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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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3 March 2025 |
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Main ID: |
NCT02698579 |
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Date of registration:
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17/02/2016 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
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Scientific title:
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Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product |
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Date of first enrolment:
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January 22, 2016 |
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Target sample size:
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64 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT02698579 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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Algeria
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Argentina
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Australia
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Brazil
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France
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Germany
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Italy
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Netherlands
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United Kingdom
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United States
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Contacts
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Name:
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Vinod K Prasad, MD, FRCP |
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Address:
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Telephone:
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Email:
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Affiliation:
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bluebird bio, Inc. |
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Key inclusion & exclusion criteria
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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
Exclusion Criteria:
- There are no exclusion criteria for this study
Age minimum:
N/A
Age maximum:
19 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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X-Linked Adrenoleukodystrophy (X-ALD)
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Adrenoleukodystrophy (ALD)
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Cerebral Adrenoleukodystrophy (CALD)
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Intervention(s)
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Genetic: No interventional drug product utilized in this follow-up study
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Primary Outcome(s)
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Major functional disability (MFD)-free survival
[Time Frame: 15 years post-drug-product infusion]
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Number of participants with malignancies
[Time Frame: 15 years post-drug-product infusion]
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Number of participants who experience graft versus host disease (GVHD)
[Time Frame: 15 years post-drug-product infusion]
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Number of participants with immune-related adverse events (AEs)
[Time Frame: 15 years post-drug-product infusion]
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Number of participants with new or worsening neurologic disorders
[Time Frame: 15 years post-drug-product infusion]
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Number of participants with new or worsening hematologic disorders
[Time Frame: 15 years post-drug-product infusion]
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Secondary Outcome(s)
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Change from baseline in neurological function score (NFS)
[Time Frame: 15 years post-drug-product infusion]
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Number of participants who undergo subsequent stem cell transplantation
[Time Frame: 15 years post-drug-product infusion]
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Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
[Time Frame: 15 years post-drug-product infusion]
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Secondary ID(s)
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LTF-304
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2015-002805-13
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2024-513904-33-00
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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