Clinical trials: Beta-Thalassemia
Transfusion-dependent Beta-Thalassemia
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent…
- Trial name
- NCT02633943
- Testing
- Safety and efficacy assessments
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Genetix Biotherapeutics Inc.
Patients With Thalassemia Intermedia,, Congenital Dyserythropoietic Anemia Type I
GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin…
- Trial name
- GDF 15 (NCT01201135)
- Volunteers
- 80 people
- Sponsor
- Wolfson Medical Center
Thalassemia
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
- Trial name
- NCT03402191
- Testing
- L-arginine, Sildenafil
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sherief Abd-Elsalam
Myelodysplastic Syndromes, Beta-thalassemia
A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic…
- Trial name
- NCT07681440
- Testing
- Luspatercept
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Bristol-Myers Squibb
Beta Thalassemia Major, Thalassemia Majors (Beta-Thalassemia Major)
The Effect of Laughter Yoga in Beta Thalassaemia Major Patients: a Randomised Controlled Trial
The aim of this study was to evaluate the effects of laughter yoga on Fatigue, Depression-Anxiety-Stress, Sleep quality, Haematological values and Blood transfusion number in Beta Thalassemia Major patients.
- Trial name
- NCT06980662
- Testing
- laughter yoga
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Ataturk University
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
B Thalassemia
Assessment of Cognitive Functions, Psychological Symptoms, and Quality of Life Among B Thalassemia Patients and Their Clinical Correlates.
Multiple studies showed the negative impact thalassemia disease and its therapy have on the cognition of these patients. This study aims to assess and compare the cognition, psychological impact and quality of life among patients…
- Trial name
- NCT05105659
- Testing
- Wechsler IQ for Adults, MAS (Memory assessment scale), Health-Related Quality of Life (HRQoL)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assiut University
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
- Trial name
- NCT02380079
- Testing
- SCD-101
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Invenux, LLC
Thalassemia Major
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients.
- Trial name
- NCT03992001
- Testing
- Blood component A, Blood component B
- Where
- Italy
- Volunteers
- 55 people
- Sponsor
- Università degli Studi di Ferrara
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
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
AL-Hijama in Thalassmia Major
Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major
1. Evaluate the therapeutic effects, health benefits, and immunological effect of Al-hijamah in children with beta thalassemia major. 2.
- Trial name
- Al-hijamah (NCT02761395)
- Testing
- Al-hijamah, Deferasirox
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Beta-Thalassemia
A Study of EPEG in Beta Thalassemia Patients
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
- Trial name
- NCT02950857
- Testing
- EPEG
- Where
- Thailand
- Volunteers
- 6 people
- Sponsor
- Prolong Pharmaceuticals
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
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
Sickle Cell Disease
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease
This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).
- Trial name
- NCT07431398
- Testing
- Pociredir
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Fulcrum Therapeutics
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
- Trial name
- NCT06479616
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Fudan University
Thalassemia Major
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload…
- Trial name
- NCT05303506
- Testing
- serum ferritin and AST
- Volunteers
- 50 people
- Sponsor
- Assiut University
Beta-Thalassemia
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.
- Trial name
- NCT06717932
- Where
- China
- Volunteers
- 8 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd