Adrenoleukodystrophy (ALD), Cerebral Adrenoleukodystrophy (CALD), X-Linked Adrenoleukodystrophy (X-ALD)
Conditions
Keywords
Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
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 (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.
Interventions
GENETICNo 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.
Sponsors
Genetix Biotherapeutics Inc.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
No minimum to 19 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Major functional disability (MFD)-free survival | 15 years post-drug-product infusion | The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement. |
| Number of participants with malignancies | 15 years post-drug-product infusion | — |
| Number of participants who experience graft versus host disease (GVHD) | 15 years post-drug-product infusion | — |
| Number of participants with immune-related adverse events (AEs) | 15 years post-drug-product infusion | — |
| Number of participants with new or worsening hematologic disorders | 15 years post-drug-product infusion | — |
| Number of participants with new or worsening neurologic disorders | 15 years post-drug-product infusion | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants who undergo subsequent stem cell transplantation | 15 years post-drug-product infusion | — |
| Change from baseline in neurological function score (NFS) | 15 years post-drug-product infusion | 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. |
| Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI) | 15 years post-drug-product infusion | 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. |
Countries
Argentina, Australia, Brazil, France, Germany, Italy, Netherlands, United Kingdom, United States
Contacts
STUDY_DIRECTORVinod K Prasad, MD, FRCP
Genetix Biotherapeutics Inc.
Outcome results
None listed