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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02698579
Ethics application status
Date submitted
17/02/2016
Date registered
3/03/2016
Date last updated
19/08/2024

Titles & IDs
Public title
Long-term Follow-up of Participants 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
2015-002805-13
Secondary ID [2] 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
Treatment: Other - No interventional drug product utilized in this follow-up study

Long-term followup - Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies).


Treatment: Other: No interventional drug product utilized in this follow-up study
Participants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104.

The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Major functional disability (MFD)-free survival
Timepoint [1] 0 0
15 years post-drug-product infusion
Primary outcome [2] 0 0
Number of participants with malignancies
Timepoint [2] 0 0
15 years post-drug-product infusion
Primary outcome [3] 0 0
Number of participants who experience graft versus host disease (GVHD)
Timepoint [3] 0 0
15 years post-drug-product infusion
Primary outcome [4] 0 0
Number of participants with immune-related adverse events (AEs)
Timepoint [4] 0 0
15 years post-drug-product infusion
Primary outcome [5] 0 0
Number of participants with new or worsening hematologic disorders
Timepoint [5] 0 0
15 years post-drug-product infusion
Primary outcome [6] 0 0
Number of participants with new or worsening neurologic disorders
Timepoint [6] 0 0
15 years post-drug-product infusion
Secondary outcome [1] 0 0
Number of participants who undergo subsequent stem cell transplantation
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
Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
Timepoint [3] 0 0
15 years post-drug-product infusion

Eligibility
Key inclusion criteria
* Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
* Have received eli-cel in a parent clinical study
Minimum age
No limit
Maximum age
19 Years
Sex
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/01/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2038
Actual
Sample size
Target
Accrual to date
Final
64
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
Argentina
State/province [4] 0 0
Caba
Country [5] 0 0
Brazil
State/province [5] 0 0
São Paulo
Country [6] 0 0
France
State/province [6] 0 0
Cedex
Country [7] 0 0
Germany
State/province [7] 0 0
Leipzig
Country [8] 0 0
Italy
State/province [8] 0 0
Rome
Country [9] 0 0
Netherlands
State/province [9] 0 0
Utrecht
Country [10] 0 0
United Kingdom
State/province [10] 0 0
England
Country [11] 0 0
United Kingdom
State/province [11] 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 participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104).

After completing a parent clinical study (approximately 2 years), eligible participants 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/study/NCT02698579
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Vinod K Prasad, MD, FRCP
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/study/NCT02698579