A follow-on study in patients who have received gene therapy for Beta-thalassemia in the TIGET-BTHAL study to assess safety and effectiveness of treatment.
- Conditions
- Beta-thalassemiaMedDRA version: 20.1Level: LLTClassification code 10054660Term: Thalassemia betaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2017-001366-14-IT
- Lead Sponsor
- GLAXOSMITHKLINE RESEARCH AND DEVELOPMENT
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 10
Participants are eligible to be included in the study only if all of the following criteria apply:
1.Participants who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with GSK2696277.
2.For adults; capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. For children; informed assent and/or consent in writing signed by the subject and/or parent(s) / legal representative (according to local regulations and age of the subject).
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
None
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method