Primary Immunodeficiency Diseases: Clinical Trials

297 completed, 61 recruiting, 61 unknown, 31 active not recruiting. 128 Phase 2, 128 Phase 3, 86 Phase 1, 40 Not applicable, 31 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	128	Completed 66, Recruiting 16, Terminated 15, Active not recruiting 12, Unknown 7, Withdrawn 6, Enrolling by invitation 3, Not yet recruiting 2, Suspended 1
Phase 3	128	Completed 96, Active not recruiting 11, Recruiting 5, Terminated 5, Enrolling by invitation 4, Withdrawn 4, Not yet recruiting 2, Unknown 1
Phase 1	86	Completed 50, Recruiting 10, Active not recruiting 8, Unknown 7, Terminated 6, Enrolling by invitation 2, Not yet recruiting 1, Suspended 1, Withdrawn 1
Not applicable	40	Completed 18, Unknown 12, Recruiting 3, Terminated 3, Active not recruiting 2, Withdrawn 2
Phase 4	31	Completed 21, Unknown 4, Terminated 2, Withdrawn 2, Not yet recruiting 1, Recruiting 1
Early Phase 1	4	Not yet recruiting 2, Completed 1, Terminated 1

Follow Primary Immunodeficiency Diseases: new trials by email Literature Review of Primary Immunodeficiency Diseases trials Research Report on Primary Immunodeficiency Diseases

Trials newest first · largest first

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
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt No intervention - Hereditary Angioedema (HAE)		Not yet recruiting	Takeda	100	2026-12-01	2026-06-18
Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department Haloperidol - Cannabinoid Hyperemesis Syndrome	Early Phase 1	Not yet recruiting	University of Illinois at Chicago	100	2026-11-01	2026-09-01
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins TAK-664 - Primary Immunodeficiency Diseases (PID)	Phase 4	Not yet recruiting	Takeda	9	2026-11-01	2026-09-08
Follow-up of the Cohort of Newborns Screened at Birth Using TREC Analysis Data collection - Severe Combined Immunodeficiencies (SCID)		Not yet recruiting	Nantes University Hospital	450	2026-11-01	2026-07-15
25BT056: Basket Study of Viral Cytotoxic T Lymphocytes Virus-Specific Cytotoxic T Lymphocytes (vCTLs) - EBV Viremia, CMV Viremia	Early Phase 1	Not yet recruiting	Children's Hospital of Philadelphia	35	2026-11-01	2026-10-22
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea No intervention - Hereditary Angioedema (HAE)		Recruiting	Takeda	35	2026-09-21	2026-10-15
Knowledge of Inborn Errors of Immunity Among Pediatric Physicians Primary Immunodeficiency Diseases		Not yet recruiting	Assiut University	347	2026-09-20	2026-09-25
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
Biomarkers for Babies and Young Children With Ataxia Telangiectasia Ataxia Telangiectasia		Not yet recruiting	University of Nottingham	56	2026-09-01	2026-07-16
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs - Bone Marrow Failure, Hemoglobinopathies	Phase 2	Not yet recruiting	Fred Hutchinson Cancer Center	40	2026-09-01	2026-06-22
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL) KVD900 600 mg - Hereditary Angioedema	Phase 4	Recruiting	KalVista Pharmaceuticals, Ltd.	100	2026-08-17	2026-09-17
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
Exercise Intervention and Immunodeficiency App-based exercise intervention - Primary Antibody Deficiency Disorders	Not applicable	Recruiting	Yale University	20	2026-07-17	2026-07-23
Primitive Immunodeficiency, Intrauterine Devices and Menstrual Hygiene Products Questionnaire - Primary Immune Deficiency Diseases		Recruiting	Assistance Publique - Hôpitaux de Paris	200	2026-06-01	2026-10-22
A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema Technochrom C1-INH Kit - Hereditary Angioedema (HAE)	Not applicable	Recruiting	Takeda	514	2026-05-12	2026-05-19
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder	Phase 2	Recruiting	Paul Szabolcs	25	2026-05-04	2026-05-15
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia No intervention - Hereditary Angioedema (HAE)		Recruiting	Takeda	50	2026-04-30	2026-05-11
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

Showing 20 of 535. Sign in to see all of them.

Narrower conditions

Angioedemas, Hereditary (169)  ·  Severe Combined Immunodeficiency (57)  ·  Ataxia Telangiectasia (42)  ·  Wiskott-Aldrich Syndrome (41)  ·  Immunologic Deficiency Syndromes (38)  ·  X-Linked Combined Immunodeficiency Diseases (28)  ·  Angioedema (26)  ·  Common Variable Immunodeficiency (26)  ·  Granulomatous Disease, Chronic (22)  ·  Lymphohistiocytosis, Hemophagocytic (20)  ·  Immune System Diseases (19)  ·  Anemia, Sickle Cell (18)  ·  Genetic Diseases, Inborn (18)  ·  Hyper-IgM Immunodeficiency Syndrome (18)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (535)  ·  Genetic Diseases, Inborn (517)  ·  Immune System Diseases (516)  ·  Immunologic Deficiency Syndromes (497)  ·  Cardiovascular Diseases (221)  ·  Vascular Diseases (214)  ·  Skin and Connective Tissue Diseases (206)  ·  Skin Diseases (195)  ·  Skin Diseases, Vascular (171)  ·  Hereditary Complement Deficiency Diseases (169)  ·  Hypersensitivity, Immediate (169)  ·  Hypersensitivity (168)  ·  Urticaria (166)  ·  Angioedema (143)

Interventions

gamma-Globulins (34)  ·  Complement C1 Inhibitor Protein (25)  ·  Busulfan (17)  ·  Hizentra (14)  ·  Cyclophosphamide (12)  ·  berotralstat (12)  ·  fludarabine (12)  ·  icatibant (12)  ·  Alemtuzumab (11)  ·  Sodium Chloride (11)  ·  Solutions (10)  ·  conestat alfa (10)  ·  fludarabine phosphate (10)  ·  lanadelumab (10)  ·  Melphalan (9)

Sponsors

Shire (39)  ·  National Institute of Allergy and Infectious Diseases (NIAID) (35)  ·  Takeda (28)  ·  CSL Behring (22)  ·  BioCryst Pharmaceuticals (17)  ·  Baxalta now part of Shire (14)  ·  KalVista Pharmaceuticals, Ltd. (12)  ·  National Human Genome Research Institute (NHGRI) (10)  ·  Octapharma (10)  ·  Pharming Technologies B.V. (9)  ·  Grifols Therapeutics LLC (8)  ·  Pharvaris Netherlands B.V. (8)  ·  Federal Research Institute of Pediatric Hematology, Oncology and Immunology (7)  ·  Johann Wolfgang Goethe University Hospital (7)  ·  Assistance Publique - Hôpitaux de Paris (6)

Countries

United States (285)  ·  Germany (83)  ·  United Kingdom (81)  ·  France (75)  ·  Canada (72)  ·  Italy (61)  ·  Spain (49)  ·  Poland (43)  ·  Israel (42)  ·  Hungary (39)  ·  Australia (37)  ·  Netherlands (31)  ·  Japan (30)  ·  Czechia (26)  ·  Austria (25)

Top investigators

Paul Szabolcs, MD (5)  ·  Stefan Zielen, Prof. Dr. (5)  ·  Anurag Relan, MD (4)  ·  Suk S De Ravin, M.D. (4)  ·  Gulbu Uzel, M.D. (3)  ·  Lauge Farnaes, MD (3)  ·  Mitchell S Cairo, MD (3)  ·  Nizar Mahlaoui, MD (3)  ·  Peter Bauer, MD (3)  ·  Alexei Maschan, Professor (2)  ·  Angela Smith, MD (2)  ·  Chaim Roifman, MD (2)

Results publications

PMID 36326435, PMID 36408587, PMID 20818886, PMID 23312695, PMID 23878046, PMID 24712435, PMID 25075835, PMID 20004776, PMID 23548529, PMID 23818196, PMID 24139498, PMID 24290292, PMID 24331379, PMID 25295500, PMID 27262745, PMID 30261899, PMID 34931880, PMID 35075289, PMID 36818468, PMID 42159953, PMID 42268494, PMID 20818888, PMID 20920772, PMID 21130380, PMID 21725047, PMID 22765833, PMID 22856635, PMID 23484892, PMID 23548526, PMID 23987198, PMID 24077845, PMID 24412910, PMID 25198193, PMID 27220317, PMID 28284978, PMID 30193840, PMID 30626238, PMID 31268374, PMID 34036490, PMID 34477998, PMID 35671392, PMID 36153561, PMID 36774155, PMID 37028510, PMID 38446574, PMID 40171989, PMID 40380356, PMID 40739200, PMID 40769455, PMID 40886933

NIH funding

2018 (6), 2015 (5), 2009 (4), 2008 (3), 2016 (3), 2023 (3), 2004 (2), 2019 (2), P30CA015704 (2), U54HL096458 (2), 2014 (1), 2025 (1), K12HD043494 (1), P01HL036444 (1), P01HL053749 (1)