Clinical trials: Thalassemia Intermedia
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZE-T (NCT04770779)
- Testing
- Mitapivat
- Where
- 75 sites in United States, Brazil and 17 more
- Volunteers
- 258 people
- Sponsor
- Agios Pharmaceuticals, Inc.
MRI Scans
Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy
The overall goal of this research is to help develop a new magnetic resonance (MR) method, Quantitative Susceptibility Mapping (QSM), to improve the measurement of liver iron concentrations without the need for a liver biopsy.
- Trial name
- NCT04171635
- Testing
- Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
- Where
- 2 sites in United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Beta-Thalassemia
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.
- Trial name
- NCT00069862
- Testing
- Deferitrin (GT56-252), desferoxamine (DFO)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Beta-Thalassemia
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in…
- Trial name
- NCT06308159
- Testing
- Vebeglogene autotemcel
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Lantu Biopharma
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
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
Sickle Cell Anemia, Sickle Cell Thalassemia
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys.
- Conditions
- Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss
- Trial name
- NCT05675436
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Thalassemia, Beta
The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
Objectives Primary objectives: To determine the efficacy of Hydroxyurea in the study participants. Hypothesis: The study will result in either maintenance or rise in hemoglobin as compared to the control treatment.
- Trial name
- NCT03183375
- Testing
- Hydroxyurea
- Where
- Pakistan
- Volunteers
- 100 people
- Sponsor
- Dr.Saqib Hussain Ansari
Thalassemia, Iron Overload
Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AmloThal (NCT01395199)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- University of Campinas, Brazil
Sickle Cell Disease, Beta Thalassemia-Major
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a…
- Trial name
- NCT02435901
- Testing
- alemtuzumab (Campath IH), Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Northwell Health
HCV Infection, Beta Thalassemia Major
PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders
This is a prospective, controlled, open-label, pharmacokinetic study. This study aims at studying the PK of sofosbuvir, ledipasvir and sofosbuvir metabolite (GS-331007) in HCV infected children with hematological Disorders.
- Trial name
- NCT04353986
- Testing
- Sofosbuvir and Ledipasvir
- Where
- Egypt
- Volunteers
- 24 people
- Sponsor
- Ain Shams University
Beta-Thalassemia
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-thalassemia.
- Trial name
- NCT03271541
- Testing
- Bitopertin
- Where
- 4 sites in Italy, Lebanon and 1 more
- Volunteers
- 12 people
- Sponsor
- Hoffmann-La Roche
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
Iron Overload, Oxidative Stress
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
The aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production…
- Conditions
- Iron Overload, Oxidative Stress, Thalassemia Major
- Trial name
- NCT04292314
- Testing
- Omega 3, Nigella Sativa Oil, Hydroxyurea
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Beta-Thalassemia, Thalassemia Major
Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using…
- Trial name
- SEQDFPDFO (NCT00733811)
- Testing
- Deferiprone (DFP) and Deferoxamine (DFO), Deferiprone (DFP)
- Where
- Italy
- Volunteers
- 213 people
- Sponsor
- Azienda Ospedaliera V. Cervello
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Hepatitis C, Fibrosis
The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj Hospital
The primary objective is to assess the prevalence and genotypes of HCV infection in thalassemia major and thalassemia intermediate patients who have received blood transfusion in the Department of Medicine, Faculty of Medicine…
- Conditions
- Hepatitis C, Fibrosis, Thalassemia
- Trial name
- NCT02904252
- Testing
- Transient elastography
- Where
- Thailand
- Volunteers
- 158 people
- Sponsor
- Mahidol 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