Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

NCT03275051 | Active Not Recruiting

• 2 other identifiers: 2077572017-001366-14
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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).

Conditions

Beta Thalassaemia

Keywords

Beta Thalassaemia; previously GSK2696277; Lentiviral vector; TIGET-BTHAL; Gene therapy

Outcome Measures

Primary Outcomes (2)

• Number of subjects with absence of abnormal clonal proliferation (ACP)

  Clonal proliferation describes the selection and reproduction of only one type of cell.

  Up to 6 years

• Number of subjects with Polyclonal engraftment

  Integration site analysis will be performed on different hematopoietic lineages from peripheral blood and/or bone marrow. Polyclonality of hematopoiesis is defined as \>1000 unique integration sites retrieved at specified time points. The number of subjects with polyclonality of hematopoiesis will be estimated.

  Up to 6 years

Secondary Outcomes (20)

• Number of subjects with reduction in red blood cells (RBC) transfusion volume

  Up to 6 years

• Number of subjects with reduction in transfusion rate up to transfusion independence

  Up to 6 years

• Number of subjects with transfusion independence

  Up to 6 years

• Hemoglobin (Hb) levels in subjects achieving transfusion independence

  Up to 6 years

• Number of subjects with sustained engraftment of genetically corrected cells

  Up to 6 years

Study Arms (1)

All subjects

EXPERIMENTAL

Subjects who have received treatment with OTL-300 in and completed study TIGET-BTHAL will be included in this study. Subjects received OTL-300 injection administered intraosseously in TIGET-BTHAL study. No study treatment will be administered in this study (207757).

Other: Safety and Efficacy assessments

Interventions

Safety and Efficacy assessments OTHER

Safety and efficacy assessment of OTL-300 in subjects with transfusion dependent beta-thalassemia will be performed.

All subjects

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• None

Sponsors & Collaborators

• IRCCS San Raffaele: lead
• Telethon Institute for Gene Therapy (OSR-TIGET): collaborator

Study Sites (1)

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan, 20132, Italy

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Safety

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Orchard Clinical Trials

  Ospedale San Raffaele- Telething Institute for Gene Therapy (SR-TIGET)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: No study treatment will be administered.
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dr

Model Details: 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.

Study Record Dates

• First Submitted: August 16, 2017
• First Posted: September 7, 2017
• Study Start: October 4, 2017
• Primary Completion: December 1, 2025
• Study Completion (Estimated): June 1, 2026
• Last Updated: November 23, 2022

Record last verified: 2022-11

Locations

IT (1)