Clinical trials: Sickle Cell Disorders
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, 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 Disease
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can…
- Trial name
- SUN (NCT03587272)
- Testing
- Alemtuzumab, low dose total body irradiation, Sirolimus
- Where
- 7 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Robert Nickel
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
Pain, Sickle Cell Disease
Intranasal Fentanyl Versus Intravenous Morphine in the Treatment of Severe Painful Sickle Cell Crises in Children
Sickle cell anaemia is an inherited blood disorder which results in abnormal sickle shaped red blood cells which do not fit well through small blood vessels.
- Trial name
- NCT03682211
- Testing
- Fentanyl Citrate, Morphine sulphate
- Where
- Ireland
- Volunteers
- 31 people
- Sponsor
- University College Dublin
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
Sickle Cell Disease, Hb-SS Disease With Vasoocclusive Pain
Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain Crisis
The purpose of this study is to determine whether giving abciximab (ReoPro) to children with sickle cell disease who are hospitalized for acute pain crisis will improve their pain and shorten the time spent in the hospital, when…
- Conditions
- Sickle Cell Disease, Hb-SS Disease With Vasoocclusive Pain, Hemoglobin SS Disease With Vasoocclusive Crisis, Other Sickle Cell Disease With Vaso-Occlusive Pain
- Trial name
- NCT01932554
- Testing
- Abciximab, Intravenous hydration, Ibuprofen
- Where
- United States
- Sponsor
- St. Louis University
Sickle Cell Disease
Liver Fibrosis in Sickle Cell Disease
Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions.
- Trial name
- NCT02007746
- Testing
- Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of Miami
Sickle Cell Anemia in Children
Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE)
This study will 1) Evaluate the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania; 2) Obtain…
- Trial name
- NCT03948867
- Testing
- Hydroxyurea, Elevated Arm TCD Examination, Normal Arm TCD Examination
- Where
- 2 sites in United States, Tanzania
- Volunteers
- 202 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Genetic and Haematological Modifiers of SCD Severity in Kaduna State, Northern Nigeria
This study is aimed to assess the genetic and haematological modifiers of disease severity among patients with Sickle Cell Disease (SCD) in Kaduna State, northern Nigeria.
- Trial name
- SCA (NCT05837871)
- Volunteers
- 400 people
- Sponsor
- Ahmadu Bello University Teaching Hospital
Sickle Cell Disease, COVID-19
COVID-19 Vaccine Response in Sickle Cell Disease
The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination.
- Trial name
- NCT05139992
- Testing
- COVID-19 Vaccine
- Where
- 8 sites in United States
- Volunteers
- 59 people
- Sponsor
- ASH Research Collaborative
Sickle Cell Disease
Is the Preoperative Preparation of Sickle Cell Patients Optimal: Assessment of Practices and Post-operative Complications
Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis…
- Trial name
- NCT04264871
- Where
- Belgium
- Volunteers
- 250 people
- Sponsor
- Queen Fabiola Children's University Hospital
Chronic Illness, Hematologic Malignancy
Distracting Through Procedural Pain and Distress
Children with acute and chronic illness undergo frequent, painful, and distressing procedures. This randomized control trial was used to evaluate the effectiveness of guided imagery (GI) vs virtual reality (VR) on the procedural…
- Conditions
- Chronic Illness, Hematologic Malignancy, Bone Marrow Transplant Infection, Oncology
- Trial name
- NCT04892160
- Testing
- Guided Imagery, Virtual Reality
- Where
- 2 sites in United States
- Volunteers
- 67 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Sickle Cell
Diastolic Dysfunction in Sickle Cell Disease During Vaso-occlusive Crisis
This study is designed to investigate the presence and absence of abnormal heart muscle contractions during sickle cell crises. You will be asked to do echocardiography during and after your admission to the hospital.
- Trial name
- NCT01683968
- Where
- Saudi Arabia
- Volunteers
- 216 people
- Sponsor
- King Abdullah International Medical Research Center
Sickle Cell Disease
Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging
The investigators will explore barriers to improving self-efficacy, or the ability to feel in control of their disease, and medication adherence with text messaging through surveys and interviews with adolescents and adults with…
- Trial name
- NCT02785848
- Testing
- Interviews and Surveys
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Vanderbilt University
Sickle Cell Disease, Anemia
SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain
The overall goal of the project is to reduce pain-related, 30-day readmission rates for sickle cell disease (SCD) patients. The investigators want to see if a mobile phone application (app) can help decrease the need for repeat…
- Conditions
- Sickle Cell Disease, Anemia, Anemia, Hemolytic, Anemia, Sickle Cell
- Trial name
- NCT03006718
- Testing
- PROMIS for Pain Management App
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Julia Finkel
Pulmonary Hypertension, Lung Disease
Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients.
- Conditions
- Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant
- Trial name
- NCT00041574
- Testing
- Inhaled Nitric Oxide
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease
New Hemolysis Parameters in Sickle Cell Disease
The 6-minute walk (6MWT) test is used in adults and children affected by a wide range of chronic diseases to evaluate their sub-maximal exercise capacity.
- Trial name
- HEMODREP (NCT03040908)
- Volunteers
- 150 people
- Sponsor
- Queen Fabiola Children's University Hospital
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