Clinical trials: Anemia, Hemolytic
Paroxysmal Nocturnal Hemoglobinuria
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers.
- Trial name
- NCT05490017
- Testing
- KP104
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Kira Pharmacenticals (US), LLC.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
- Trial name
- COMMODORE 1 (NCT04432584)
- Testing
- Crovalimab, Eculizumab
- Where
- 80 sites in United States, Belgium and 22 more
- Volunteers
- 190 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
A safety and immunogenicity study in long-term treatment of Eculizumab (JSC "GENERIUM", Russian Federation) in patients with paroxysmal nocturnal hemoglobinuria, who have been involved earlier in the clinical trials of Eculizumab…
- Trial name
- NAP (NCT04679103)
- Testing
- Eculizumab (JSC GENERIUM, Russia)
- Where
- 9 sites in Russia
- Volunteers
- 50 people
- Sponsor
- AO GENERIUM
Sickle Cell Disease
A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet
This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally.
- Trial name
- NCT01430091
- Testing
- Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT])
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Chronic Cutaneous Ulcer, Sickle Cell Disease
Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
- Trial name
- D-SCOUT (NCT04058197)
- Testing
- Deferoxamine Product
- Where
- 4 sites in United States
- Sponsor
- TauTona Group
Paroxysmal Nocturnal Hemoglobinuria
A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria
This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.
- Trial name
- NCT04058158
- Testing
- SB12 (proposed eculizumab biosimilar), Soliris (eculizumab)
- Where
- 27 sites in India, Malaysia and 6 more
- Volunteers
- 50 people
- Sponsor
- Samsung Bioepis Co., Ltd.
Sickle Cell Disease, Umbilical Cord Blood
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment…
- Conditions
- Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
- Trial name
- NCT04594031
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- 2 sites in United States
- Sponsor
- ExCellThera inc.
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch…
- Trial name
- ACCESS 2 (NCT05131204)
- Testing
- Cemdisiran, Eculizumab, Pozelimab
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Regeneron Pharmaceuticals
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio
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
Sickle Cell Disease
Retrospective Real World Oxbryta® Data Collection and Analysis Study
The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300…
- Trial name
- RETRO (NCT04930328)
- Testing
- Oxbryta® (voxelotor) 500-mg Tablets
- Where
- 12 sites in United States
- Volunteers
- 216 people
- Sponsor
- Pfizer
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Paroxysmal Nocturnal Hemoglobinuria
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100.
- Trial name
- NCT03181633
- Testing
- ACH-0144471
- Where
- 4 sites in Italy, New Zealand and 1 more
- Volunteers
- 8 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hemochromatosis
Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug
- Trial name
- NCT00350662
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 2 sites in Egypt, Turkey (Türkiye)
- Volunteers
- 95 people
- Sponsor
- Lipomed
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension.
- Trial name
- ASSET-2 (NCT00313196)
- Testing
- bosentan
- Volunteers
- 12 people
- Sponsor
- Actelion
Systemic Lupus Erythematosus, Autoimmune Hemolytic Anemia (AIHA)
Evaluation of Safety, Pharmacokinetics and Pharmacodynamics of Arnovie101, an mRNA-LNP-Based In Vivo CAR-T Therapy, for the Treatment of B Cell-Mediated Autoimmune Diseases (SLE and AIHA)
This is an open lable and single arm study designed to evaluate the safety, PK and PD of Arnovie101 in B cell-mediated Autoimmune Disease
- Trial name
- NCT07629596
- Testing
- Arnovie101 Infusion Intravenous
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Circunited BioPharma (Shenzhen) Co., Ltd.
Cold Agglutinin Disease
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
The purpose of the study is to determine the efficacy of pegcetacoplan administration compared to placebo in increasing hemoglobin (Hgb) level from baseline and avoiding transfusion in participants with primary cold agglutinin…
- Trial name
- NCT05096403
- Testing
- Pegcetacoplan
- Where
- 44 sites in United States, Austria and 12 more
- Volunteers
- 24 people
- Sponsor
- Swedish Orphan Biovitrum
Warm Autoimmune Hemolytic Anemia (wAIHA)
Safety, Pharmacokinetics, and Efficacy of Subcutaneous Isatuximab in Adults With Warm Autoimmune Hemolytic Anemia (wAIHA)
Primary Objectives: Part A: To evaluate the safety and tolerability of subcutaneous injections of isatuximab in adults with wAIHA Part B: To evaluate the efficacy of the selected dose in adults with wAIHA Secondary Objectives…
- Trial name
- NCT04661033
- Testing
- Isatuximab SAR650984
- Where
- 6 sites in United States, France and 4 more
- Volunteers
- 8 people
- Sponsor
- Sanofi
Warm Antibody Autoimmune Hemolytic Anemia
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
- Trial name
- NCT03764618
- Testing
- Fostamatinib disodium
- Where
- 102 sites in United States, Australia and 20 more
- Volunteers
- 90 people
- Sponsor
- Rigel Pharmaceuticals
Hairy Cell Leukemia
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of…
- Trial name
- PROXY (NCT04125290)
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- AstraZeneca