Clinical trials: Sickle Cell Disorder
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
Iron Overload, Sickle Cell Disease
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron…
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- NCT02443545
- Testing
- Deferiprone
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 134 people
- Sponsor
- ApoPharma
Sickle Cell Disease
SMYLS Multi-site Trial
The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms.
- Trial name
- NCT06035939
- Testing
- Voice Crisis Alert V2, Electronic educational materials
- Where
- United States
- Volunteers
- 272 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Sickle Cell Trait
Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)
The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying…
- Trial name
- NCT06071377
- Testing
- Biologic Specimen Collection
- Where
- 8 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Alliance for Sickle Cell Centers
Acute Chest Syndrome, Sickle Cell Disease
Ultrasound Acute Chest Syndrome Sickle Cell Disease
Feasibility and reliability of ultrasound in the inpatient hematology setting.
- Trial name
- POCUS (NCT06880679)
- Testing
- Point of Care Ultrasound
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Indiana University
Cystic Fibrosis, Asthma
Measures of Respiratory Health (MRH)
The Lung Clearance Index, measured by multiple breath washout, is a measure of lung function that is considered a research tool in Canada as the device used to measure it is not approved by Health Canada.
- Conditions
- Cystic Fibrosis, Asthma, Sickle Cell Anemia, Bronchiolitis Obliterans
- Trial name
- MRH (NCT02657837)
- Where
- Canada
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- The Hospital for Sick Children
Sickle Cell Disease
Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who…
- Trial name
- IMPROVE (NCT02061202)
- Testing
- Mometasone Furoate
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- Jeffrey Glassberg
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Hypoxic Red Blood Cells in Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia.
- Conditions
- Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Trial name
- NCT06743113
- Testing
- Hemanext ONE System, Conventional RBCs
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Hemanext
Vaso-Occlusive Pain Episode in Sickle Cell Disease
Lidocaine for Opioid Sparing in Vaso-occlusive Crisis of Sickle Cell Disease
The purpose of the study is to determine whether adding lidocaine to standard of care in pain management during severe vaso-occlusive crisis has an effect on the cumulative opioid consumption expressed as morphine milligram…
- Trial name
- LidoVOC (NCT07274254)
- Testing
- Lidocain, Standard of care
- Where
- 12 sites in France, Guadeloupe
- Volunteers
- 104 people
- Sponsor
- Nantes University Hospital
Sickle Cell Disease
Cannabidiol in Sickle Cell Disease
Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.
- Trial name
- SPICE (NCT06930703)
- Testing
- Cannabidiol
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
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, Multiple Myeloma
ASH Research Collaborative Data Hub
Benign and malignant hematologic diseases are relatively rare conditions within the spectrum of medical practice in any one site of care.
- Trial name
- NCT05775224
- Where
- United States
- Volunteers
- 100,000 people
- Sponsor
- ASH Research Collaborative
Sickle Cell Disease
A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
- Trial name
- NCT05431088
- Testing
- Osivelotor
- Where
- 80 sites in United States, Brazil and 5 more
- Volunteers
- 389 people
- Sponsor
- Pfizer
Sickle Cell Anemia, Acute Chest Syndrome
Bi-Level Positive Airway Ventilation for Acute Chest Syndrome
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing.
- Trial name
- NCT01589926
- Testing
- Bi-level positive airway pressure device
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease
Vitamin D Supplementation in Children With Sickle Cell Disease
Sickle cell disease (SCD) is a genetic disease characterized by abnormal hemoglobin, the main constituent of red blood cells. People with SCD have nutritional deficiencies, and vitamin D deficiency is one of the most common.
- Trial name
- NCT03417947
- Testing
- Vitamin D bolus
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- St. Justine's Hospital
Sickle Cell Disease, Sickle Cell Anemia in Children
PINPOINT: Gaming Technology for SCD Pain
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
- Trial name
- Pinpoint II (NCT04579926)
- Testing
- Pinpoint app
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Hemophilia, Thrombosis
ATHNdataset Registry
The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems.
- Conditions
- Hemophilia, Thrombosis, Hemophilia A, Hemophilia B
- Trial name
- NCT06820515
- Where
- United States
- Volunteers
- 200,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Sickle Cell Disease
Real World Patient Characteristics and Treatment Patterns From Crizanlizumab Use: Preliminary Analysis From Select Sickle Cell Centers
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with patients who had initiated crizanlizumab between 2019 and 2022.
- Trial name
- NCT06662825
- Where
- United States
- Volunteers
- 376 people
- Sponsor
- Novartis
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 Disease
Predictors and Outcomes in Patients With Sickle Cell Disease
Children with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea.
- Trial name
- NCT03431935
- Where
- 2 sites in United States
- Volunteers
- 94 people
- Sponsor
- St. Jude Children's Research Hospital