Clinical trials: Sickle - Beta Thalassemia
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease, Sickle B+ Thalassemia
Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality…
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04675645
- Testing
- HU-Go app
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Beta-thalassemia Major, Sickle Cell Disease
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: The…
- Conditions
- Beta-thalassemia Major, Sickle Cell Disease, Iron Hemosiderosis
- Trial name
- NCT01511848
- Testing
- DFP (ferriprox) and deferasirox (ICL 670), DFP, DFO
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Ain Shams University
Patients With Thalassemia Intermedia,, Congenital Dyserythropoietic Anemia Type I
GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin…
- Trial name
- GDF 15 (NCT01201135)
- Volunteers
- 80 people
- Sponsor
- Wolfson Medical Center
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Sickle Cell Trait
Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada
Sickle cell disease (SCD) is an inherited blood disorder associated with acute illness and organ damage. In high resource settings, early screening and treatment greatly improve quality of life.
- Conditions
- Sickle Cell Disease, Sickle Cell Trait, Beta-Thalassemia, Sickle Cell-Beta Thalassemia
- Trial name
- NCT05506358
- Testing
- High performance liquid chromatography, Automated sickling test, HbS solubility test
- Where
- 3 sites in Canada, Nepal
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
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
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
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
Sickle Cell Disease, Sickle Beta Zero Thalassemia
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and…
- Conditions
- Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04688411
- Testing
- MED-Go App
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Hemoglobin SC Disease, Sickle Cell Anemia
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation…
- Trial name
- NCT00005783
- Testing
- Recombinant-methionyl human stem cell factor
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease, Sickle-Cell; Hb-SC
A Study of IMR-687 in Healthy Adult Volunteers
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
- Trial name
- NCT02998450
- Testing
- IMR-687
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Hemoglobinopathies, Sickle Cell Disease
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Thalassemia
- Trial name
- NCT00153985
- Testing
- Busulfex, Fludarabine, Alemtuzumab
- Where
- 6 sites in United States
- Volunteers
- 2 people
- Sponsor
- Dana-Farber Cancer Institute
Leukemia, Lymphoma
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well…
- Conditions
- Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies
- Trial name
- NCT01049854
- Testing
- Full Intensity with TBI, Full Intensity, Reduced Intensity
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Thalassemia, Sickle Cell Disease
Iron Overload and Growth Velocity in Thalassemia and Sickle Cell Anemia
Iron overload impaired growth in Thalassemia patients due to iron deposition in the endocrine glands, including the hypophysis and gonads.
- Trial name
- NCT00972231
- Testing
- Medical Chart Summary
- Where
- Israel
- Volunteers
- 93 people
- Sponsor
- HaEmek Medical Center, Israel
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics