Clinical trials: Sickle Cell Disorders
Children With Sickle Cell Disease at Mulago Hospital
Vitamin D Supplementation in Children With Sickle Cell Disease
Children aged 6 months to 12 years of age will be randomised to receive vitamin D 60,000IU once a month for 3 months or a placebo. The vitamin D will be in form of granules supplied in sachets.
- Trial name
- VIDS (NCT04662476)
- Testing
- Vitamin D3
- Volunteers
- 662 people
- Sponsor
- Makerere University
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Sickle Cell Anemia
Study of Clotrimazole and Hydroxyurea in Patients With Sickle Cell Syndromes
OBJECTIVES: Determine the effectiveness of the combined use of clotrimazole and hydroxyurea on a specific panel of red cell characteristics in patients with sickle cell syndromes.
- Trial name
- NCT00004404
- Testing
- clotrimazole, hydroxyurea
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is…
- Trial name
- NCT04166526
- Testing
- FDNIRS-DCS, MRI
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Boston Children's Hospital
Blood Disease, Cerebrovascular Accident
Stroke Prevention in Sickle Cell Anemia (STOP 2)
To determine how long blood transfusions are needed for primary stroke prevention. Also, to determine the duration of risk associated with abnormal transcranial Doppler ultrasound (TCD) and to determine the specificity of the…
- Conditions
- Blood Disease, Cerebrovascular Accident, Anemia, Sickle Cell
- Trial name
- NCT00006182
- Testing
- Blood Transfusion
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
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, Sickle Cell Anaemia
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children.
- Conditions
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Trial name
- RESCUE (NCT07282210)
- Testing
- Senicapoc
- Where
- 4 sites in Canada
- Volunteers
- 105 people
- Sponsor
- Biossil Inc.
Sickle Cell Disease, Beta-thalassemia
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions.
- Trial name
- NCT07599176
- Testing
- Total Body Irradiation, Alemtuzumab, Abatacept
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Avascular Bone Necrosis in Sickle Cell Disease: a Pediatric Study.
Avascular necrosis (AVN) is a serious complication of sickle cell disease, especially in pediatric patients where the prevalence is between 3% and 8% and are more frequent in patients with multiple vaso-occlusive crisis (VOC).
- Trial name
- OsteoSCD (NCT05203991)
- Where
- Italy
- Volunteers
- 32 people
- Sponsor
- San Luigi Gonzaga Hospital
Sickle Cell Disease
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare…
- Trial name
- NCT05810181
- Testing
- Interview
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Anemia, Sickle Cell
A Self-Management Intervention for Youth With Sickle Cell Disease and Their Families: Phase I
This study is being conducted to test an intervention for children and adolescents ages 8-17 years with sickle cell disease and their families.
- Trial name
- NCT03069430
- Testing
- SELFY (Self Management for Youth with SCD)
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Sickle Cell Disease, Obesity, Childhood
Obesity in Pediatric Sickle Cell Disease: A New Phenomenon
The objective of this project is to determine the prevalence of hypertension, hyperlipidemia and hyperglycemia in the pediatric population with sickle cell disease who are obese in Mississippi compared to those pediatric patients…
- Conditions
- Sickle Cell Disease, Obesity, Childhood, Hypertension, Hyperlipidemia in Children
- Trial name
- NCT04676113
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Mississippi Medical Center
Anemia, Sickle Cell
A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects
This study will provide information regarding the metabolic pathway of GBT440, the need for evaluation of potential drug-drug interactions, and the need for studies in special populations.
- Trial name
- NCT02497924
- Testing
- GBT440
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
- Trial name
- NCT03132324
- Testing
- INCB059872
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Incyte Corporation
Sickle Cell Anemia
Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease. II.
- Trial name
- NCT00004408
- Testing
- poloxamer 188
- Volunteers
- 300 people
- Sponsor
- Mast Therapeutics, Inc.
Sickle Cell Disease
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.
- Trial name
- NCT01783691
- Testing
- NKTT120
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- NKT Therapeutics
Sickle Cell Anemia, Sickle Cell Thalassemia
Long Term Follow up in Sickle Cell Patients Treated by Hydroxyurea
Hydroxyurea was found to be a good treatment in adult patients with sickle cell anemia with significant decrease in the frequency of vaso-occlusive crises and other crises related to SCA.
- Trial name
- NCT00480974
- Testing
- Clinical follow up and laboratory analysis
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease, Adverse Childhood Experiences
Early Life Exposures Among Children With Sickle Cell Disease
This study is being conducted to determine the relationship between early childhood exposures, such as Adverse Childhood Experiences, Social Determinants of Health and nutrition/breastfeeding, among children with sickle cell…
- Conditions
- Sickle Cell Disease, Adverse Childhood Experiences, Breastfeeding
- Trial name
- NCT05377372
- Testing
- Breastfeeding support group, Observation
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease (SCD)
Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
The purpose of this study was to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 2 to <18 years with a history of Vaso-Occlusive Crisis (VOC) with or without…
- Trial name
- NCT03474965
- Testing
- Crizanlizumab
- Where
- 35 sites in United States, Belgium and 12 more
- Volunteers
- 117 people
- Sponsor
- Novartis Pharmaceuticals
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