Clinical trials: Beta-Thalassemia
Beta-Thalassemia
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting.
- Trial name
- NCT06271512
- Testing
- No Intervention
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Genetix Biotherapeutics Inc.
Thalassemia Major
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in…
- Trial name
- NCT03637556
- Testing
- DST-0509, Jadenu, Exjade
- Where
- 4 sites in United States, Thailand
- Volunteers
- 25 people
- Sponsor
- DisperSol Technologies, LLC
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
- Conditions
- Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
- Trial name
- NCT05444894
- Testing
- EDIT-301
- Where
- 8 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Editas Medicine, Inc.
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene