Clinical trials: Hereditary Red Blood Cell Disorder (Disorder)
- Condition Hereditary Red Blood Cell Disorder (Disorder)
- Funder type Industry (pharma, biotech, devices)
Contraception
Study of Treatment With the Combination of Drospirenone Plus Ethinyl Estradiol Plus Levomefolate Calcium in Mexican Women Seeking Contraception
The objective of this study is to compare the red blood cell (RBC) and plasma concentrations of folate in subjects treated with drospirenone (DRSP)/ethinyl estradiol (EE) plus levomefolate calcium (L-5-MTHF) at 24 weeks with…
- Trial name
- NCT01902264
- Testing
- EE20/DRSP/L-5-MTHF (Beyaz, BAY98-7071)
- Where
- Mexico
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Bayer
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Thalassemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia.
- Trial name
- BEYOND (NCT03342404)
- Testing
- Luspatercept, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Greece and 4 more
- Volunteers
- 145 people
- Sponsor
- Celgene
Erythrocyte Transfusion, Beta-Thalassemia
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require…
- Trial name
- BELIEVE (NCT02604433)
- Testing
- Luspatercept
- Where
- 76 sites in United States, Australia and 13 more
- Volunteers
- 336 people
- Sponsor
- Celgene
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Nervous System Disorder, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX…
- Trial name
- IEDAT-01 (NCT01255358)
- Testing
- Dexamethasone
- Where
- 2 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Quince Therapeutics S.p.A.
Sickle Cell Disease, Vaso-occlusive Crisis
A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT05348915
- Testing
- Inclacumab
- Where
- 50 sites in United States, Brazil and 8 more
- Volunteers
- 242 people
- Sponsor
- Pfizer
Beta-thalassemia
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to…
- Trial name
- NCT05567458
- Testing
- Luspatercept
- Where
- 10 sites in China
- Volunteers
- 94 people
- Sponsor
- Bristol-Myers Squibb
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered
To evaluate the relative bioavailability of ticagrelor for the different formulations. A randomized cross-over design has been chosen to minimize the effects of between-subject variability and any period effects on the overall…
- Trial name
- NCT03126695
- Testing
- Ticagrelor granule, Ticagrelor pediatric tablets, Ticagrelor pediatric tablets suspended in water
- Where
- Germany
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Sickle Cell Disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult…
- Trial name
- SPARKLE (NCT06439082)
- Testing
- Crizanlizumab
- Where
- 36 sites in United States, Brazil and 3 more
- Volunteers
- 354 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Hypoxic Red Blood Cells in Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia.
- Conditions
- Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Trial name
- NCT06743113
- Testing
- Hemanext ONE System, Conventional RBCs
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Hemanext
Sickle Cell Disease
Biotin-Acridine Red Cell Exchange Kinetics
The primary objective of the study is to describe the recovery and survival of pathogen-reduced (PR) red blood cells (RBCs) prepared and stored with the INTERCEPT Blood System for RBCs® (hereafter referred to as INTERCEPT RBCs)…
- Trial name
- BARK (NCT07155031)
- Testing
- Pathogen reduced red blood cells
- Sponsor
- Cerus Corporation
Anemia
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
- Trial name
- NCT05891249
- Testing
- Luspatercept
- Where
- 9 sites in India
- Volunteers
- 85 people
- Sponsor
- Bristol-Myers Squibb
Thalassemia Major
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
- Trial name
- NCT01740531
- Testing
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Where
- 3 sites in Italy, Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Cerus Corporation
Sickle Cell Anemia
Evaluation of the AMICUS RBCx System in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.
- Trial name
- NCT02372877
- Testing
- Amicus Red Cell Exchange in SCD patients
- Where
- 6 sites in United States
- Volunteers
- 83 people
- Sponsor
- Fenwal, Inc.
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Sickle Cell Disease
Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.
- Trial name
- ESSENTIAL (NCT01736657)
- Testing
- Red blood cell exchange in sickle cell
- Where
- 5 sites in United States
- Volunteers
- 73 people
- Sponsor
- Terumo BCT