Clinical trials: Sickle Cell Disease (SCD)
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Sickle Cell Disease
Medication Adherence in Youth With Sickle Cell Disease (SCD)
Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD.
- Trial name
- NCT02143076
- Testing
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not…
- Trial name
- SIMCS SCD (NCT06563440)
- Testing
- Digital app and information system
- Where
- 2 sites in United States, Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia Crisis
Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises?
The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room.
- Trial name
- NCT03541980
- Testing
- Acetaminophen, Normal saline
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Newark Beth Israel Medical Center
Stroke
Hydroxyurea to Prevent Stroke in Children With Sickle Cell Anemia and Elevated TCD Flow Velocity
The purpose of this study is to assess prospectively the efficacy of hydroxyurea therapy in the setting of cerebrovascular disease, manifest as conditional or abnormal transcranial doppler ultrasonography (TCD) flow velocities…
- Trial name
- NCT00402480
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Duke University
Anemia, Sickle Cell, Sickle Cell Disease
Preventing Sickle Cell Kidney Disease
Untreated hypertension and renal injury are risk factors for increased morbidity and mortality in sickle cell disease, yet early markers of progressive disease have not been identified and therapies to prevent the development of…
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease, Kidney Disease, Hypertension
- Trial name
- NCT02373241
- Testing
- Losartan
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Pulmonary Hypertension
Serum Brain Natriuretic Peptide Levels and Pulmonary Hypertension in Pediatric Sickle Cell Patients
The purpose of this study is 1. To determine if Brain natriuretic peptide levels correlates with elevated tricuspid regurgitation flow velocity levels in pediatric patients with sickle cell disease 2.
- Trial name
- NCT01023451
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Akron Children's Hospital
Sickle Cell Anemia
A Phase II Trial of Regadenoson in Sickle Cell Anemia
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case…
- Trial name
- NCT01788631
- Testing
- Regadenoson
- Where
- 12 sites in United States
- Volunteers
- 100 people
- Sponsor
- Dana-Farber Cancer Institute
Sickle Cell Disease
Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease
This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis)…
- Trial name
- NCT01033227
- Testing
- sodium nitrite injection, usp
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Los Angeles
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Other Sickle-cell Disorders With Crisis, Unspecified
Quality of Life Study for Sickle Cell Patients Treated With Jobelyn (Sorghum Bicolor Extract)
The purpose of this study is to determine the antioxidant effect of prolonged use of sorghum bicolor (jobelyn) to increase the level of plasma superoxide dismutase and glutathione reductase in patients with sickle cell disease…
- Trial name
- NCT01704794
- Testing
- Folic Acid + Paludrine + Jobelyn (500mg), Folic Acid + Paludrine + Jobelyn (250mg), Folic Acid + Paludrine + Jobelyn (2mg)
- Where
- Nigeria
- Volunteers
- 96 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Glutamine Role in Preventing Vaso-occlusive Crisis Among SCD Patients
Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients
- Trial name
- Glu_SCD_Egy (NCT05371184)
- Testing
- L-Glutamine, Oral Powder for Reconstitution, Standard of care
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Vaso-Occlusive Pain Episode in Sickle Cell Disease, Sickle Cell Disease (SCD)
Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis
The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are: Does Ketamine lower the number of times participants need to be…
- Trial name
- NCT07682662
- Testing
- Standardized Low-dose bolus IV Ketamine within 30 minutes of arrival and every 1 hour as needed for max of 3 doses., Standard Care (in control arm)
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- University of Mississippi Medical Center
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Vitamin D Deficiency, Sickle Cell Disease
Vitamin D Status in Children With Sickle Cell Disease Living in Lyon, France
Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population.
- Trial name
- EVADDREP (NCT04694976)
- Testing
- 25-OH Vitamin D measure
- Where
- France
- Volunteers
- 130 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Disease, Pulmonary Disease
Cooperative Assessment of Late Effects for SCD Curative Therapies
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Trial name
- COALESCE (NCT05153967)
- Where
- 5 sites in United States
- Volunteers
- 377 people
- Sponsor
- Vanderbilt University Medical Center