Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells.
- Trial name
- NCT00489281
- Testing
- Cyclophosphamide, Fludarabine, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Acute Chest Syndrome, Sickle Cell Disease
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death.
- Trial name
- INNOSTAPED (NCT01089439)
- Testing
- Nitric oxide by inhalation INOMAX
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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 Anemia in Children, Sickle Cell Disease
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year.
- Trial name
- PUSHUP (NCT05285917)
- Testing
- Hydroxyurea
- Where
- Angola
- Volunteers
- 400 people
- Sponsor
- Brown University
Sickle Cell Disease
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications…
- Trial name
- R34 pK/PD (NCT02447874)
- Testing
- Arginine, Arginine (Loading), Arginine (Continuous)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Emory University
Data Collection, Sickle Cell Disease
Data Collection For Patients With Sickle Cell Disease
To create a database of clinical information, participant /disease characteristics, and treatment history collected from patients with Sickle Cell Disease.
- Trial name
- NCT07911618
- Testing
- data collection
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- M.D. Anderson Cancer Center
Sickle Cell Disease
Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease
This is a single site nonrandomized pilot clinical trial of the feasibility, acceptability, and efficacy of a combination treatment involving digital Cogmed working memory skills training, social support components, and memory…
- Trial name
- ALL IN (NCT06770010)
- Testing
- Cogmed+
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's National Research Institute
Sickle Cell Disease
Biotin-RBC Transfusion in SCD
This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion…
- Trial name
- NCT04426591
- Testing
- Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Marianne Yee
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 and Thalassemia
Allograft for Sickle Cell Disease and Thalassemia
The design of the study incorporates the following features: 1. This is a phase II study to determine the safety and therapeutic potential of a new transplant approach (disease-free survival, graft versus myeloma effect) and to…
- Trial name
- NCT02038478
- Testing
- Donor Stem Cell Transplantation
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease, Hemoglobinopathies
Non-Myeloablative Conditioning and Bone Marrow Transplantation
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia.
- Trial name
- NCT01850108
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide (CTX)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Vanderbilt-Ingram Cancer Center
Sickle Cell Anemia in Children, Vitamin A Deficiency in Children
Nutritional Outcomes After Vitamin A Supplementation in Subjects With SCD
This study establishes the safety and efficacy of vit A supplementation doses (3000 and 6000 IU/d) over 8 weeks in children with SCD-SS, ages 9 and older and test the impact of vit A supplementation on key functional and clinical…
- Trial name
- NCT03632876
- Testing
- retinyl palmitate
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Disease
Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease
The purpose of this research study is to test the safety and efficacy of a drug called Plerixafor. Plerixafor is approved by the US FDA for use in increasing blood stem cell counts before collection in cancer patients.
- Trial name
- NCT02193191
- Testing
- Plerixafor
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Disease, Acute Chest Syndrome
Effect of Inhaled Nitric Oxide in Acute Chest Syndrome (INOSTA Study)
Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults.
- Trial name
- INOSTA (NCT00748423)
- Testing
- Nitric Oxide
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pulmonary Hypertension, Lung Disease
Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients.
- Conditions
- Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant
- Trial name
- NCT00041574
- Testing
- Inhaled Nitric Oxide
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Mallinckrodt
Chronic Pain, Sickle Cell Disease
MBI for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease
This study examines the efficacy of a mindfulness-based intervention designed to support pain and sleep management in adolescents and young adults with sickle cell disease.
- Conditions
- Chronic Pain, Sickle Cell Disease, Sleep
- Trial name
- REMedy_RCT (NCT07797582)
- Testing
- Mindfulness-based intervention
- Volunteers
- 48 people
- Sponsor
- University of Illinois Chicago
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Acute Chest Syndrome
sPLA2 in EBC During Acute Chest Syndrome
Secretory phosholipases A2 (sPLA2) are significantly elevated in the plasma of sickle cell disease patients with acute chest syndrome (ACS), and similar enzymes have been measured in exhaled breath condensate (EBC), which is…
- Trial name
- NCT03250585
- Testing
- Exhaled Breath Condensate (EBC), Plasma Sample
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Virginia Commonwealth University
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
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.