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NCT Number: NCT02698579

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

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.

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Key information

Conditions

Cerebral Adrenoleukodystrophy (cALD) Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Adrenoleukodystrophy (ALD) Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Peroxisomal Disorders X-Linked Adrenoleukodystrophy (X-ALD) X-Linked Intellectual Disability

Age range

Up to 19 year

Sex eligibility

Male

Study type

Observational

Primary location

Instituto Neurogenia, Caba, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No interventional drug product utilized in this follow-up study

Genetic

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.

Primary outcomes

  1. Major functional disability (MFD)-free survival

    Time frame: 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.

  2. Number of participants with malignancies

    Time frame: 15 years post-drug-product infusion

  3. Number of participants who experience graft versus host disease (GVHD)

    Time frame: 15 years post-drug-product infusion

  4. Number of participants with immune-related adverse events (AEs)

    Time frame: 15 years post-drug-product infusion

  5. Number of participants with new or worsening hematologic disorders

    Time frame: 15 years post-drug-product infusion

  6. Number of participants with new or worsening neurologic disorders

    Time frame: 15 years post-drug-product infusion

Secondary outcomes

  1. Number of participants who undergo subsequent stem cell transplantation

    Time frame: 15 years post-drug-product infusion

  2. Change from baseline in neurological function score (NFS)

    Time frame: 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.

  3. Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Genetix Biotherapeutics Inc.

Industry

Registry information

Official study title

Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Important dates

Study start
2016
Primary completion
2038
Study completion
2038
First posted
Mar 3, 2016
Registry last updated
Feb 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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