Clinical trial NCT03275051: Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)
Official title: A Long-term Safety and Efficacy follow-on Study in Participants With Transfusion Dependent Beta-thalassemia Who Have Previously Received OTL-300 (Formerly Know as GSK2696277)) and Completed the TIGET-BTHAL Study
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Beta Thalassaemia
Keywords: Beta Thalassaemia
previously GSK2696277
Lentiviral vector
TIGET-BTHAL
Gene therapy
Lead sponsor: IRCCS San Raffaele
Sponsor class: OTHER
Collaborators: name: Telethon Institute for Gene Therapy (OSR-TIGET)
First posted: 2017-09-07
Enrollment: 9
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: No study treatment will be administered in this study. Subjects who have received treatment with OTL-300 in and completed study TIGET-BTHAL will be included in this study.
Primary purpose: OTHER

Brief summary: OTL-300 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 OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).


Eligibility 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 OTL-300.
* 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

Primary outcomes:
measure: Number of subjects with absence of abnormal clonal proliferation (ACP)
timeFrame: Up to 6 years
measure: Number of subjects with Polyclonal engraftment
timeFrame: Up to 6 years
Secondary outcomes:
measure: Number of subjects with reduction in red blood cells (RBC) transfusion volume
timeFrame: Up to 6 years
measure: Number of subjects with reduction in transfusion rate up to transfusion independence
timeFrame: Up to 6 years
measure: Number of subjects with transfusion independence
timeFrame: Up to 6 years
measure: Hemoglobin (Hb) levels in subjects achieving transfusion independence
timeFrame: Up to 6 years
measure: Number of subjects with sustained engraftment of genetically corrected cells
timeFrame: Up to 6 years
measure: Number of subjects with overall survival
timeFrame: Up to 6 years
measure: Number of subjects with adverse events (AEs), serious AEs (SAEs)
timeFrame: Up to 6 years
measure: Clinical chemistry laboratory parameters as a measure of safety
timeFrame: Up to 6 years
measure: Hematology laboratory parameters as a measure of safety
timeFrame: Up to 6 years
measure: Urinalysis as a measure of safety
timeFrame: Up to 6 years
measure: Occurrence of viral infections as a measure of safety
timeFrame: Up to 6 years
measure: Screening for occurrence of antibodies against viruses and toxoplasma as a measure of safety
timeFrame: Up to 6 years
measure: Functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) scores
timeFrame: Up to 6 years
measure: Short-Form-36 (SF-36) scores
timeFrame: Up to 6 years
measure: Pediatric Quality of Life (PedsQL) questionnaire scores
timeFrame: Up to 6 years
measure: Evaluation of growth in pediatric subjects
timeFrame: Up to 6 years
measure: Assessment of hormonal levels in pediatric subjects
timeFrame: Up to 6 years
measure: Changes in puberty status as assessed by clinical examination
timeFrame: Up to 6 years
measure: Changes in puberty status as assessed by Tanner scale (TS)
timeFrame: Up to 6 years
measure: Changes in puberty status as assessed by general questioning
timeFrame: Up to 6 years
Other outcomes:
Locations:
city: Milan
country: Italy
facility: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Study officials:
affiliation: Ospedale San Raffaele- Telething Institute for Gene Therapy (SR-TIGET)
name: Orchard Clinical Trials
role: PRINCIPAL_INVESTIGATOR
References: