Clinical trials: Hemoglobinopathies
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Hemoglobinopathies
Non-Myeloablative Conditioning and Bone Marrow Transplantation
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia.
- Trial name
- NCT01850108
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide (CTX)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Vanderbilt-Ingram Cancer Center
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Anemia, Sickle ß0-Thalassemia
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT01179217
- Testing
- L-glutamine
- Where
- 31 sites in United States
- Volunteers
- 230 people
- Sponsor
- Emmaus Medical, Inc.
Sickle Cell Disease
Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow
The purpose of this study is to learn if it is possible and safe to treat persons with severe sickle cell disease (SCD) by bone marrow transplant (BMT) from human leukocyte antigen (HLA) half-matched related donors.
- Trial name
- TRANSFORM (NCT02757885)
- Testing
- Bone Marrow Transplant (BMT), Hydroxyurea, Thiotepa
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Sickle Cell Disease
Acceptability, Feasibility and Safety of a Yoga Program for Chronic Pain in Sickle Cell Disease
Chronic Pain is associated with morbidity and poor quality of life in patients with Sickle Cell Disease (SCD). Complementary therapies, such as yoga are beneficial in patients with non-SCD chronic pain conditions.
- Trial name
- NCT03694548
- Testing
- Part A Survey, Part B Yoga Program
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- Emory University
Sickle Cell Disease
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS)…
- Trial name
- NCT04983264
- Testing
- GBT021601
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Pfizer
Vaso-occlusive Crisis, Sickle Cell Anemia Crisis
Hyperbaric Oxygen Therapy in Sickle Cell Pain
Sickle cell anemia is a genetic blood disorder where red blood cells are abnormally shaped, crescent- or sickle-shaped, instead of the normal, round shape.
- Trial name
- HAVOC (NCT03412045)
- Testing
- hyperbaric oxygen therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
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
Sickle Cell Disease (SCD)
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older.
- Trial name
- NCT04662931
- Testing
- crizanlizumab
- Where
- 7 sites in India
- Volunteers
- 140 people
- Sponsor
- Novartis Pharmaceuticals
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
A Safety Study of Eptifibatide in Patients With Sickle Cell Disease
This study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises.
- Trial name
- NCT00834899
- Testing
- Eptifibatide
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Aspirin Prophylaxis in Sickle Cell Disease
Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by…
- Trial name
- START (NCT00178464)
- Testing
- aspirin
- Volunteers
- 11 people
- Sponsor
- University of Rochester
Sickle Cell Disease
A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
Sickle Cell Disease
Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia.
- Trial name
- NCT01077921
- Testing
- Propranolol
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Laura M. De Castro, MD
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Sickle Cell Disease, Sickle Cell Anemia
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape.
- Trial name
- NCT01000155
- Testing
- vorinostat
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Dana-Farber Cancer Institute
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center