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.
Study Type
OBSERVATIONAL
Enrollment
64
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.
Mattel Children's Hospital-UCLA
Los Angeles, California, United States
Lucile Packard Children's Hospital - Stanford
Palo Alto, California, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Instituto Neurogenia
Caba, Argentina
Women's and Children's Hospital
North Adelaide, Australia
Hospital das Clínicas da Universidade de São Paulo
São Paulo, Brazil
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, France
Universitätsklinikum Leipzig AöR
Leipzig, Germany
Ospedale Pediatrico Bambino Gesù
Rome, Italy
...and 3 more locations
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
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