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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02698579




Registration number
NCT02698579
Ethics application status
Date submitted
17/02/2016
Date registered
3/03/2016
Date last updated
24/12/2019

Titles & IDs
Public title
Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Scientific title
Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Secondary ID [1] 0 0
LTF-304
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral Adrenoleukodystrophy (CALD) 0 0
Adrenoleukodystrophy (ALD) 0 0
X-Linked Adrenoleukodystrophy (X-ALD) 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Safety and efficacy assessments

Subjects treated with Lenti-D - Subjects who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical trial) and who meet the eligibility criteria for the study LTF-304.


Other interventions: Safety and efficacy assessments
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of subjects who experience graft versus host disease (GVHD)
Timepoint [1] 0 0
15 years post-drug-product infusion
Primary outcome [2] 0 0
Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion)
Timepoint [2] 0 0
15 years post-drug-product infusion
Primary outcome [3] 0 0
All drug product-related AEs
Timepoint [3] 0 0
15 years post-drug-product infusion
Primary outcome [4] 0 0
All SAEs (regardless of relatedness to drug product)
Timepoint [4] 0 0
15 years post-drug-product infusion
Primary outcome [5] 0 0
Serious or non-serious immune-related AEs and new or worsening hematologic or neurologic disorders or malignancies
Timepoint [5] 0 0
15 years post-drug-product infusion
Primary outcome [6] 0 0
Incidence of vector-derived RCL, assessed from archived samples as clinically indicated.
Timepoint [6] 0 0
5 years post-drug-product infusion
Primary outcome [7] 0 0
Monitoring for clinical signs of oncogenesis
Timepoint [7] 0 0
15 years post-drug-product infusion
Primary outcome [8] 0 0
MFD-free survival
Timepoint [8] 0 0
15 years post-drug-product infusion
Primary outcome [9] 0 0
Monitoring for clonal dominance using integration site analysis (ISA)
Timepoint [9] 0 0
15 years post-drug-product infusion
Secondary outcome [1] 0 0
Overall Survival of subjects with cerebral adrenoleukodystrophy (CALD) treated with Lenti-D
Timepoint [1] 0 0
15 years post-drug-product infusion
Secondary outcome [2] 0 0
Change from baseline in Neurological Function Score (NFS)
Timepoint [2] 0 0
15 years post-drug-product infusion
Secondary outcome [3] 0 0
Change in gadolinium enhancement (GdE) status from last MRI performed in parent study.
Timepoint [3] 0 0
15 years post-drug-product infusion

Eligibility
Key inclusion criteria
- 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.

- Have received Lenti-D Drug Product in a parent clinical study.

- Able to comply with study requirements.
Minimum age
No limit
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- There are no exclusion criteria for this Study.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
Algeria
State/province [4] 0 0
Constantine
Country [5] 0 0
Argentina
State/province [5] 0 0
Buenos Aires
Country [6] 0 0
Brazil
State/province [6] 0 0
São Paulo
Country [7] 0 0
France
State/province [7] 0 0
Cedex
Country [8] 0 0
United Kingdom
State/province [8] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
bluebird bio
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a multi-center, long-term safety and efficacy follow-up study for subjects with
cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent
clinical study.

After completing a parent clinical study (approximately 2 years), eligible subjects will be
followed for an additional 13 years for a total of 15 years post-drug product infusion. No
investigational drug product will be administered in this study.
Trial website
https://clinicaltrials.gov/show/NCT02698579
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Elizabeth McNeil, MD
Address 0 0
bluebird bio, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02698579