Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Analysis of T-Cell Immune Reconstitution Following Allogeneic Hematopoietic BMT for Severe SCD
In this study, patient blood samples from NMA transplants will be provided by Pittsburgh, and samples from myeloablative transplants will be provided by Atlanta (comparative controls).
- Trial name
- NCT00228631
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Emory University
Sickle Cell Disease, Hyperbaric Oxygen Therapy
Hyperbaric-oxygen Therapy (HBOT) Versus Placebo for Treating Vaso-Occlusive Crisis (VOC) in Sickle Cell Disease (SCD)
This is a randomised, controlled, double-blind, placebo trial of HBOT (intervention) superiority in the treatment of VOC in SCD, to demonstrate the effectiveness of HBOT for the decrease in pain level in the treatment of SCD-VOC.
- Conditions
- Sickle Cell Disease, Hyperbaric Oxygen Therapy, Vaso-occlusive Crisis
- Trial name
- HBOT-SCD (NCT05289700)
- Testing
- HBOT in Hyperbaric chamber
- Where
- 3 sites in France, Switzerland
- Volunteers
- 100 people
- Sponsor
- University Hospital, Geneva
Sickle Cell Disease, Executive Dysfunction
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork…
- Conditions
- Sickle Cell Disease, Executive Dysfunction, Cognitive Impairment, Attention Deficit/Hyperactivity Disorder (ADHD)
- Trial name
- NCT07226219
- Testing
- Extended-Release Methylphenidate
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cells Disease
Vitamin D and Bisphosphonates in the Treatment of Sickle Cell Disease
Sickle cell disease (SCD) is a worldwide distributed hereditary red cell disorder, which affects approximately 75,000 individuals in the United States and almost 20,000- 25,000 subjects in Europe, this latter mainly related to…
- Trial name
- NCT02972138
- Where
- 3 sites in Italy
- Volunteers
- 80 people
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
Sickle Cell Anemia, Sickle Cell Disease
Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas
The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) study is the first placebo-controlled randomized clinical trial of hydroxyurea treatment in a malaria endemic region.
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM-MTD (NCT03128515)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 187 people
- Sponsor
- Indiana University
Sickle Cell Disease
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with…
- Trial name
- NCT00445978
- Testing
- Sapropterin Dihydrochloride
- Where
- 12 sites in United States
- Volunteers
- 32 people
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Anemia
Study of Beet Juice for Patients With Sickle Cell Anemia
The investigators hypothesize that increasing plasma nitrite using dietary nitrate will improve platelet function and red cell deformability and decrease MCHC in patients with sickle cell disease.
- Trial name
- NCT02162225
- Testing
- beet juice (Unbeetable)
- Where
- United States
- Sponsor
- Wake Forest University Health Sciences
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
HbE Trait, Hemoglobinopathies
Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
Hemoglobinopathies are inherited disorders that affect haemoglobin and pose a considerable global health challenge. This research addresses the knowledge gap regarding the prevalence of a typical haemoglobin fraction within urban…
- Trial name
- Hb Variants (NCT07305181)
- Where
- Bangladesh
- Volunteers
- 8,400 people
- Sponsor
- Bangladesh Bioscience Research Group
Sickle Cell Disease, Iron Overload
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Trial name
- NCT00110617
- Testing
- Deferasirox (ICL670), Deferoxamine (DFO)
- Where
- 59 sites in United States, Canada
- Volunteers
- 212 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anaemia
Mobile-Directly Observed Therapy on Adherence to Hydroxyurea
To examine the effect of mobile-directly observed therapy (mDOT) on adherence to HU (mDOT-HuA) adults with SCA at Muhimbili National Hospital (MNH) in Tanzania.
- Trial name
- mDOT (NCT02844673)
- Testing
- Hydroxyurea, Mobile Directly Observed Therapy
- Where
- Tanzania
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Muhimbili University of Health and Allied Sciences
Pulmonary Hypertension, Sickle Cell Disease
A Pilot Study of Chronic Red Blood Cell Transfusion in Sickle Cell Disease-Associated Pulmonary Hypertension
Pulmonary hypertension, a complication associated with an increased risk of death, is common in patients with sickle cell disease.
- Trial name
- NCT00850369
- Testing
- RBC transfusion
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease
Our primary objective is to assess whether inhaling vaporized cannabis ameliorates chronic pain in patients with sickle cell disease (SCD).
- Trial name
- Cannabis-SCD (NCT01771731)
- Testing
- Cannabis
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of California, San Francisco
Hemoglobinopathies, Anemia, Sickle Cell
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high…
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia
- Trial name
- NCT00034528
- Testing
- Busulfan, Fludarabine, FK506
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member…
- Trial name
- HaploSCD (NCT01461837)
- Testing
- CD34 selected T-cell depleted allogeneic SCT
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- New York Medical College
Sickle Cell Disease
A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease
Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family)…
- Trial name
- NCT01279616
- Testing
- Fludarabine monophosphate, Rituximab, Busulfan
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Anemia in Children, Sickle Cell Disease
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year.
- Trial name
- PUSHUP (NCT05285917)
- Testing
- Hydroxyurea
- Where
- Angola
- Volunteers
- 400 people
- Sponsor
- Brown University
Sickle Cell Disease, Chronic Pain
MUSic Therapy to Improve Quality Of Life in Sickle Cell Disease (MUSIQOLS)
The purpose of this pilot study is to investigate the effects of a 6-session music therapy protocol on the pain, mood, quality of life, coping skills, and self-efficacy of adult patients with sickle cell disease (SCD) as compared…
- Trial name
- NCT03556657
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
Iron Mediated Vascular Disease in Sickle Cell Anemia Patients
The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function.
- Trial name
- NCT01239901
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Children's Hospital Los Angeles
Osteonecrosis
Functional Outcomes Following Hip Core Decompression in Younger Participants With Osteonecrosis
Although uncommon in the general pediatric population, osteonecrosis (ON) is prevalent in children and adolescents with cancer, particularly among those that have had chronic exposure to glucocorticoids and among those having…
- Trial name
- NCT02458937
- Testing
- Functional Mobility Assessment, GAITRite® System, Range of Motion
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- St. Jude Children's Research Hospital
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