Clinical trials: Thrombotic Microangiopathy
Thrombotic Microangiopathies, ITP Immune-Mediated Thrombocytopenia
TGD001 Treatment in Thrombotic Microangiopathies
This is a Phase 1/2, prospective, adaptive design trial of TGD001 in participants with suspicion or clinical diagnosis of acute immune thrombotic thrombocytopenic purpura (iTTP) episodes and participants with suspicion or…
- Trial name
- NCT07459114
- Testing
- TGD001
- Volunteers
- 70 people
- Sponsor
- TargED Biopharmaceuticals B.V.
Atypical Hemolytic Uremic Syndrome
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy.
- Trial name
- APPELHUS (NCT04889430)
- Testing
- Iptacopan
- Where
- 36 sites in United States, Brazil and 6 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and…
- Trial name
- ALX-TMA-502 (NCT07732361)
- Where
- 43 sites in United States, Belgium and 8 more
- Volunteers
- 307 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathy
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
- Trial name
- NCT04543591
- Testing
- Ravulizumab, Best supportive care
- Where
- 66 sites in United States, Australia and 15 more
- Volunteers
- 148 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
- Trial name
- NCT05795140
- Testing
- Iptcaopan 200 mg
- Where
- 9 sites in Brazil, China and 4 more
- Volunteers
- 65 people
- Sponsor
- Novartis Pharmaceuticals
Atypical Hemolytic Uremic Syndrome
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.
- Trial name
- NCT05935215
- Testing
- Iptacopan
- Where
- 31 sites in China, France and 6 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Hematopoietic Stem Cell Transplant Associated Thrombotic Microangiopathy (HSCT-TMA)
Single Patient Expanded Access Treatment Plan For The Investigational Product Narsoplimab
The purpose of the EAP is to provide access to hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) patients who may benefit from treatment with narsoplimab based on the medical judgement of their…
- Trial name
- NCT04247906
- Testing
- Narsoplimab
- Sponsor
- Omeros Corporation
Thrombotic Microangiopathy
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA.
- Trial name
- NCT04557735
- Testing
- Ravulizumab, Best Supportive Care
- Where
- 26 sites in United States, Israel and 5 more
- Volunteers
- 41 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome
Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the platelet count change from baseline and safety of OMS721 (narsoplimab) in adults and adolescents with atypical hemolytic uremic syndrome (aHUS).
- Trial name
- aHUS (NCT03205995)
- Testing
- OMS721 (narsoplimab)
- Where
- 4 sites in United States, Lithuania and 2 more
- Volunteers
- 6 people
- Sponsor
- Omeros Corporation
Transplant-Associated Thrombotic Microangiopathy
A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.
- Trial name
- NCT05148299
- Testing
- Pegcetacoplan
- Where
- 14 sites in United States, France and 3 more
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Atypical Hemolytic-Uremic Syndrome
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
- Trial name
- NCT01522183
- Where
- 141 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Immune Thrombocytopenia, Blood Platelet Disorder
The Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of HMPL-523 in Adult Subjects With Immune Thrombocytopenia (ITP)
This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.
- Conditions
- Immune Thrombocytopenia, Blood Platelet Disorder, Hematologic Diseases, Purpura, Thrombocytopenic
- Trial name
- NCT06291415
- Testing
- HMPL-523
- Where
- 28 sites in United States, Australia and 3 more
- Sponsor
- Hutchmed
Thrombotic Microangiopathies
Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
- Trial name
- NCT04784455
- Testing
- nomacopan (rVA576)
- Where
- 9 sites in United States, Poland and 1 more
- Volunteers
- 10 people
- Sponsor
- AKARI Therapeutics
Thrombotic Microangiopathies, Hematopoietic Stem Cell Transplantation
Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).
- Trial name
- NCT05855083
- Testing
- Biological: narsoplimab
- Where
- 16 sites in United States, Germany and 3 more
- Volunteers
- 18 people
- Sponsor
- Omeros Corporation
Systemic Lupus Erythematosus
Study to Evaluate Safety, Tolerability and Pharmacodynamics of KP104 in Participants With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus
This study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KP104 in participants with systemic lupus erythematosus (SLE)-Thrombotic microangiopathy (TMA).
- Trial name
- NCT05504187
- Testing
- KP104
- Volunteers
- 24 people
- Sponsor
- Kira Pharmacenticals (US), LLC.
Thrombotic Microangiopathy
Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger
This study will investigate the efficacy and safety of ravulizumab compared to placebo in adult participants with thrombotic microangiopathy (TMA) associated with a trigger.
- Trial name
- NCT04743804
- Testing
- Ravulizumab, Best Supportive Care
- Where
- 97 sites in United States, Belgium and 11 more
- Volunteers
- 16 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathies
Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies
The purpose of this study is to assess the safety, efficacy, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of OMS721 in patients with thrombotic microangiopathies (TMA).
- Trial name
- NCT02222545
- Testing
- OMS721
- Where
- 24 sites in United States, Belgium and 10 more
- Volunteers
- 58 people
- Sponsor
- Omeros Corporation
Atypical Hemolytic Uremic Syndrome, aHUS
Observational Study of Elizaria® in aHUS Patients
It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome
- Trial name
- NCT04749810
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 50 people
- Sponsor
- AO GENERIUM
Stem Cell Transplant Complications, Thrombotic Microangiopathies
Study in Adult and Pediatric Patients With HSCT-TMA
This is an observational, retrospective study designed to assess outcomes in patients diagnosed with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) who were not treated with complement…
- Trial name
- NCT04970004
- Testing
- No intervention
- Where
- United States
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
- Trial name
- NCT03303313
- Testing
- Cemdisiran
- Where
- 12 sites in Bosnia and Herzegovina, Canada and 8 more
- Sponsor
- Alnylam Pharmaceuticals