Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing
The goal of this clinical trial is to evaluate whether a 4-week music therapy (MT) intervention can reduce chronic pain and improve psychosocial outcomes in youth with sickle cell disease (SCD).
- Trial name
- NCT07562451
- Testing
- Music Therapy, Standard of Care
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Emory University
Nocturnal Enuresis, Anemia, Sickle Cell
Desmopressin for Bedwetting in Children With SCD
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
- Trial name
- NCT04420585
- Testing
- Desmopressin
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Montefiore Medical Center
Anorexia, Thalassemia
Clinical Diagnosis of Teenagers Admitted to Pediatric Departments
This study intends to analyze the clinical characteristics of teenagers admitted to pediatric wards due to organic illness and non organic diagnosis, including patients suffering from chronic diseases and comorbidity.
- Conditions
- Anorexia, Thalassemia, Sickle Cell Anemia, Chronic Renal Failure
- Trial name
- NCT00735540
- Testing
- Observational
- Where
- Israel
- Volunteers
- 1,389 people
- Sponsor
- HaEmek Medical Center, Israel
Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.
- Conditions
- Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications, Sickle Cell Organ Damage, Sickle Cell Life Expectancy and Risk Factors for Early Death
- Trial name
- NCT05213572
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Crisis, Acute Pain
Low-dose Ketamine vs Morphine for Vaso-occlusive Crisis in Sicklers
This clinical trial will inform of the role of Low dose ketamine in the acute treatment of severe painful sickle cell crisis in children in a day-case sickle cell centre.
- Trial name
- KEM-VOC (NCT02434939)
- Testing
- Low dose ketamine, Morphine
- Where
- Uganda
- Volunteers
- 240 people
- Sponsor
- Makerere University
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
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 Disease
Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders, Pain Crisis
- Trial name
- NCT02433158
- Testing
- Rivipansel
- Where
- 97 sites in United States, Canada
- Volunteers
- 154 people
- Sponsor
- GlycoMimetics Incorporated
Sickle Cell Anemia
Hydoxyurea Exposure in Lactation A Pharmacokinetics Study (HELPS)
To examine the pharmacokinetics and distribution of oral hydroxyurea when administered as a single dose to lactating women
- Trial name
- HELPS (NCT02990598)
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Cardiovascular Complications of Sickle Cell Disease
In this research study, we are using heart imaging exams and blood testing, in order to gain an improved understanding of the pulmonary (lung) hypertension and cardiovascular (heart) complications that often occur in sickle cell…
- Trial name
- NCT01044901
- Testing
- MRI, Transthoracic Echocardiography, tonometry, EKG
- Where
- United States
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Sickle Cell Disease
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS)…
- Trial name
- NCT04983264
- Testing
- GBT021601
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Pfizer
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Sickle Cell Disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD).
- Trial name
- HIBISCUS KIDS (NCT06198712)
- Testing
- Etavopivat
- Where
- 18 sites in Canada, France and 5 more
- Volunteers
- 95 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease, Umbilical Cord Blood
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment…
- Conditions
- Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
- Trial name
- NCT04594031
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- 2 sites in United States
- Sponsor
- ExCellThera inc.
Sickle Cell Anemia
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality.
- Trial name
- NCT02090296
- Testing
- Hydroxyurea
- Where
- United States
- Sponsor
- Children's National Research Institute
Sickle-Cell Disease with Crisis
Analgesic Effect of Parecoxib Versus Morphine in SCD Patients Presenting to the Emergency Department
Introduction: This study focuses on the treatment of painful crises in Sickle Cell Disease (SCD) patients using Paracoxib, a non-opioid, compared to Morphine.
- Trial name
- PASC (NCT06315920)
- Testing
- Parecoxib, Morphine
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- Oman Medical Speciality Board
Sickle Cell Disease
Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease
This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of…
- Trial name
- NCT04285827
- Testing
- CSL889
- Where
- 15 sites in United States, Netherlands and 1 more
- Volunteers
- 28 people
- Sponsor
- CSL Behring
Sickle Cell Disease, Cerebral Stroke
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography
The purpose of the study protocol is to identify imaging biomarkers for brain tissue under high metabolic stress at risk for permanent injury.
- Trial name
- HDDOT CVR (NCT07776444)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease, Infant, Newborn, Disease
DREPAMASSE Study - Evaluation of a Newborn Screening for Sickle Cell Disease by Tandem Mass Spectrometry
Three methods are actually used in newborn screening for sickle cell disease (SCD) in France: isoelectric focusing, high performance liquid chromatography and capillary electrophoresis.
- Conditions
- Sickle Cell Disease, Infant, Newborn, Disease, Drepanocytosis
- Trial name
- DREPAMASSE (NCT03985501)
- Testing
- SCD newborn screening with the MS/MS method from Zentech
- Where
- 3 sites in France
- Volunteers
- 1,431 people
- Sponsor
- Hospices Civils de Lyon
Hematologic Diseases, Anaemia, Sickle Cell
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years
This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D.
- Trial name
- NCT05660265
- Testing
- GSK4172239D
- Where
- 6 sites in United States
- Volunteers
- 11 people
- Sponsor
- GlaxoSmithKline