Clinical trials: Thalassemia Intermedia
Beta-thalassemia Major
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients
Background: Three iron chelators now available on the market differ in toxicity and organ specificity; evidence on standardized chelation protocol remains inconclusive, but patients with transfusion-dependent beta-thalassemia…
- Trial name
- NCT02198508
- Testing
- DFX(Deferasirox), DFP(Deferiprone)
- Volunteers
- 13 people
- Sponsor
- China Medical University Hospital
Beta-Thalassemia Trait, Coronary Artery Disease
B-Thalasaemia Trait and Its Possible Cardioprotective Role
Our purpose is to investigate whether thallasaemia trait offers protection against cardiovascular events and whether this can be applied to specific characteristics of these people ( better lipid profile , better blood pressure…
- Trial name
- NCT02428023
- Where
- Greece
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- 404 General Military Hospital, Larissa
Thalassemia
Assessment of Nutritional Status and Role of Insulin-like Growth Factor-1 in Children
Many studies done on children with thalassemia have shown a variable prevalence of defective IGF-I concentrations have been shown to be low in the majority of children, with or without GH deficiency.
- Trial name
- NCT05529550
- Where
- Egypt
- Volunteers
- 102 people
- Sponsor
- Menoufia University
Sickle Cell Anemia in Children
Gum Arabic as Anti-oxidant, Anti-inflammatory and Fetal Hemoglobin Inducing Agent in Sickle Cell Anemia Patients
To study the efficacy of Gum Arabic as an anti-oxidant, anti-inflammatory and Fetal Hemoglobin-inducing agent among Sickle Cell Disease children. Half of participants will receive Gum Arabic and the other half will receive placebo
- Trial name
- GA&SCA (NCT04191213)
- Testing
- Acacia Senegal extract, Pectin
- Where
- Sudan
- Volunteers
- 100 people
- Sponsor
- Al-Neelain University
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Transfusion-dependent Beta-Thalassemia
Β-Thalassemia Treatment with KL003 Cell Injection
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with…
- Trial name
- NCT06280378
- Testing
- KL003 Cell Injection Drug Product
- Where
- 2 sites in China
- Volunteers
- 41 people
- Sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
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
Thalassemia Intermedia
Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
The study hypothesis that treatment with Erythropoietin (EPO) combined with Human Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia intermedia patients.
- Trial name
- NCT01624038
- Testing
- Hydroxyurea ,Epiao, hydroxyurea, blood transfusion
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Ain Shams 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
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients.
- Trial name
- NCT06239389
- Testing
- Thalidomide 100mg, Hydroxyurea
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Beta-Thalassemia
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)
In β-thalassaemia and Sickle Cell Disease (SCD), a significant production of fetal haemoglobin (HbF) may reduce the severity of clinical course and reactivation of γ-globin gene expression in adulthood.
- Trial name
- THALA-RAP (NCT04247750)
- Testing
- Sirolimus 0.5 mg
- Where
- 4 sites in Italy
- Volunteers
- 45 people
- Sponsor
- Università degli Studi di Ferrara
β-thalassemia Major
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
- Trial name
- MACS2163 (NCT01724138)
- Testing
- Deferasirox
- Sponsor
- Novartis Pharmaceuticals
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Sickle Cell Disease, Beta Thalassemia Intermedia
Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3.
- Trial name
- NCT05883254
- Testing
- RNA Binding Protein Pumilio1 (PUM1) Expression by reverse transcriptase quantatative PCR
- Volunteers
- 80 people
- Sponsor
- Assiut University
Thalassemia Intermedia, Thalassemia Major
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a…
- Conditions
- Thalassemia Intermedia, Thalassemia Major, Growth Delay, Puberty, Delayed
- Trial name
- NCT06931912
- Testing
- MENBS clinical interventions
- Where
- China
- Volunteers
- 385 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
β-thalassemia Major
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-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
- NCT05776173
- Testing
- BD211
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Sickle Cell Disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical…
- Trial name
- NYMC-571 (NCT02675959)
- Testing
- Defibrotide
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
β Thalassemia
A Study of IMR-687 in Subjects With Beta Thalassemia
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
- Trial name
- NCT04411082
- Testing
- IMR-687
- Where
- 36 sites in Denmark, France and 11 more
- Volunteers
- 122 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.