Clinical trials: Thalassemia Intermedia
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.
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
Beta-Thalassemia, Hepatitis C
Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus
Patients with transfusion dependent Beta Thalassemia suffer from a high incidence of Hepatitis C infection especially in developed countries as Egypt.
- Trial name
- NCT03961828
- Testing
- Ledipasvir 90 MG / Sofosbuvir 400 MG [Harvoni]
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Tanta University
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
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia
Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia
The current study is to investigate the potential roles of N-acetyl cysteine and Alpha-lipoic acid in patients with beta-thalassemia.
- Trial name
- NAC/ALA (NCT07157722)
- Testing
- Iron chelating intervention, N Acetyl cysteine 600mg, Alpha Lipoic Acid 600 MG Oral Tablets
- Where
- Egypt
- Volunteers
- 66 people
- Sponsor
- Tanta University
Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity
- Trial name
- NCT05584956
- Testing
- L_carnitine, Sildenafil
- Where
- Egypt
- Volunteers
- 44 people
- Sponsor
- Tanta University
Sickle Cell Anemia, Sickle - Beta Thalassemia
Hepcidin Levels in Sickle Cell Disease (SCD)
The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when…
- Trial name
- NCT02258997
- Volunteers
- 42 people
- Sponsor
- Kenneth Ataga, MD