Register a trial

Please note that the ANZCTR website will be unavailable from 1:00pm until 2:00pm (AEST) on Thursday 10th of April for website maintenance. Please be sure to log out of the system in order to avoid any loss of data. Thank you and apologies for any inconvenience caused.


The ANZCTR website is back online for trial registration and updates. We apologise for any inconvenience caused while the site was inactive.


With activity expected to increase on the ANZCTR again, there may be extended wait times while we process pending studies, with priority being given to those trials submitted in February. Thank you for your patience.


Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements.
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
Assessment method [1] 0 0
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
Timepoint [1] 0 0
15 years post-drug-product infusion
Primary outcome [2] 0 0
Number of participants with malignancies
Assessment method [2] 0 0
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)
Assessment method [3] 0 0
Timepoint [3] 0 0
15 years post-drug-product infusion
Primary outcome [4] 0 0
Number of participants with immune-related adverse events (AEs)
Assessment method [4] 0 0
Timepoint [4] 0 0
15 years post-drug-product infusion
Primary outcome [5] 0 0
Number of participants with new or worsening hematologic disorders
Assessment method [5] 0 0
Timepoint [5] 0 0
15 years post-drug-product infusion
Primary outcome [6] 0 0
Number of participants with new or worsening neurologic disorders
Assessment method [6] 0 0
Timepoint [6] 0 0
15 years post-drug-product infusion
Secondary outcome [1] 0 0
Number of participants who undergo subsequent stem cell transplantation
Assessment method [1] 0 0
Timepoint [1] 0 0
15 years post-drug-product infusion
Secondary outcome [2] 0 0
Change from baseline in neurological function score (NFS)
Assessment method [2] 0 0
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.
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)
Assessment method [3] 0 0
Contrast enhancement (gadolinium enhancement; GdE+) on brain MRI represents a clinically important radiographic biomarker of active neuroinflammatory disease and poor prognosis (in untreated patients). As such, assessment of the number of participants who remained negative for gadolinium enhancement (GdE-) was conducted for this outcome measure.
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
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


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