Clinical trials: Hemolysis
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Phase I Study Evaluating Safety, Tolerability, PK and PD of Single and Multiple Doses of TUL321 Capsule in Healthy Participants and Food Effect on PK
- Trial name
- NCT07760467
- Testing
- Part1: TUL321, Part2: TUL321
- Where
- China
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Zhuhai United Laboratories Co., Ltd.
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
Hemolytic Disease of the Fetus and Newborn
A Study on Hemolytic Disease of the Fetus and Newborn (HDFN) Through Global Registry
The purpose of this non-interventional study is to prospectively evaluate the risk of anemia (decreased red blood cells) in fetuses (baby before birth) and neonates (baby just after birth) of pregnant participants who are at risk…
- Trial name
- GERANIUM (NCT07194070)
- Testing
- Standard of Care
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 175 people
- Sponsor
- Janssen Research & Development, LLC
Atypical Hemolytic Uremic
Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China
- Trial name
- Soliris (NCT05876351)
- Testing
- Eculizumab
- Where
- 6 sites in China
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either…
- Trial name
- NCT07416162
- Where
- 3 sites in South Korea
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
- Trial name
- NAP (NCT04671810)
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 44 people
- Sponsor
- AO GENERIUM
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
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
Tonsillitis
A Multicenter, Randomized, Double-Blind, Double-Dummy Study Of Azithromycin SR Versus Amoxicillin For The Treatment Of Strep Throat In Children
The objective was to determine if a single 60 mg/kg dose of azithromycin SR was as safe and effective as a 10-day regimen of amoxicillin (45 mg/kg/day, given in divided doses every 12 hours) when used to treat children with strep…
- Trial name
- NCT00643149
- Testing
- amoxicillin, azithromycin SR
- Where
- 46 sites in United States, Canada and 3 more
- Volunteers
- 693 people
- Sponsor
- Pfizer
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies…
- Trial name
- NCT06933914
- Testing
- MY008211A tablets
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Wuhan Createrna Science and Technology Co., Ltd
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Anti-neutrophil Cytoplasmic Antibody-associated Vasculitis
A Study in Healthy Volunteers to Evaluate the Drug-Drug Interaction Potential of CCX168 With Concomitant Medications
The primary objective of this study will be to evaluate the drug-drug interaction potential of CCX168 with concomitant medications, as either a perpetrator or a victim, following oral administration of CCX168 to healthy…
- Trial name
- NCT06004947
- Testing
- CCX168, Midazolam, Celecoxib
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Amgen
Warm Autoimmune Hemolytic Anemia, Cold Agglutinin Disease
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
This study is to assess the safety, tolerability, preliminary efficacy, and pharmacokinetics of APL-2 in subjects with warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD).
- Trial name
- NCT03226678
- Testing
- APL-2
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Crohn's Disease
A Study to Test the Effect of CDP870 in the Treatment of Crohn's Disease Over 26 Weeks, Comparing CDP870 to a Dummy Drug (Placebo)
A 26 week study to examine the efficacy, safety and pharmacokinetics of CDP870 in Crohn's disease
- Trial name
- NCT00152490
- Testing
- Certolizumab Pegol (CDP870)
- Volunteers
- 604 people
- Sponsor
- UCB Pharma
Crohn's Disease
Study to Test the Effect of CDP870 in the Treatment of Crohn's Disease Over 26 Weeks, Comparing CDP870 to a Dummy Drug (Placebo), Following 3 Doses of Active Drug (CDP870).
A 26 week maintenance study of CDP870 in Crohn's disease
- Trial name
- NCT00152425
- Testing
- Certolizumab Pegol (CDP870)
- Volunteers
- 392 people
- Sponsor
- UCB Pharma
Beta-Thalassemia
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe…
- Trial name
- NCT04938635
- Testing
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID
- Where
- 7 sites in United States, Bulgaria and 1 more
- Sponsor
- Vifor (International) Inc.
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire