Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Sickle Cell Disease
Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease
The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment.
- Trial name
- COMPARE-VOE (NCT03933397)
- Testing
- Patient-Specific Protocol, Weight-based Protocol, Morphine
- Where
- 6 sites in United States
- Volunteers
- 328 people
- Sponsor
- Duke University
Sickle Cell Disease
A Study of SHP655 (rADAMTS13) in Sickle Cell Disease
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants.
- Trial name
- RAISE (NCT03997760)
- Testing
- TAK-755
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Shire
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Sickle Cell Anemia, Acute Chest Syndrome
Bi-Level Positive Airway Ventilation for Acute Chest Syndrome
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing.
- Trial name
- NCT01589926
- Testing
- Bi-level positive airway pressure device
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Sickle Cell Disease
Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis
The purpose of this study is to determine the safety and efficacy of intravenous magnesium in shortening the duration of a pain crisis and to determine the health-related quality of life and short term outcomes of children…
- Trial name
- MAGiC (NCT01197417)
- Testing
- Intravenous Magnesium Sulfate
- Where
- 8 sites in United States
- Volunteers
- 208 people
- Sponsor
- Medical College of Wisconsin
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
Vaso-occlusive Crisis, Sickle Cell Anemia Crisis
Hyperbaric Oxygen Therapy in Sickle Cell Pain
Sickle cell anemia is a genetic blood disorder where red blood cells are abnormally shaped, crescent- or sickle-shaped, instead of the normal, round shape.
- Trial name
- HAVOC (NCT03412045)
- Testing
- hyperbaric oxygen therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
Sickle Cell Trait
Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach
The hemoglobinopathy newborn screen (NBS) performed on all neonates in the U.S. allows for early life-saving medical care for infants with sickle cell disease (SCD), an autosomal recessive genetic disorder.
- Trial name
- NCT05387564
- Testing
- SCT Documentation and Disclosure Toolkit (SCT-DD)
- Where
- United States
- Volunteers
- 114 people
- Sponsor
- Nemours Children's Clinic
Sickle Cell Disease, Sickle Cell Anemia
Sickle Cell Disease (SCD) Bone Pain Study
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis
- Trial name
- NCT05283148
- Testing
- Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
Aspirin Prophylaxis in Sickle Cell Disease
Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by…
- Trial name
- START (NCT00178464)
- Testing
- aspirin
- Volunteers
- 11 people
- Sponsor
- University of Rochester
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 Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with…
- Trial name
- NCT02633397
- Testing
- Riociguat
- Where
- 20 sites in United States
- Volunteers
- 130 people
- Sponsor
- Mark Gladwin
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
Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease
The purpose of this study is to use cardiac magnetic resonance imaging (CMR) and echocardiographic tissue Doppler imaging to demonstrate a unique restrictive cardiomyopathy of sickle cell disease.
- Trial name
- NCT02410811
- Testing
- Cardiac magnetic resonance imaging (CMR)
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Anemia, Sickle Cell
Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow.
- Trial name
- NCT00094887
- Testing
- Nitric Oxide
- Where
- 11 sites in United States
- Volunteers
- 150 people
- Sponsor
- Mallinckrodt
Iron Overload, Sickle Cell Disease
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron…
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- NCT02443545
- Testing
- Deferiprone
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 134 people
- Sponsor
- ApoPharma