.V. (5) · Bio Products Laboratory (4) · KalVista Pharmaceuticals, Ltd. (4) · CSL Limited (3) · Ionis Pharmaceuticals, Inc. (3) · Kedrion S.p.A. (3) · Pharming Technologies B.V. (3) · Quince Therapeutics S.p.A. (3)
United States (86) · Canada (45) · Germany (42) · Spain (36) · United Kingdom (36) · Italy (31) · Hungary (29) · Poland (29) · France (25) · Japan (25) · Australia (24) · Israel (24) · Netherlands (18) · Bulgaria (16) · Austria (15)
Chaim Roifman, MD (2) · Dirk Thye, MD (2) · Eric Wolford (2) · James Moy, MD (2) · Lauge Farnaes, MD (2) · Luciana Ferrara (2) · Akhilesh Chouksey, MD (1) · Alexei Maschan, Professor (1) · Angela Smith, MD (1) · Anurag Relan, MD (1) · Bruce Zuraw, MD (1)
PMID 36326435, PMID 20818886, PMID 23312695, PMID 23548529, PMID 23878046, PMID 24712435, PMID 34931880, PMID 36408587, PMID 42159953, PMID 20818888, PMID 20920772, PMID 21130380, PMID 22765833, PMID 22856635, PMID 23484892, PMID 23548526, PMID 23987198, PMID 24412910, PMID 27220317, PMID 28284978, PMID 30626238, PMID 31268374, PMID 34036490, PMID 36153561, PMID 36818468, PMID 37028510, PMID 40380356, PMID 40886933, PMID 42268494, PMID 17073887, PMID 19767078, PMID 20454851, PMID 20674826, PMID 20818887, PMID 21195947, PMID 21424824, PMID 21553933, PMID 21884533, PMID 21988742, PMID 22123383, PMID 22192966, PMID 22212346, PMID 22526590, PMID 22846381, PMID 22856636, PMID 22909164, PMID 23167682, PMID 23535096, PMID 24111645, PMID 24266596
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