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

Active, not recruiting

• Conditions: Adrenoleukodystrophy (ALD); Cerebral Adrenoleukodystrophy (CALD); X-Linked Adrenoleukodystrophy (X-ALD)
• Interventions: Genetic: No interventional drug product utilized in this follow-up study

• Registration Number: NCT02698579
• Lead Sponsor: bluebird bio

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-01-22
• Study First Submitted: 2016-02-17
• Study First Submitted QC: 2016-03-02
• Study First Posted: 2016-03-03
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-15
• Last Update Posted: 2024-08-19
• Primary Completion: 2038-08
• Study Completion: 2038-08

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 64

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

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Exclusion Criteria

• There are no exclusion criteria for this study

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Long-term followup	No interventional drug product utilized in this follow-up study	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).

Primary Outcome Measures

Name	Time	Method
Number of participants with new or worsening neurologic disorders	15 years post-drug-product infusion
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 new or worsening hematologic disorders	15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)	15 years post-drug-product infusion

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (13)

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

Women's and Children's Hospital; 🇦🇺 North Adelaide, Australia

Hospital das Clínicas da Universidade de São Paulo; 🇧🇷 São Paulo, Brazil

Ospedale Pediatrico Bambino Gesù; 🇮🇹 Rome, Italy

Hôpital Bicêtre; 🇫🇷 Le Kremlin-Bicêtre, Cedex, France

Prinses Maxima Center; 🇳🇱 Utrecht, Netherlands

Universitätsklinikum Leipzig AöR; 🇩🇪 Leipzig, Germany

Royal Free London Hospital; 🇬🇧 London, England, United Kingdom

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Instituto Neurogenia; 🇦🇷 Caba, Argentina