Clinical trials: Neonatal Anemia
Sickle Cell Disease
A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with…
- Trial name
- NCT02633397
- Testing
- Riociguat
- Where
- 20 sites in United States
- Volunteers
- 130 people
- Sponsor
- Mark Gladwin
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 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
Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study
The investigators long-term goal is to demonstrate the effectiveness of acupuncture for the treatment of adults with chronic pain due to sickle cell disease (SCD), a debilitating pain syndrome characterized by acute and chronic…
- Trial name
- NCT04156399
- Testing
- Acupuncture
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Illinois Chicago
Pulmonary Hypertension, Sickle Cell Disease
Macitentan in Pulmonary Hypertension of Sickle Cell Disease
This is a pilot study to assess the safety and efficacy of macitentan in patients with pulmonary hypertension of sickle cell disease. This study will enroll approximately 10 subjects.
- Trial name
- MENSCH (NCT02651272)
- Testing
- macitentan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Boston University
Skin Ulcers, Sickle Cell Anemia
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II.
- Trial name
- NCT00004412
- Testing
- Arginine Butyrate, Standard local care dressing
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Susan P. Perrine
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Open-Label Extension of Voxelotor
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
- Trial name
- NCT04188509
- Testing
- Voxelotor
- Where
- 20 sites in United States, Egypt and 3 more
- Volunteers
- 162 people
- Sponsor
- Pfizer
Neutropenia, Sickle Cell Disease
Decitabine for High-Risk Sickle Cell Disease
Background: In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin…
- Trial name
- NCT01375608
- Testing
- Decitabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia.
- Trial name
- BEYOND (NCT03342404)
- Testing
- Luspatercept, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Greece and 4 more
- Volunteers
- 145 people
- Sponsor
- Celgene
Neural Tube Defects, Anemia
Post T-plant Infusion of Allogeneic Cytokine Induced Killer (CIK) Cells as Consolidative Therapy in Myelodysplastic Syndromes/Myeloproliferative Disorders
Allogeneic stem cell transplantation (transplant of blood cells from another individual) is a treatment option for patients with myelodysplasia or myeloproliferative Disorders.
- Conditions
- Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure
- Trial name
- NCT01392989
- Testing
- CIK cells, Cyclosporine, Mycophenolate Mofetil
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Everett Meyer
Sickle Cell Disease
A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet
This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally.
- Trial name
- NCT01430091
- Testing
- Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT])
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease
Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.
- Trial name
- ESSENTIAL (NCT01736657)
- Testing
- Red blood cell exchange in sickle cell
- Where
- 5 sites in United States
- Volunteers
- 73 people
- Sponsor
- Terumo BCT
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS)
Sirolimus for Autoimmune Disease of Blood Cells
Treatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune system.
- Conditions
- Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura
- Trial name
- NCT00392951
- Testing
- sirolimus
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Philadelphia
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
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD).
- Trial name
- ViSionSerenity (NCT04817670)
- Testing
- VIT-2763 120 mg, VIT-2763 360 mg, VIT-2763 240 mg
- Where
- 22 sites in United States, France and 3 more
- Volunteers
- 25 people
- Sponsor
- Vifor (International) Inc.
Hemoglobin SC Disease
Treatment of Hemoglobin SC Disease With Hydroxyurea
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications.
- Trial name
- SCYTHE (NCT02336373)
- Testing
- hydroxyurea
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Baylor College of Medicine
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Iron Overload, Excessive Body Iron Burden
Massive Iron Deposit Assessment
Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital