Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

• Register: EUCTR
• Last refreshed on: 26 October 2020
• Main ID: EUCTR2015-002805-13-GB
• Date of registration: 09/09/2015
• Prospective Registration: Yes
• Primary sponsor: bluebird bio, Inc
• Public title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
• Scientific title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
• Date of first enrolment: 27/11/2015
• Target sample size: 60
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002805-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Algeria, Argentina, Australia, Brazil, France, United Kingdom, United States

Contacts

• Name: Clinical Trial Information
• Affiliation: bluebird bio, Inc.

• Name: Clinical Trial Information
• Affiliation: bluebird bio, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
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 eli - cel Product in a parent clinical study.
3.Able to comply with study requirements.

Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
There are no exclusion criteria for this Study.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Cerebral Adrenoleukodystrophy (CALD)
MedDRA version: 20.0 Level: PT Classification code 10051260 Term: Adrenoleukodystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Pharmaceutical Form:

Primary Outcome(s)

Primary end point(s): 1. Safety evaluations include:
•Proportion of subjects who experience graft versus host disease (GVHD)
•Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion)
•All drug product-related AEs through 15 years post-drug-product infusion
•All serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product)
• Immune-related AEs through 15 years post-drug product infusion
• Incidence of vector-derived RCL, assessed from archived samples as clinically indicated.
•The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.)
•The number of subjects with clonal predominance
2. Efficacy Endpoints include:
•MFD-free survival

Main Objective: - Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel)administered in parent clinical studies.
- Monitor for long-term efficacy of eli-cel administered in parent clinical studies.

Secondary Objective: Not Applicable

Timepoint(s) of evaluation of this end point: Visits are scheduled every 6 months for years 2 to 5 post-drug-product infusion and then annually through 15 years post-drug-product infusion.

Secondary Outcome(s)

Secondary end point(s): Secondary efficacy endpoints include the following:
- Overall survival.
- Change from Baseline (defined in parent study) in NFS.
- Gadolinium enhancement (GdE) status

Timepoint(s) of evaluation of this end point: As above

Secondary ID(s)

NCT02698579

LTF-304

Source(s) of Monetary Support

bluebird bio, Inc

Ethics review

Status: Approved
Approval date: 27/11/2015
Contact: