Clinical trials: Hemoglobinopathies
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Chronic Obstructive Pulmonary Disease, Heart Failure
PATient Navigator to rEduce Readmissions
Staying out of the hospital is valued by patients and their caregivers. Their interests converge with those of hospitals now that high 30-day readmission rates for some conditions place hospitals at risk for financial penalties…
- Conditions
- Chronic Obstructive Pulmonary Disease, Heart Failure, Sickle Cell Disease, Myocardial Infarction
- Trial name
- PArTNER (NCT02114515)
- Testing
- Hospital usual care, Navigator intervention, Peer-led telephone support line
- Where
- United States
- Volunteers
- 1,029 people
- Sponsor
- University of Illinois at Chicago
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting
The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events.
- Trial name
- ESCAPED (NCT02411396)
- Where
- 3 sites in United States
- Volunteers
- 483 people
- Sponsor
- Johns Hopkins University
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease.
- Trial name
- NCT01794000
- Testing
- Prasugrel
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 341 people
- Sponsor
- Eli Lilly and Company
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
Sickle Cell Anemia, Children, Only
Shared-Decision Making for Hydroxyurea
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent…
- Trial name
- ENGAGE-HU (NCT03442114)
- Testing
- Hydroxyurea SDM Toolkit, Clinician Pocket Guide
- Where
- 11 sites in United States
- Volunteers
- 176 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Improving Self-Management in Adolescents With Sickle Cell Disease
The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a…
- Trial name
- SCThrive (NCT02851615)
- Testing
- SCThrive Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver.
- Trial name
- NCT01186419
- Testing
- SPD602 (FBS0701, SSP-004184)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 51 people
- Sponsor
- Shire
Anemia, Sickle Cell
A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers
This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO)…
- Trial name
- NCT03926819
- Testing
- HBI-002
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Hillhurst Biopharmaceuticals, Inc.
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
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease
This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease.
- Trial name
- FTC (NCT02273310)
- Testing
- Problem-Solving Skills Training for Disease Management
- Volunteers
- 83 people
- Sponsor
- University of Pennsylvania
Thalassemia
Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia.
- Trial name
- NCT00872833
- Where
- 9 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Carelon Research
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.
Acute Respiratory Distress Syndrome
Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome
A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive…
- Trial name
- SiVent (NCT02582957)
- Testing
- Sigh breaths
- Where
- 15 sites in United States
- Volunteers
- 524 people
- Sponsor
- University of Minnesota
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Sickle Cell Disease
Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease
This project will evaluate AppliedVR's EaseVRx - a multi-modal, skills-based, 8-week, virtual reality, home intervention - in an exploratory randomized controlled trial for self-management of chronic pain among Black, young…
- Trial name
- NCT04906707
- Testing
- EaseVRx, Audio-only version of EaseVRx
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Emory University