null - FindTrial

Long-term Follow-up of Subjects Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-002245-11-FR

Enrollment

Registered

2014-05-15

Start date

2013-11-12

Completion date

Unknown

Last updated

2024-09-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Subjects treated with gene therapy drug products in a bluebird bio-sponsored or associated clinical study will be invited to participate in this long-term follow-up study to monitor the safety and efficacy of the drug products. To date, subjects in this study have been treated with bluebird bio gene therapy drug products developed to treat the following indications: beta-thalassaemia major MedDRA version: 17.0 Level: LLT Classification code 10055579 Term: Sickle-cell beta thalassemia System O

Interventions

Product Name: LentiGlobin HPV569 Pharmaceutical Form: Suspension for injection

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Provision of written informed consent for this study by subject, or as applicable, subject’s parent(s)/ legal guardian(s). 2. Previously treated with drug product in a bluebird bio-sponsored or associated clinical study. Are the trial subjects under 18? no Number of subjects for this age range: 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

Exclusion criteria

Exclusion criteria: 1.Met the VCN discontinuation criterion in the parent study, as follows: Undetectable VCN (<0.002 copies per cell) in relevant peripheral blood samples for 2 consecutive measurements at least 1 month apart, minimally at 12 months post-transplant. An additional measurement must have been performed in unsorted peripheral blood, and the VCN must have been =0.0003 to confirm the previous findings.

Design outcomes

Primary

Measure	Time frame
Main Objective: Monitor the long-term safety and efficacy of gene therapy drug products used in bluebird bio-sponsored or associated clinical studies. ;Secondary Objective: Not Applicable;Primary end point(s): Subjects treated with gene therapy drug products will be monitored for 15 years post-transplant. ;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.	—

Measure

Time frame

Main Objective: Monitor the long-term safety and efficacy of gene therapy drug products used in bluebird bio-sponsored or associated clinical studies. ;Secondary Objective: Not Applicable;Primary end point(s): Subjects treated with gene therapy drug products will be monitored for 15 years post-transplant. ;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.

—

Secondary

Measure	Time frame
Secondary end point(s): Not Applicable;Timepoint(s) of evaluation of this end point: Not Applicable	—

Countries

France, Germany, Greece, Italy, United Kingdom

Contacts

Public ContactPatrice MARCHAND

NUVISAN ONCOLOGY SA

patrice.marchand@nuvisan.com33145154082

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026