Clinical trials: Hemoglobinopathy (Disorder)
Sickle Cell Disease
Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease
The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease. Sickle cell disease (SCD) is the most common genetic disorder in the United States, and occurs primarily…
- Trial name
- NCT02222246
- Testing
- Hydromorphone (Standardized, weight-based dosing), Morphine Sulfate (Standardized, weight-based dosing), Hydromorphone (Patient Specific dosing)
- Where
- 2 sites in United States
- Volunteers
- 106 people
- Sponsor
- Duke University
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT00125788
- Testing
- L-glutamine
- Where
- 5 sites in United States
- Volunteers
- 70 people
- Sponsor
- Emmaus Medical, Inc.
Sickle Cell Disease
Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers
The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care.
- Trial name
- NCT02863068
- Testing
- Topical Sodium Nitrite
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- caterina P minniti
Anemia, Sickle Cell
Trial of Oral Glutamine in Patients With Sickle Cell Anemia
Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have…
- Trial name
- NCT00131508
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- St. Jude Children's Research Hospital
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Sickle Cell Disease, Thalassemia
Effects of Transfusion of Older Stored Red Cells
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: whether a similar effect (older stored blood is associated with worse outcomes) is seen…
- Trial name
- NCT01534676
- Testing
- Transfusion, Blood, Chelation therapy
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Columbia University
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.
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
Pulmonary Hypertension, Sickle Cell Disease
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease…
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Trial name
- NCT01048905
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
Building Adaptive Coping and Knowledge to Improve Daily Life
The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs.
- Trial name
- Back2Life (NCT04602728)
- Testing
- Back2Life
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Emory University
Anemia, Sickle Cell, Transfusion Hemosiderosis
Clinical Importance of Treating Iron Overload in Sickle Cell Disease
Hypothesis: The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function.
- Trial name
- NCT00981370
- Testing
- deferasirox
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Los Angeles
Hematologic Diseases, Anemia, Sickle Cell
Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.
- Trial name
- NCT00006400
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 193 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Regional Anesthesia for Sickle Cell Crisis Using Ultrasound in The Emergency Department: Phase I
The hypothesis of this study is: Femoral nerve blocks can feasibly be performed on patients with Sickle Cell Disease and painful crisis in the Emergency Department.
- Trial name
- RESCUED (NCT01256281)
- Testing
- Femoral Nerve Block
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Sickle Cell Disease
Realizing Effectiveness Across Continents With Hydroxyurea (REACH)
REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for for pediatric patients with sickle cell anemia (SCA).
- Trial name
- REACH (NCT01966731)
- Testing
- Hydroxyurea
- Where
- 4 sites in Angola, Democratic Republic of the Congo and 2 more
- Volunteers
- 635 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Anemia, Sickle Cell
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization.
- Trial name
- NCT00530270
- Testing
- Dexamethasone
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Anemia
Prasugrel Versus Placebo in Adult Sickle Cell Disease
The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30…
- Trial name
- NCT01167023
- Testing
- Prasugrel
- Where
- 14 sites in United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University