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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02698579
Registration number
NCT02698579
Ethics application status
Date submitted
17/02/2016
Date registered
3/03/2016
Date last updated
7/02/2024
Titles & IDs
Public title
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
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Scientific title
Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
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Secondary ID [1]
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2015-002805-13
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Secondary ID [2]
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LTF-304
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cerebral Adrenoleukodystrophy (CALD)
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Adrenoleukodystrophy (ALD)
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X-Linked Adrenoleukodystrophy (X-ALD)
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Condition category
Condition code
Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Metabolic and Endocrine
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0
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Metabolic disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Other interventions - Lenti-D Drug Product
Lenti-D Drug Product - Participants 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 will be followed in this observation study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent study).
Other interventions: Lenti-D Drug Product
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Major functional disability (MFD)-free survival
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Assessment method [1]
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The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
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Timepoint [1]
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15 years post-drug-product infusion
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Primary outcome [2]
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Number of participants with malignancies
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Assessment method [2]
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Timepoint [2]
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15 years post-drug-product infusion
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Primary outcome [3]
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Number of participants who experience graft versus host disease (GVHD)
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Assessment method [3]
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Timepoint [3]
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15 years post-drug-product infusion
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Primary outcome [4]
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Number of participants with immune-related adverse events (AEs)
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Assessment method [4]
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0
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Timepoint [4]
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15 years post-drug-product infusion
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Primary outcome [5]
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Number of participants with new or worsening hematologic disorders
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Assessment method [5]
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Timepoint [5]
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15 years post-drug-product infusion
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Primary outcome [6]
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Number of participants with new or worsening neurologic disorders
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Assessment method [6]
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Timepoint [6]
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15 years post-drug-product infusion
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Secondary outcome [1]
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Number of participants who undergo subsequent stem cell transplantation
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Assessment method [1]
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Timepoint [1]
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15 years post-drug-product infusion
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Secondary outcome [2]
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Change from baseline in neurological function score (NFS)
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Assessment method [2]
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The NFS is a 25-point score used to evaluate the severity of gross neurologic dysfunction in CALD by scoring 15 symptoms (functional domains) across 6 categories. Listed here are the 15 symptoms followed by their maximal score out of 25 points: a) Hearing / auditory processing problems-1, b) Aphasia / apraxia-1, c) Loss of communication-3, d) Vision impairment /field cut-1, e) Cortical blindness-2, f) Swallowing / other central nervous system (CNS) dysfunctions-2, g) Tube feeding-2, h) Running difficulties / hyperreflexia-1, i) Walking difficulties / spasticity / spastic gait (no assistance)-1, j) Spastic gait (needs assistance)-2, k) Wheelchair dependence-2, l) Complete loss of voluntary movement-3, m) Episodes of incontinence -1, n) Total incontinence-2, o) Nonfebrile seizures-1. A score of "0" denotes absence of clinical signs of cerebral disease. Maximal signs within a domain score the total of all grades within that domain.
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Timepoint [2]
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15 years post-drug-product infusion
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Secondary outcome [3]
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Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
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Assessment method [3]
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GdE status means participants who will report GdE negative (-) or positive (+).
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Timepoint [3]
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15 years post-drug-product infusion
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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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- There are no exclusion criteria for this study
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
22/01/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/08/2038
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Actual
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Sample size
Target
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Accrual to date
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Final
64
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Women's and Children's Hospital - North Adelaide
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Recruitment postcode(s) [1]
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- North Adelaide
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Massachusetts
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Country [3]
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United States of America
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State/province [3]
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Minnesota
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Country [4]
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Argentina
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State/province [4]
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Caba
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Country [5]
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Brazil
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State/province [5]
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São Paulo
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Country [6]
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France
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State/province [6]
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Cedex
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Country [7]
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Germany
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State/province [7]
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Leipzig
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Country [8]
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Italy
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State/province [8]
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Rome
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Country [9]
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Netherlands
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State/province [9]
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Utrecht
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Country [10]
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United Kingdom
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State/province [10]
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England
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Country [11]
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United Kingdom
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State/province [11]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
bluebird bio
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Address
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Country
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Ethics approval
Ethics application status
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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.
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02698579
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Vinod K Prasad, MD, FRCP
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Address
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bluebird bio, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02698579
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