Clinical trials: Hemoglobinopathy (Disorder)
Vaso-occlusive Crisis, Reduction in Hospitalizations
Apixaban in Patients With Sickle Cell Disease
In patients with SCD, the use of low dose anticoagulation as an outpatient may lead to a significant decrease in morbidity and as a result, decrease healthcare utilization and costs.
- Conditions
- Vaso-occlusive Crisis, Reduction in Hospitalizations, Sickle Cell Disease
- Trial name
- NCT02179177
- Testing
- Apixaban
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Nirmish Shah
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Hemoglobin SC Disease
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells.
- Trial name
- NCT00532883
- Testing
- Hydroxyurea, Magnesium Pidolate
- Where
- 19 sites in United States
- Volunteers
- 44 people
- Sponsor
- St. Jude Children's Research Hospital
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
Using Patient-Centered Guidelines in a Technology Platform to Improve Health Care in Adults With Sickle Cell Disease
SCD is an inherited disorder of hemoglobin that affects over 100,000 Americans, most of whom live in low-resourced neighborhoods.
- Trial name
- NCT03629678
- Testing
- mobile health application
- Where
- 2 sites in United States
- Volunteers
- 67 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease
Acceptability, Feasibility and Safety of a Yoga Program for Chronic Pain in Sickle Cell Disease
Chronic Pain is associated with morbidity and poor quality of life in patients with Sickle Cell Disease (SCD). Complementary therapies, such as yoga are beneficial in patients with non-SCD chronic pain conditions.
- Trial name
- NCT03694548
- Testing
- Part A Survey, Part B Yoga Program
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- Emory University
Sickle Cell Disease, Renal Function Disorder
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the…
- Trial name
- NCT02522104
- Testing
- Siklos
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Theravia
Sickle Cell Disease
Assessing the Safety of Buprenorphine in People With Sickle Cell Disease
This study will assess the safety of changing pain medications (opioids) adult sickle cell patients take to another type of medication therapy (buprenorphine). Patients will be asked questions about their quality of life.
- Trial name
- NCT03492099
- Testing
- buprenorphine
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Johns Hopkins University
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease
Treatment of Sickle Cell Anemia With Stem Cell Transplant
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor.
- Conditions
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- Trial name
- NCT01350232
- Testing
- Fludarabine, Cytarabine, Cellular Infusions
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Pain, Sickle Cell Disease
Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally
The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive…
- Trial name
- COMPARE (NCT01380197)
- Testing
- Morphine, Nubain
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Children's Healthcare of Atlanta
Sickle Cell Disease
Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting
The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events.
- Trial name
- ESCAPED (NCT02411396)
- Where
- 3 sites in United States
- Volunteers
- 483 people
- Sponsor
- Johns Hopkins University
Sickle Cell Disease
Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
- Trial name
- PROSPECT (NCT04930445)
- Testing
- Oxbryta® (voxelotor) 500mg Tablets
- Where
- 43 sites in United States
- Volunteers
- 265 people
- Sponsor
- Pfizer
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
Anemia, Sickle Cell
Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
- Trial name
- NCT02187003
- Testing
- Rivipansel
- Where
- 142 sites in United States, Canada
- Volunteers
- 345 people
- Sponsor
- Biossil Inc.
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)
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
Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease
This project will evaluate AppliedVR's EaseVRx - a multi-modal, skills-based, 8-week, virtual reality, home intervention - in an exploratory randomized controlled trial for self-management of chronic pain among Black, young…
- Trial name
- NCT04906707
- Testing
- EaseVRx, Audio-only version of EaseVRx
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Emory University
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
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
Sickle Cell Disease, Stroke
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease.
- Trial name
- HUPrevent (NCT01389024)
- Testing
- Hydroxyurea
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Johns Hopkins University