Clinical trials: Hemoglobinopathy (Disorder)
Splenectomy
Physiology and Pathologies Linked to Human Splenic Function : Direct and Ex-vivo Perfusion Explorations
Human splenic physiology remains poorly understood due to lack of functional exploration. However, through its ability to recognize alterations or modifications in circulating cells and to trigger an innate and adaptive response…
- Trial name
- SPLEENVIVO (NCT06418256)
- Testing
- Spleen, blood and plasma collection
- Where
- 5 sites in France
- Volunteers
- 100 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Thalassemia, Hepatitis C
Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial
Sustained Viral Response following 12-week therapy (SVR 12) with sofosbuvir/ledipasvir in transfusion-dependent patients with HCV genotype 1-6 Secondary Objective(s): Assessment of transfusion requirements Adverse events Efficacy…
- Trial name
- NCT03032666
- Testing
- sofosbuvir/velpatasvir
- Where
- Lebanon
- Volunteers
- 7 people
- Sponsor
- Ala'a Sharara
Anemia, Sickle Cell
A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers
This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO)…
- Trial name
- NCT03926819
- Testing
- HBI-002
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Hillhurst Biopharmaceuticals, Inc.
Sickle Cell Disease
Ameliorating Attention Problems in Children With Sickle Cell Disease (SCD)
The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on…
- Trial name
- NCT01411280
- Testing
- methylphenidate
- Where
- 2 sites in United States
- Volunteers
- 34 people
- Sponsor
- Temple University
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.
- Trial name
- NCT06924970
- Testing
- Tebapivat
- Where
- 20 sites in United States, Belgium and 5 more
- Volunteers
- 59 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Anemia
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality.
- Trial name
- NCT02090296
- Testing
- Hydroxyurea
- Where
- United States
- Sponsor
- Children's National Research Institute
Sickle Cell Disease
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease
This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease.
- Trial name
- FTC (NCT02273310)
- Testing
- Problem-Solving Skills Training for Disease Management
- Volunteers
- 83 people
- Sponsor
- University of Pennsylvania
Thalassemia
Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia.
- Trial name
- NCT00872833
- Where
- 9 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Carelon Research
Sickle Cell Disease
Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease
The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD)…
- Trial name
- NCT02678143
- Testing
- Alemtuzumab, Cyclophosphamide, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Phase 1 Study of Zoledronic Acid in Sickle Cell Disease
The long-term goal of this study is to learn if Zoledronic Acid can prevent or reduce pain in sickle cell disease. The goal of this study is to learn about the safety of Zoledronic Acid in persons with sickle cell disease who…
- Trial name
- NCT00639392
- Testing
- Zoledronic Acid
- Where
- United States
- Sponsor
- Virginia Commonwealth University
Sickle Cell Disease, Cerebral Stroke
Cerebral Oxygen Metabolism in Children
The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life.
- Trial name
- COMIC (NCT04157179)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Sickle Cell Anemia
Endothelial Function in Patients With Sickle Cell Anemia Before and After Sildenafil
The investigators would like to study the endothelial function in sickle cell patients without pulmonary hypertension in an in vivo method during a steady state condition before and after sildenafil treatment for 1 month, and to…
- Trial name
- NCT00937144
- Testing
- Viagra (Sildenafil)
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- The Baruch Padeh Medical Center, Poriya
Sickle Cell Disease
A Stress and Pain Self-management m-Health App for Adult Outpatients With Sickle Cell Disease
Our long-term goal is to reduce stress and improve sickle cell disease (SCD) pain control with less opioid use through an intervention with self-management relaxation/distraction exercises (RDE), named You Cope, We Support…
- Trial name
- NCT04484272
- Testing
- Self-monitoring of pain, stress, and opioid use + alerts/reminders + use of YCWS. [three video banks of RDE + Support])., Self-monitoring of pain, stress, and opioid use + alerts/reminders.
- Where
- United States
- Volunteers
- 239 people
- Sponsor
- University of Florida
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
Acute Respiratory Distress Syndrome
Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome
A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive…
- Trial name
- SiVent (NCT02582957)
- Testing
- Sigh breaths
- Where
- 15 sites in United States
- Volunteers
- 524 people
- Sponsor
- University of Minnesota
Sickle Cell Disease
Dietary Intake and Dietary Behaviors in Adults With Sickle Cell Disease
Background: Sickle Cell Disease (SCD) causes blood cells form a crescent shape. It is caused by a genetic mutation in the hemoglobin gene.
- Trial name
- NCT05170412
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease
Integrative Training Program for Pediatric Sickle Cell Pain
This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and…
- Trial name
- I-STRONG SCD (NCT06691867)
- Testing
- I-STRONG, Enhanced Usual Care (EUC)
- Where
- 4 sites in United States
- Volunteers
- 155 people
- Sponsor
- Emory University