Last updated: 06/23/2025 08:50:24
Long-term safety and efficacy follow-up of subjects treated with GSK2696277 for transfusion dependent beta-thalassemia in San Raffaele Telethon Institute of Gene Therapy-Beta Thalassemia (TIGET-BTHAL) study
GSK study ID
207757
Clinicaltrials.gov ID
EudraCT ID
EU CT Number
Not applicable
Trial status
Recruiting
Recruiting
Trial overview
Official title: A long-term safety and efficacy follow-on study in participants with transfusion dependent beta-thalassemia who have previously received GSK2696277 (autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene) and completed the TIGET-BTHAL study
Trial description: GSK2696277 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. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of GSK2696277 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects who have undergone gene therapy are at risk of delayed adverse events. Thus, this study is designed as a longer term safety and efficacy follow-up of TIGET-BTHAL for a further six years (i.e. until eight years post-gene therapy) in subjects who previously completed the TIGET-BTHAL study. Approximately 10 subjects who have received treatment with GSK2696277 in and completed study TIGET-BTHAL will be included in this study. The entire post-gene therapy follow-up will be of eight years i.e. two years in TIGET-BTHAL plus six years in this study.
Primary purpose:
Treatment
Trial design:
Single Group Assignment
Masking:
None (Open Label)
Allocation:
Not applicable
Primary outcomes:
Number of subjects with absence of abnormal clonal proliferation (ACP)
Timeframe: Up to 6 years
Number of subjects with Polyclonal engraftment
Timeframe: Up to 6 years
Secondary outcomes:
Number of subjects with reduction in red blood cells (RBC) transfusion volume
Timeframe: Up to 6 years
Number of subjects with reduction in transfusion rate up to transfusion independence
Timeframe: Up to 6 years
Number of subjects with transfusion independence
Timeframe: Up to 6 years
Hemoglobin (Hb) levels in subjects achieving transfusion independence
Timeframe: Up to 6 years
Number of subjects with sustained engraftment of genetically corrected cells
Timeframe: Up to 6 years
Number of subjects with overall survival
Timeframe: Up to 6 years
Number of subjects with adverse events (AEs), serious AEs (SAEs)
Timeframe: Up to 6 years
Clinical chemistry laboratory parameters as a measure of safety
Timeframe: Up to 6 years
Hematology laboratory parameters as a measure of safety
Timeframe: Up to 6 years
Urinalysis as a measure of safety
Timeframe: Up to 6 years
Occurrence of viral infections as a measure of safety
Timeframe: Up to 6 years
Screening for occurrence of antibodies against viruses and toxoplasma as a measure of safety
Timeframe: Up to 6 years
Functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scores
Timeframe: Up to 6 years
Short-Form-36 (SF-36) scores
Timeframe: Up to 6 years
Pediatric Quality of Life (PedsQL) questionnaire scores
Timeframe: Up to 6 years
Evaluation of growth in pediatric subjects
Timeframe: Up to 6 years
Assessment of hormonal levels in pediatric subjects
Timeframe: Up to 6 years
Changes in puberty status as assessed by clinical examination
Timeframe: Up to 6 years
Changes in puberty status as assessed by Tanner scale (TS)
Timeframe: Up to 6 years
Changes in puberty status as assessed by general questioning
Timeframe: Up to 6 years
Interventions:
Enrollment:
10
Primary completion date:
2026-01-05
Observational study model:
Not applicable
Time perspective:
Not applicable
Clinical publications:
Not applicable
Medical condition
beta Thalassaemia
Product
GSK2696277
Collaborators
San Raffaele Telethon Institute of Gene Therapy (SR-TIGET)
Study date(s)
October 2017 to May 2026
Type
Interventional
Phase
2
Participation criteria
Sex
Female & Male
Age
3+ years
Accepts healthy volunteers
No
- Subjects who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with GSK2696277.
- For adults; capable of giving signed informed consent. 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).
- None
Inclusion and exclusion criteria
Inclusion criteria:
- Subjects who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with GSK2696277.
- For adults; capable of giving signed informed consent. 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).
Exclusion criteria:
- None
Trial location(s)
Study documents
No study documents available.
Results overview
Study Results yet to be posted
Recruitment status
Recruiting
Actual primary completion date
Not applicable
Actual study completion date
Not applicable
Plain language summaries
Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.
Additional information about the trial
Additional information
Not applicable
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