Clinical trials: Hemoglobinopathy
Sickle Cell Disease
Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study
The investigators long-term goal is to demonstrate the effectiveness of acupuncture for the treatment of adults with chronic pain due to sickle cell disease (SCD), a debilitating pain syndrome characterized by acute and chronic…
- Trial name
- NCT04156399
- Testing
- Acupuncture
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Illinois Chicago
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease
Imatinib for Pain in Sickle Cell Anemia
In this protocol, the investigators propose to evaluate the biochemical effects of imatinib on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally following the guidelines previously established…
- Trial name
- IMPACT (NCT03997903)
- Testing
- Imatinib Mesylate
- Where
- 2 sites in United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Indiana University
Sickle Cell Disease, Sickle Cell Anemia
Microvascular Blood Flow in Sickle Cell Anemia
Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape.
- Trial name
- NCT01566890
- Testing
- regadenoson infusion with contrast-enhanced ultrasound, contrast-enhanced ultrasound
- Where
- 2 sites in United States
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Versiti Blood Health
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Iron Overload, Sickle Cell Disease
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- FIRST (NCT02041299)
- Testing
- Deferiprone, Deferoxamine
- Where
- 33 sites in United States, Brazil and 6 more
- Volunteers
- 230 people
- Sponsor
- ApoPharma
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome
Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome.
- Trial name
- NCT02697240
- Testing
- Intravenous citrulline
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease
A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet
This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally.
- Trial name
- NCT01430091
- Testing
- Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT])
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Sickle Cell Disease
SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease
The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.
- Trial name
- NCT04289311
- Testing
- Voice Crisis Alert V2
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Medical University of South Carolina
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
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease
Treatment of Sickle Cell Anemia With Stem Cell Transplant
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor.
- Conditions
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- Trial name
- NCT01350232
- Testing
- Fludarabine, Cytarabine, Cellular Infusions
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio
Pulmonary Hypertension, Sickle Cell Disease
Macitentan in Pulmonary Hypertension of Sickle Cell Disease
This is a pilot study to assess the safety and efficacy of macitentan in patients with pulmonary hypertension of sickle cell disease. This study will enroll approximately 10 subjects.
- Trial name
- MENSCH (NCT02651272)
- Testing
- macitentan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Boston University
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Sickle Cell Disease, Sickle Cell Crisis
The Use of Warmed Saline in Vaso-occlusive Episodes
The purpose of this study is to determine if warming the intravenous (IV) fluid given to patients with Sickle Cell Disease who are experiencing painful episodes known as Vaso-Occlusive Episodes; will decrease rates of hospital…
- Trial name
- NCT02316366
- Testing
- Astoflo Plus fluid warmer
- Volunteers
- 80 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia
The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients The secondary purpose is to assess the effect of L-glutamine on…
- Trial name
- NCT00586209
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Anemia, Sickle Cell
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization.
- Trial name
- NCT00530270
- Testing
- Dexamethasone
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida