Hereditary Complement Deficiency Diseases: Clinical Trials

173 trials for Hereditary Complement Deficiency Diseases, newest first

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115 completed, 14 recruiting, 12 active not recruiting, 9 terminated. 61 Phase 3, 35 Phase 2, 22 Phase 1, 11 Phase 4, 5 Not applicable.

By phase and status

PhaseTrialsStatus
Phase 361Completed 44, Active not recruiting 5, Enrolling by invitation 4, Recruiting 4, Terminated 2, Withdrawn 2
Phase 235Completed 21, Active not recruiting 5, Withdrawn 3, Recruiting 2, Terminated 2, Enrolling by invitation 1, Unknown 1
Phase 122Completed 18, Active not recruiting 3, Recruiting 1
Phase 411Completed 8, Recruiting 1, Terminated 1, Withdrawn 1
Not applicable5Completed 3, Recruiting 1, Terminated 1
Early Phase 11Terminated 1

Follow Hereditary Complement Deficiency Diseases: new trials by email Literature Review of Hereditary Complement Deficiency Diseases trials Research Report on Hereditary Complement Deficiency Diseases

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
No intervention - Hereditary Angioedema (HAE)
Not yet recruitingTakeda1002026-12-012026-06-18
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
No intervention - Hereditary Angioedema (HAE)
RecruitingTakeda352026-09-212026-10-15
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)
KVD900 600 mg - Hereditary Angioedema
Phase 4RecruitingKalVista Pharmaceuticals, Ltd.1002026-08-172026-09-17
A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema
Technochrom C1-INH Kit - Hereditary Angioedema (HAE)
Not applicableRecruitingTakeda5142026-05-122026-05-19
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
No intervention - Hereditary Angioedema (HAE)
RecruitingTakeda502026-04-302026-05-11
Donidalorsen Treatment in Children With Hereditary Angioedema
Donidalorsen - Hereditary Angioedema (HAE)
Phase 3RecruitingIonis Pharmaceuticals, Inc.202026-04-212026-09-02
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 3RecruitingADARx Pharmaceuticals, Inc.902026-04-022026-09-04
Real-life Ecological Momentary Assessment of Lived Burden in Hereditary AngioEdema
ecological momentary assessment - Hereditary Angioedema With C1 Esterase Inhibitor Deficiency, Hereditary Angioedema - Type 1
RecruitingIstituti Clinici Scientifici Maugeri SpA302026-02-232026-03-04
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
No intervention - Hereditary Angioedema (HAE)
CompletedTakeda372026-02-052026-07-14
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 3RecruitingPharvaris Netherlands B.V.482025-10-162026-09-21
A Long-Term Study of Navenibart in Participants With Hereditary Angioedema
navenibart - Hereditary Angioedema (HAE)
Phase 3Enrolling by invitationAstria Therapeutics, Inc.1452025-10-012026-07-30
Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL)
Berinert, Placebo for Berinert - Kidney Transplant
Phase 2RecruitingNational Institute of Allergy and Infectious Diseases (NIAID)1802025-09-222026-02-27
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
ADX-324, Placebo - Hereditary Angioedema, HAE
Phase 3RecruitingADARx Pharmaceuticals, Inc.902025-08-282026-09-10
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
No intervention - Hereditary Angioedemas
RecruitingCSL Behring2002025-07-212026-06-03
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
Garadacimab - Hereditary Angioedema
Phase 4CompletedCSL Behring182025-04-282026-07-23
A Study Observing US Patients With HAE Type I or II Who Take Icatibant to Treat HAE Attacks
Inapplicable - Hereditary Angioedema
CompletedKalVista Pharmaceuticals, Ltd.882025-04-212026-01-13
A Study of Navenibart in Participants With Hereditary Angioedema
navenibart, Placebo - Hereditary Angioedema (HAE)
Phase 3Active not recruitingAstria Therapeutics, Inc.1572025-03-202026-06-30
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 2RecruitingInstitute for Asthma and Allergy102025-03-102025-07-02
Suicide Ideation in Hereditary Angioedema
Hereditary Angioedema (HAE), Suicidal Ideation
Not yet recruitingIvan Cherrez Ojeda1002025-03-012025-02-10
HAE Burden and Crisis Management
Hereditary Angioedema (HAE)
CompletedUniversity Hospital, Grenoble3002025-03-012026-04-29

Showing 20 of 173. Sign in to see all of them.

Narrower conditions

Angioedemas, Hereditary (169)  ·  Angioedema (26)  ·  Genetic Diseases, Inborn (9)  ·  Hereditary Angioedema Types I and II (9)  ·  Rare Diseases (6)  ·  Immune System Diseases (4)  ·  Cardiovascular Diseases (3)  ·  Hereditary Angioedema Type III (3)  ·  Hypersensitivity (3)  ·  Urticaria (3)  ·  Vascular Diseases (3)  ·  Acquired angioedema (2)  ·  Common Variable Immunodeficiency (2)  ·  Communicable Diseases (2)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (173)  ·  Immunologic Deficiency Syndromes (172)  ·  Primary Immunodeficiency Diseases (172)  ·  Immune System Diseases (169)  ·  Skin and Connective Tissue Diseases (169)  ·  Hypersensitivity, Immediate (167)  ·  Skin Diseases (167)  ·  Skin Diseases, Vascular (167)  ·  Cardiovascular Diseases (166)  ·  Hypersensitivity (166)  ·  Urticaria (166)  ·  Vascular Diseases (166)  ·  Genetic Diseases, Inborn (164)  ·  Angioedema (143)

Interventions

Complement C1 Inhibitor Protein (25)  ·  berotralstat (12)  ·  icatibant (12)  ·  Sodium Chloride (10)  ·  conestat alfa (10)  ·  lanadelumab (10)  ·  sebetralstat (9)  ·  SERPING1 protein, human (8)  ·  donidalorsen (6)  ·  ecallantide (6)  ·  IONIS-PKK-LRx (5)  ·  avoralstat (5)  ·  Cyclosporine (2)  ·  RNA, Small Interfering (2)  ·  Anti-Bacterial Agents (1)

Sponsors

Shire (39)  ·  Takeda (19)  ·  BioCryst Pharmaceuticals (17)  ·  KalVista Pharmaceuticals, Ltd. (12)  ·  CSL Behring (11)  ·  Pharming Technologies B.V. (8)  ·  Pharvaris Netherlands B.V. (8)  ·  Ionis Pharmaceuticals, Inc. (6)  ·  Astria Therapeutics, Inc. (5)  ·  University Hospital, Grenoble (4)  ·  ADARx Pharmaceuticals, Inc. (3)  ·  CENTOGENE GmbH Rostock (3)  ·  Intellia Therapeutics (3)  ·  Istituti Clinici Scientifici Maugeri SpA (3)  ·  AO GENERIUM (2)

Countries

United States (85)  ·  Germany (60)  ·  United Kingdom (52)  ·  Canada (48)  ·  Italy (44)  ·  France (38)  ·  Spain (37)  ·  Poland (33)  ·  Israel (32)  ·  Hungary (31)  ·  Netherlands (27)  ·  Australia (26)  ·  Austria (23)  ·  Bulgaria (22)  ·  Japan (22)

Top investigators

Anurag Relan, MD (4)  ·  Lauge Farnaes, MD (3)  ·  Peter Bauer, MD (3)  ·  Joanne Collier, MBChB, FFPM, Dip Stats (OU) (2)  ·  Oksana A. Markova, MD (2)  ·  Adrian Gadano, MD (1)  ·  Aleena Banerji, M.D. (1)  ·  Anne Pagnier (1)  ·  Bruce Zuraw, MD (1)  ·  Bruno Giannetti, MD (1)  ·  David Hoover, MD (1)

Results publications

PMID 36326435, PMID 36408587, PMID 20818886, PMID 23312695, PMID 23878046, PMID 24712435, PMID 23548529, PMID 42159953, PMID 42268494, PMID 20818888, PMID 20920772, PMID 21130380, PMID 22765833, PMID 22856635, PMID 23484892, PMID 23548526, PMID 23987198, PMID 25198193, PMID 28284978, PMID 36153561, PMID 36774155, PMID 37028510, PMID 40171989, PMID 40380356, PMID 40769455, PMID 40886933, PMID 42318595, PMID 1538192, PMID 15577929, PMID 16375711, PMID 17073887, PMID 19767078, PMID 20674826, PMID 20818887, PMID 21195947, PMID 21884533, PMID 21988742, PMID 22123383, PMID 22192966, PMID 22856636, PMID 22909164, PMID 23167682, PMID 23535096, PMID 24111645, PMID 24266596, PMID 24438203, PMID 24749847, PMID 24980392, PMID 25295804, PMID 25426834

NIH funding

2018 (3), 2004 (2), 2015 (2), 2023 (2), 2008 (1), 2009 (1), 2019 (1), 2025 (1)