Clinical trials: Thalassemia, Beta
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Thalassemia Majors (Beta-Thalassemia Major), Haplo-identical Donors
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
- Trial name
- NCT06734520
- Testing
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Where
- 2 sites in China
- Volunteers
- 3 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Transfusion Dependent Beta Thalassemia
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with…
- Trial name
- NCT06678165
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 3 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Myelodysplastic Syndrome, Beta Thalassemia
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia.
- Trial name
- NCT06073860
- Testing
- Luspatercept
- Where
- 2 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- Bristol-Myers Squibb
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness…
- Trial name
- NCT06664541
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Georgetown University
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
Beta Thalassemia Major, Sickle Beta 0 Thalassemia
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies).
- Conditions
- Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia, Sickle Beta Thalassemia
- Trial name
- NCT04776850
- Testing
- Bortezomib, Busulfan, Cyclophosphamide
- Sponsor
- M.D. Anderson Cancer Center
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
Thalassemia Major
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT).
- Trial name
- NCT04009525
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 823 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Beta-thalassemia, Glioma
Plasticizer Exposure and Its Consequences on Health
Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects…
- Conditions
- Beta-thalassemia, Glioma, Healthy Controls, Transfusion-dependent Beta-Thalassemia
- Trial name
- PEACH (NCT06512298)
- Where
- Austria
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Medical University of Vienna
Beta-Thalassemia
Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen
repeated transfusions are the mainstay of disease management in most patients with transfusion dependent beta thalassemia.iron overload predispose to oxidative stress and tissue injury.
- Trial name
- NCT06509581
- Testing
- vitamin E
- Where
- Egypt
- Volunteers
- 180 people
- Sponsor
- Ain Shams University
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
Preterm Birth, Oxygen Toxicity
Fetal Haemoglobin and Cerebral and Peripheral Oxygenation.
The aim of this study is to investigate the relationship between cerebral and peripheral oxygenation and oxygen extraction, as measured by NIRS (near-infrared spectroscopy ), and the FHbF (fraction of fetal hemoglobin) and…
- Conditions
- Preterm Birth, Oxygen Toxicity, Fetal Hemoglobin
- Trial name
- HbFIN (NCT04802629)
- Testing
- Near-infrared spectroscopy
- Where
- Austria
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Beta Thalassemia Major
Inspiratory Muscle Training in Children With Beta Thalasemia
respiratory complications are very prevalent in children with beta thalasemia major (BTM), so choosing inspiratory muscle trianing to decrease of prevent it and to improve pulmoanry funntions is important
- Trial name
- NCT06466304
- Testing
- inspiratory muscle training
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Ahram Canadian University
Sickle Cell Disease, Beta Thalassemia
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease.
- Trial name
- NCT00408447
- Testing
- Busulfan, Fludarabine, Alemtuzumab
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Columbia University
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
EKLF Mutations in Beta Thalassemia Patients
Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients
Detection of KLF1 gene mutations in patients with beta thalassemia considering the alpha and beta molecular status of these patients.
- Trial name
- NCT06433570
- Testing
- multiplex PCR
- Volunteers
- 100 people
- Sponsor
- Assiut University
B Thalassemia
Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia
to evaluate thyroid function ,HbA1C,\&lipid profile in thalassemic patient to correlate thyroid function , Hba1c , lipid profile e ferritin level in thalassemic patient
- Trial name
- NCT06377865
- Volunteers
- 72 people
- Sponsor
- Assiut University