Clinical trials: Anemia, Neonatal
Pulmonary Hypertension, Sickle Cell Anemia
A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease.
- Trial name
- ASSET-3 (NCT00360087)
- Testing
- Bosentan
- Volunteers
- 236 people
- Sponsor
- Actelion
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
This cross-sectional prevalence assessment study involves a single blood draw in specific patient populations to assess for enzymatic and genomic evidence for acquired pyruvate kinase deficiency.
- Conditions
- Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Hereditary Hemolytic Anemia, Myelodysplastic Syndromes
- Trial name
- NCT04902833
- Testing
- Blood Draw
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Massachusetts General Hospital
Nursing Caries
Nonpharmacological Approaches and Parental Education in Children With Sickle Cell Disease
Aim: Sickle cell disease (SCD) is a hereditary disease. Approximately 5% of the world's population carries trait genes for haemoglobin disorders, mainly, sickle-cell disease and thalassaemia.
- Trial name
- NCT04121247
- Testing
- nonpharmacological approaches to parents of children with sickle cell disease training program
- Where
- Turkey (Türkiye)
- Volunteers
- 163 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Sickle Cell Disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years…
- Trial name
- NCT04774536
- Testing
- CRISPR_SCD001
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Mark Walters, MD
Sickle Cell Anemia, Pulmonary Hypertension
Tricuspid Regurgitant Jet Velocity as an Independent Marker for Mortality in Sickle Cell Anemia
The purpose of this study is to evaluate patients with pulmonary hypertension and sickle cell disease who have had multiple echocardiograms.
- Trial name
- NCT01783093
- Testing
- Data Collection
- Where
- United States
- Volunteers
- 209 people
- Sponsor
- Namita Sood
POEMS Syndrome, Amyloidosis
A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
- Conditions
- POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Trial name
- NCT05263817
- Testing
- CD19/BCMA CAR T-cells
- Where
- China
- Volunteers
- 75 people
- Sponsor
- Zhejiang University
Drepanocytosis, Chronic Neurological Deficiency
Transfusion Exchanges and Cognition in Sickle Cell Disease
DREPA-COG is an observational study evaluating the effect of exchange transfusions on cognitive function in adults with severe sickle cell disease (SS or Sβ0).
- Trial name
- DREPA-COG (NCT07168447)
- Where
- 6 sites in France
- Volunteers
- 85 people
- Sponsor
- Centre Hospitalier St Anne
Stroke Ischemic, Sickle Cell Disease
Evaluating Barriers to Stroke Screening and Prevention in Children With Sickle Cell Disease
DISPLACE is a three part, multi-center U.S. based study to evaluate the barriers to stroke screening and prevention in children with sickle cell anemia (SCA).
- Trial name
- DISPLACE (NCT03621826)
- Testing
- Questionnaire/Interview
- Where
- United States
- Volunteers
- 5,247 people
- Sponsor
- Medical University of South Carolina
Beta-Thalassemia
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity.
- Trial name
- NCT06568926
- Testing
- Serum Ferritin level
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Sohag University
Sickle Cell Disease
Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease
This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune…
- Trial name
- NCT03298399
- Testing
- Autologous MSCs
- Sponsor
- Emory University
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
Hereditary Spherocytosis
Hereditary Spherocytosis and Vascular Function
Background : Patients with hereditary spherocytosis (HS) are characterized by increased red blood cell (RBC) fragility and a loss of RBC deformability.
- Trial name
- VASCUSPHERO (NCT04451785)
- Testing
- blood sample, Pulse wave velocity
- Where
- 2 sites in France
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
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
Preterm Birth Complication, Neonatal Morbidity and Mortality
Delayed Versus Immediate Cord Clamping in Preterm Birth
The goal of this study is to evaluate whether delayed cord clamping improves early neonatal outcomes compared with immediate clamping in preterm birth.
- Conditions
- Preterm Birth Complication, Neonatal Morbidity and Mortality, Cord Clamping, Delayed Cord Clamping
- Trial name
- PREM-CORD (NCT07699666)
- Testing
- Delayed Umbilical Cord Clamping, Immediate Umbilical Cord Clamping
- Where
- Tunisia
- Volunteers
- 200 people
- Sponsor
- Faculty of Medicine of Tunis
Sickle Cell Disease Vaso-occlusive Crisis
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with…
- Trial name
- NCT06699849
- Testing
- CSL889
- Where
- 30 sites in United States, Turkey (Türkiye) and 1 more
- Volunteers
- 70 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Dissemination and Implementation of Stroke Prevention Looking at the Care Environment
The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world…
- Trial name
- DISPLACE (NCT04173026)
- Testing
- Provider-level intervention, Patient-level intervention
- Where
- 15 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
Medication Adherence in Youth With Sickle Cell Disease (SCD)
Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD.
- Trial name
- NCT02143076
- Testing
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Chronic Myeloproliferative Disorders, Leukemia
12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004058
- Testing
- tetradecanoylphorbol acetate
- Where
- United States
- Sponsor
- University of Medicine and Dentistry of New Jersey
Sickle Cell Disease, Vaso-occlusive Pain Episode
Arginine Therapy for Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores…
- Trial name
- NCT02536170
- Testing
- L-arginine, L-arginine Loading Dose
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Emory University