Clinical trials: Sickle Cell Disorders
Sickle Cell Disease, Drepanocytosis
Links Between Cognitive Functions and Clinical, Biological and Neuroradiological Outcomes in Adults With Sickle Cell Disease.
Sickle cell disease (SCD) is an inherited blood disorder. Symptoms include acute and chronic complications. Due to progress in SCD care, patients with SCD are living longer than before and we focus more attention in chronic…
- Trial name
- Drépa-COG (NCT04244240)
- Testing
- BEARNI Tool
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Anemia, Severe Acute Malnutrition
Management of Severe Acute Malnutrition in SCD, in Northern Nigeria
Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children < 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether…
- Trial name
- NCT03634488
- Testing
- hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
- Where
- 3 sites in United States, Nigeria
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Vaso-Occlusive Pain Episode in Sickle Cell Disease, Sickle Cell Disease (SCD)
Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis
The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are: Does Ketamine lower the number of times participants need to be…
- Trial name
- NCT07682662
- Testing
- Standardized Low-dose bolus IV Ketamine within 30 minutes of arrival and every 1 hour as needed for max of 3 doses., Standard Care (in control arm)
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Trial name
- IMPACTS-2 (NCT01522196)
- Testing
- Varespladib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Anthera Pharmaceuticals
Sickle Cell Disease
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell…
- Trial name
- NCT04207320
- Testing
- αβ+ T-cell depletion with Miltenyi CliniMACS system
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Chicago
Sickle Cell Disease
Biological, Genetic and Environmental Involved in the Complications of Sickle Cell Disease
The objective of the study is to refine our knowledge on the physiopathology of the symptoms and the complications for the patients affected by a drepanocytic syndrome.
- Trial name
- NCT04205123
- Testing
- sickle cell syndrome
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- Erasme University Hospital
Analyze the Results of Cardiorespiratory Stress Tests, Resting and Stress Echocardiography
Exploration of the Parameters Influencing the Effort Limitation of Patients Suffering From Homozygous Sickle Cell Anemia
The cardiovascular complications of sickle cell disease are associated with major morbidity and mortality, as well as impaired quality of life.
- Conditions
- Analyze the Results of Cardiorespiratory Stress Tests, Resting and Stress Echocardiography, Capillary Lactatemia Measurements at the Ear
- Trial name
- PHYSIO-EXDRE (NCT06743139)
- Testing
- Echography
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Improving Emergency Department Management of Adults With Sickle Cell Disease
The objective of this study is to design, implement and test quality improvement measures to improve the care of adults with sickle cell disease in the emergency department.
- Trial name
- NCT01603160
- Testing
- Quality Improvement
- Where
- United States
- Volunteers
- 715 people
- Sponsor
- Duke University
Sickle Cell Disease
Dose-Finding Study of SC411 in Children With Sickle Cell Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD).
- Trial name
- SCOT (NCT02973360)
- Testing
- Docosahexaenoic Acid
- Where
- 11 sites in United States
- Volunteers
- 68 people
- Sponsor
- Micelle BioPharma Inc
Bone Avascular Necrosis, Sickle Cell Anemia
Decompression Coring Versus Conservative Therapy in Patients With Avascular Necrosis of the Hip Related to Sickle Cell Disease
OBJECTIVES: I. Phase II trial to determine surgical morbidity of decompression coring, including any adverse events in the perioperative period and the rate of secondary medical or surgical interventions. II.
- Trial name
- NCT00006130
- Testing
- Core decompression
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Drepanocytosis
Study of a Deformability Parameter of Red Blood Cell
Sickle-cell disease is one of the most common severe monogenic disorders in the world, it results in the synthesis of abnormal hemoglobin (HbS) instead of hemoglobin A.
- Trial name
- FITRED (NCT05850156)
- Testing
- basal blood collection, blood collection M6, blood collection M12
- Volunteers
- 130 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hematologic Diseases, Anemia, Sickle Cell
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia.
- Conditions
- Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
- Trial name
- NCT00029380
- Testing
- Sangstat, Cyclophosphamide, Busulfan
- Where
- 15 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Patient and Public Involvement and Engagement in Research With Children and Young People With Sickle Cell Disorder and Their Families
Aim: To co-produce resources for inclusive and equitable Patient and Public Involvement and Engagement in research on life-limiting conditions, with children and young people with sickle cell disorder and their families.
- Trial name
- NCT06293222
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- King's College London
Sickle Cell Disease
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell…
- Trial name
- TRANSPLANTORN2 (NCT06351462)
- Testing
- Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire
- Volunteers
- 220 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Chronic Kidney Diseases, Sickle Cell Disease
Sickle Cell Uric Acid (SCUA) - Cohort Repository
The purpose of this research is to study the causes of Sickle Cell kidney disease, as well as to collect and store samples and information about people with Sickle Cell Disease.
- Trial name
- NCT04190888
- Testing
- No intervention
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Virginia Commonwealth University
Sickle Cell Disease
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be…
- Trial name
- NCT06442761
- Testing
- Motixafortide
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- St. Jude Children's Research Hospital
Healthy Subjects, Sickle Cell Disease
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).
- Trial name
- NCT02285088
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 133 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease, Vaso-Occlusive Crisis
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the…
- Trial name
- AKTSS (NCT03296345)
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland