Clinical trials: Thalassemia, Beta
Beta-thalassemia, Serum Ferritin
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
safety and efficacy of different iron chelation therapy in transfusion dependent beta-thalaasemia patients with low serum ferritin and continued regular transfusion regimen.
- Conditions
- Beta-thalassemia, Serum Ferritin, Iron Chelation Therapy
- Trial name
- NCT01996683
- Testing
- desferal, ferriprox, blood transfusion
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Ain Shams University
Beta-Thalassemia, Thalassemia
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone.
- Trial name
- NCT07884773
- Testing
- Thalidomide, Danazol, Prednisolone
- Where
- Pakistan
- Volunteers
- 172 people
- Sponsor
- Khyber Medical University Peshawar
Hemoglobinopathies
Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh
Hemoglobinopathies represent a collection of genetic conditions that influence the structure or synthesis of haemoglobin, the protein found in red blood cells that facilitates oxygen transport from the lungs throughout the body.
- Trial name
- Hb variants (NCT07207551)
- Where
- Bangladesh
- Volunteers
- 760 people
- Sponsor
- Bangladesh Bioscience Research Group
Transfusion-dependent Beta-Thalassemia
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT06219239
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids (NCT07517133)
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Anemia, Cooley's, Beta-Thalassemia
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
The purpose of the TCRN is to accelerate research in the management of thalassemia, standardize existing treatments, and evaluate new ones in a network of clinical centers in North America.
- Conditions
- Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia
- Trial name
- NCT00000623
- Testing
- Deferoxamine, Deferiprone, Arginine
- Where
- 5 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- Carelon Research
Transfusion Dependent Beta Thalassemia
Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia
Blood transfusion and iron-chelation therapy have prolonged and improved the quality of life in patients with β-thalassemia. The improvement was mainly due to the decrease in mortality from heart failure Such a treatment…
- Trial name
- NCT07370922
- Testing
- Dulaglutide 0.75Mg/0.5Ml Inj Pen, control group (insulin)
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids-T (NCT07506863)
- Testing
- Mitapivat
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Beta-Thalassemia
Dermatological Abnormalities in Beta-thalassemia Major
to study the frequency and pattern of dermatological abnormalities in egyptian childern with beta thalassemia.relationof abnormalities to duration of disease and frequency of transfusion
- Trial name
- NCT03894605
- Testing
- thalassemia
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Assiut University
β-thalassemia
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT05773729
- Testing
- BD211
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.
- Trial name
- NCT04176653
- Where
- 5 sites in Bulgaria, United Kingdom
- Sponsor
- Silence Therapeutics plc
Incentive Spirometer, Pulmonary Function
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
The study was done to: 1. Investigate the effect of respiratory training on functional lung capacity and 2. To detect the effect of respiratory training on pulmonary functions in children with β-thalassemia major. 3.
- Conditions
- Incentive Spirometer, Pulmonary Function, Functional Capacity, Child, Only
- Trial name
- NCT07112703
- Testing
- Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing:
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Rawan Mohammed Khairy Mostafa Elsawy
Stem Cell Transplantation
A Clinical Study of Ultra-transplantation for the Treatment of Major Thalassemia Scheme
Yunnan is a high-incidence area of Eastern Mediterranean (thalassemia) in China, and the treatment cost of thalassemia patients is high, hematopoietic stem cell transplantation (HSCT) is the only means to cure thalassemia, but…
- Trial name
- UTTS (NCT06743477)
- Testing
- Supergraft
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Hu Peng
Beta Thalassaemia
Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)
OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene.
- Trial name
- NCT03275051
- Testing
- Safety and Efficacy assessments
- Where
- Italy
- Volunteers
- 9 people
- Sponsor
- IRCCS San Raffaele
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Blood Disease
Cystatin c and Beta 2 Microglobulin in Thalassemic Children.
Beta thalassemia has many complications on many systems as the renal system.So early detection of renal impairment is required in those children to decrease complications of this nephropathy.
- Trial name
- NCT03881917
- Testing
- Cystatin c and beta 2 microglobulin kits
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Assiut University
Thalassemia, Beta
Long-term Follow-up Study of BHC001 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
- Trial name
- NCT06314529
- Testing
- Assessments
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Bioray Laboratories
Beta-Thalassemia
Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major
Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions.
- Trial name
- NCT00103753
- Testing
- deferiprone
- Where
- Italy
- Volunteers
- 65 people
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust