Clinical trials: Anemia, Neonatal
Acute Chest Syndrome, Sickle Cell Disease
Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome
The purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS).
- Trial name
- NCT02098993
- Testing
- Unfractionated heparin
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig Seaman
Sickle Cell Disease
Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care.
- Trial name
- STRIDE2 (NCT02766465)
- Testing
- Busulfan, Fludarabine, r-ATG
- Where
- 35 sites in United States
- Volunteers
- 138 people
- Sponsor
- Medical College of Wisconsin
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
MDS, Immunodeficiency
Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC
Background: Stem cells are immature blood cells that grow in the bone marrow and produce all of the cells needed for normal blood and immunity.
- Conditions
- MDS, Immunodeficiency, GATA2
- Trial name
- NCT00923364
- Testing
- Cyclophosphamide (CTX, Cytoxan), Fludarabine(Fludara,Berlex Laboratories), Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Cancer Institute (NCI)
Stroke (CVA) or TIA, Diabetes
Risk Factors, Costs, and Impacts of ED Boarding
The goal of this observational study is to learn about the risk factors, costs, and operational impacts of emergency department boarding (patients admitted to the hospital but remaining in the emergency department awaiting…
- Conditions
- Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease)
- Trial name
- NCT07532564
- Where
- United States
- Volunteers
- 30,486 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
Sickle Cell Disease (SCD)
Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease
The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle…
- Trial name
- STEADFAST (NCT04053764)
- Testing
- Crizanlizuamb, Standard of Care
- Where
- 24 sites in United States, Brazil and 9 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Stroke
Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial
The goal of this study is to determine the effectiveness of blood transfusion therapy for prevention of silent cerebral infarct (stroke) in children with sickle cell anemia.
- Trial name
- SIT (NCT00072761)
- Testing
- transfusion therapy
- Where
- United States
- Volunteers
- 196 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
- Trial name
- STArT (NCT04839354)
- Testing
- L-Arginine Hydrochloride, Normal saline
- Where
- 10 sites in United States
- Volunteers
- 271 people
- Sponsor
- Claudia R. Morris
Premature Infant Disease, Transfusion Related Complication
Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Repeated transfusions have been associated with very poor outcome of preterm infants. Fetal hemoglobin (HbF) and adult Hb (HbA) have different affinity for oxygen.
- Conditions
- Premature Infant Disease, Transfusion Related Complication, Fetal Hemoglobin
- Trial name
- CB-TrIP (NCT03764813)
- Where
- 2 sites in Italy
- Volunteers
- 25 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sickle Cell Disease
iCanCope With Sickle Cell Pain
The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease.
- Trial name
- NCT03201874
- Testing
- Pain self-management, Education
- Where
- 4 sites in United States, Canada
- Volunteers
- 137 people
- Sponsor
- Seattle Children's Hospital
Sickle Cell Disease
Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease
The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease. Sickle cell disease (SCD) is the most common genetic disorder in the United States, and occurs primarily…
- Trial name
- NCT02222246
- Testing
- Hydromorphone (Standardized, weight-based dosing), Morphine Sulfate (Standardized, weight-based dosing), Hydromorphone (Patient Specific dosing)
- Where
- 2 sites in United States
- Volunteers
- 106 people
- Sponsor
- Duke University
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen
Hypothesis 1: A novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathies
- Trial name
- NCT00427661
- Testing
- Busulfan; Fludarabine; cyclosporine A and MMF
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Pittsburgh
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study)
This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on…
- Trial name
- ST3P-UP (NCT03593395)
- Testing
- Peer Mentoring [PM], Structured Education Based Transition Program STE
- Where
- 14 sites in United States
- Volunteers
- 291 people
- Sponsor
- Wake Forest University Health Sciences
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sickle Cell Disease, Virtual Reality
Role of Virtual Reality (VR) in Patients With Sickle Cell Disease (SCD)
Patients with sickle cell disease (SCD) and cancer often have complicated courses while hospitalized and often deal with pain, anxiety and depression.
- Conditions
- Sickle Cell Disease, Virtual Reality, Anxiety, Depression
- Trial name
- NCT03387033
- Testing
- Relaxation response and virtual reality (VR) session
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Duke University
Sickle Cell Disease, Depression
Designing an Implementation Strategy for Delivering Routine Mental Health Screening and Treatment
African Americans living with chronic health conditions are more likely to experience depression and other mental health disorders than their healthy counterparts, and are more likely to experience severe depression than whites…
- Conditions
- Sickle Cell Disease, Depression, Anxiety
- Trial name
- NCT04587661
- Testing
- off-the-shelf digital CBT, adapted digital CBT
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Pittsburgh