Clinical trials: Microangiopathy
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
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.
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, 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.
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
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
Diabetes Type 1
The MIND Study - Microangiopathy IN Diabetes
This clinical investigation will evaluate two contactless optical devices based on spatial frequency domain and laser speckle technology for quantification of the skin micro-circulation in patients with diabetes mellitus type 1.
- Trial name
- NCT06989190
- Where
- Sweden
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- HJN Sverige AB/Neko Health
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
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
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
Transplant Associated Microangiopathy TAM
Efficacy and Safety of LFG316 in Transplant Associated Microangiopathy (TAM) Patients
This was a randomized, SoC-controlled, open-label, multi-center study in patients with TAM after hematopoietic precursor cell transplantation (HPCT) .
- Trial name
- NCT02763644
- Testing
- LFG316 active drug, Standard of care treatment
- Where
- 6 sites in United States, France and 1 more
- Volunteers
- 7 people
- Sponsor
- Novartis Pharmaceuticals
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.
aHUS - Atypical Hemolytic Uremic Syndrome
Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy
This is a Phase II, open-label study designed to determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.
- Trial name
- NCT05684159
- Testing
- NM8074
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
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
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 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
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.
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 Microangiopathy, Thrombotic Thrombocytopenic Purpura
Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small…
- Trial name
- NCT00726544
- Testing
- ARC1779 Injection
- Where
- 17 sites in United States, Austria and 3 more
- Volunteers
- 100 people
- Sponsor
- Archemix Corp.
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