Clinical trials: Complement System
Hereditary Angioedema
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the clinical efficacy, safety, and tolerability of donidalorsen in participants with hereditary angioedema (HAE) type 1 (HAE-1), HAE type 2 (HAE-2), or HAE with normal C1-inhibitor…
- Trial name
- NCT04030598
- Testing
- Donidalorsen
- Where
- 8 sites in United States, Netherlands
- Volunteers
- 23 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
IgA Nephropathy
Study of Safety and Efficacy of LNP023 in Patients With Kidney Disease Caused by Inflammation
Efficacy and safety of LNP023 in IgAN patients
- Trial name
- NCT03373461
- Testing
- LNP023
- Where
- 56 sites in Argentina, Australia and 23 more
- Volunteers
- 112 people
- Sponsor
- Novartis Pharmaceuticals
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
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
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Hereditary Angioedema
Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema
The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent hereditary angioedema attacks.
- Trial name
- NCT04687137
- Testing
- TAK-743 300 mg
- Where
- 9 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Hereditary Angioedema, HAE
Oral Berotralstat Expanded Access Program
This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- NCT04428632
- Testing
- Berotralstat
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema…
- Trial name
- NCT01188564
- Testing
- rhC1INH
- Where
- 27 sites in United States, Bulgaria and 9 more
- Volunteers
- 75 people
- Sponsor
- Pharming Technologies B.V.
Hereditary Angioedema
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
The study objective was to describe the pharmacokinetics (PK) of one or two doses of C1 esterase inhibitor (C1INH-nf) in hereditary angioedema (HAE) subjects who were not experiencing an HAE attack.
- Trial name
- NCT00432510
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Shire
Hereditary Angioedema
EASSI - Evaluation of the Safety of Self-Administration With Icatibant
This study is being conducted to explore the clinical safety, local tolerability, convenience and effectiveness of self-treatment of hereditary angioedema (HAE) attacks with subcutaneous injections of icatibant.
- Trial name
- EASSI (NCT00997204)
- Testing
- Icatibant
- Where
- 26 sites in Argentina, Austria and 8 more
- Volunteers
- 151 people
- Sponsor
- Shire
Generalized Myasthenia Gravis
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
- Trial name
- NCT03920293
- Testing
- Ravulizumab
- Where
- 106 sites in United States, Austria and 13 more
- Volunteers
- 175 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hereditary Angioedema (HAE)
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE).
- Trial name
- APeX-1 (NCT02870972)
- Testing
- BCX7353
- Where
- 24 sites in Australia, Austria and 9 more
- Volunteers
- 75 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema (HAE)
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single subcutaneous (SC) administration of icatibant in…
- Trial name
- NCT01386658
- Testing
- icatibant
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Shire
Anti-neutrophil Cytoplasmic Antibody-associated Vasculitis
A Study to Assess the Mass Balance Recovery (Absorption, Distribution, Metabolism and Excretion) of [14C]CCX168 in Healthy Participants
The primary objective of this study is to perform mass balance following a single oral dose of [14C]CCX168 in healthy adult male participants.
- Trial name
- NCT06004960
- Testing
- [14C]CCX168
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Amgen
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II
The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started.
- Trial name
- ENABLE (NCT04130191)
- Where
- 19 sites in Austria, Germany and 5 more
- Volunteers
- 140 people
- Sponsor
- Shire
Hereditary Angioedema, HAE
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
- Trial name
- APeX-A (NCT04933721)
- Testing
- berotralstat
- Where
- 16 sites in Canada, Czechia and 9 more
- Volunteers
- 139 people
- Sponsor
- BioCryst Pharmaceuticals
ANCA-associated Vasculitis
Clinical Trial to Evaluate Safety and Efficacy of CCX168 in ANCA-Associated Vasculitis
The aim of this trial is to test the safety and efficacy of two dose regimens of the complement C5a receptor CCX168 in patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV).
- Trial name
- NCT02222155
- Testing
- CCX168 10 mg, twice daily, plus cyclophosphamide/rituximab plus glucocorticoids, CCX168 30 mg, twice daily, cyclophosphamide/rituximab plus glucocorticoids
- Where
- 43 sites in United States, Canada
- Volunteers
- 42 people
- Sponsor
- Amgen
Primary IgA Nephropathy
A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma…
- Trial name
- NCT04014335
- Testing
- IONIS-FB-LRx
- Where
- 7 sites in Australia, Canada and 2 more
- Volunteers
- 23 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
- Trial name
- OPuS-1 (NCT01984788)
- Testing
- BCX4161
- Where
- 6 sites in Germany, United Kingdom
- Volunteers
- 24 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema, HAE
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- APeX-S (NCT03472040)
- Testing
- BCX7353
- Where
- 87 sites in United States, Australia and 18 more
- Volunteers
- 387 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX7353 in healthy subjects, and single and…
- Trial name
- NCT02448264
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
- Trial name
- NCT04739059
- Testing
- CSL312
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 171 people
- Sponsor
- CSL Behring