An observational study in Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD) and X-Linked Adrenoleukodystrophy (X-ALD), sponsored by Genetix Biotherapeutics Inc.. Active, not recruiting at 13 sites in 9 countries. Open to male participants aged Up to 19 Years. Per ClinicalTrials.gov, last updated 2026-09-14.
Sponsored by Genetix Biotherapeutics Inc. · Observational
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 64 is below the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Genetix Biotherapeutics Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study will be expected to participate in this long-term follow-up study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Genetic: 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.
Major functional disability (MFD)-free survival
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
Time frame: 15 years post-drug-product infusion
Number of participants with malignancies
Time frame: 15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
Time frame: 15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)
Time frame: 15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
Time frame: 15 years post-drug-product infusion
Number of participants with new or worsening neurologic disorders
Time frame: 15 years post-drug-product infusion
Number of participants who undergo subsequent stem cell transplantation
Time frame: 15 years post-drug-product infusion
Change from baseline in neurological function score (NFS)
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.
Time frame: 15 years post-drug-product infusion
Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
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.
Time frame: 15 years post-drug-product infusion
Plan to share: Yes — 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.
Started Jan 2016 (actual). Primary completion is estimated for Aug 2038, 271 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Genetix Biotherapeutics Inc.