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.
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplantation (HSCT)
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy
The goal of this clinical trial is to evaluate the efficacy and safety of Iptacopan as a second-line treatment for high-risk hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA).
- Trial name
- NCT07347990
- Testing
- iptacopan
- Where
- China
- Volunteers
- 30 people
- Sponsor
- First Affiliated Hospital of Zhejiang University
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.
Transplant-Associated Thrombotic Microangiopathy (TA-TMA)
Safety and Efficacy of Eculizumab in High-risk TA-TMA
High-risk, complement-mediated, untreated transplant-associated thrombotic microangiopathy (hrTA-TMA) carries a very poor prognosis due to multiple organ dysfunction syndrome (MODS).
- Trial name
- NCT07707687
- Testing
- Eculizumab
- Where
- 8 sites in China
- Volunteers
- 24 people
- Sponsor
- First Affiliated Hospital of Zhejiang University
Thrombotic Microangiopathies
The Role of interferOn and Complement in SecondAry thRombotic micrioangiOpathy
Study rationale: Viral infections, such as CMV, are a risk factor for TA-TMA (transplantation-associated TMA). Viral infections increase interferon (IFN) levels and high IFN levels are associated with thrombotic microangiopathy…
- Trial name
- ROSARIO (NCT07705789)
- Testing
- blood draw, urine collection
- Volunteers
- 40 people
- Sponsor
- Sofie Dhaese
Hematopoietic Stem Cell Transplant (HSCT), Diffuse Alveolar Hemorrhage
TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant.
- Conditions
- Hematopoietic Stem Cell Transplant (HSCT), Diffuse Alveolar Hemorrhage, Thrombotic Microangiopathies, Interstitial Pneumonitis
- Trial name
- NCT04098445
- Where
- 9 sites in United States
- Volunteers
- 2,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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.
Thrombotic Microangiopathies
Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies
The main objective of this prospective multisite trial is to study the evolution of TMA (thrombotic microangiopathy) induced by gemcitabin and treated by eculizumab.
- Trial name
- GEMECULI (NCT05702996)
- Testing
- Eculizumab administration
- Where
- 2 sites in France
- Sponsor
- University Hospital, Rouen
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 Microangiopathies
Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura
Immune thrombotic thrombocytopenic purpura (iTTP) is caused by a severe, autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi…
- Trial name
- PEX-FREE (NCT06291025)
- Testing
- PEX-FREE
- Where
- 30 sites in France, Martinique and 1 more
- Volunteers
- 131 people
- Sponsor
- University Hospital, Rouen
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 Microangiopathy
N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy
This multicenter, prospective, single-arm clinical trial aims to evaluate the efficacy and safety of N-acetylcysteine (NAC) for treating Transplantation-Associated Thrombotic Microangiopathy (TA-TMA), a severe complication of…
- Trial name
- NCT07279610
- Testing
- N-Acetylcysteine (NAC) Treatment
- Where
- China
- Volunteers
- 44 people
- Sponsor
- The First Affiliated Hospital of Soochow University
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
Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic organ damage due to microvascular thrombosis.
- Conditions
- Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura, Thrombotic Microangiopathies, Microangiopathy, Thromboic
- Trial name
- TWILIGHT (NCT07205861)
- Where
- France
- Volunteers
- 1,200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Thrombotic Microangiopathies, Hemolytic Uremic Syndrome, Atypical
Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium
Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions.
- Conditions
- Thrombotic Microangiopathies, Hemolytic Uremic Syndrome, Atypical, Hemolytic-Uremic Syndrome
- Trial name
- COMPETE (NCT04745195)
- Where
- Netherlands
- Volunteers
- 42 people
- Sponsor
- Maastricht University Medical Center
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.