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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

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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02698579