ongterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)MedDRA version: 20.0Level: PTClassification code 10051260Term: AdrenoleukodystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2015-002805-13-FR
- Lead Sponsor
- bluebird bio, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 25
1. Provision of written informed consent for this study by the subject or subject’s parent(s)/ legal guardian(s) and written informed assent by subject, if applicable
2. Have received Lenti-D Drug Product in Study ALD-102
3. Able to comply with study requirements
Are the trial subjects under 18? yes
Number of subjects for this age range: 25
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Met the VCN discontinuation criterion from Study ALD-102, as follows:
the subject has undetectable VCN (<0.0003 copies per cell) in peripheral blood cells for 2 consecutive measurements at least 1 month apart and at least 12 months after drug product infusion.
This exclusion criterion does not apply to subjects who received allo-HSCT after Lenti-D Drug Product infusion in Study ALD-102
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