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

Cerebral Adrenoleukodystrophy (CALD) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02698579
• Other study ID #: LTF-304
• Secondary ID: 2015-002805-13
• Status: Active, not recruiting
• Start date: January 22, 2016
• Est. completion date: August 2038

Study information

• Verified date: February 2024
• Source: bluebird bio
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 64
• Est. completion date: August 2038
• Est. primary completion date: August 2038
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenoleukodystrophy (ALD)
• Cerebral Adrenoleukodystrophy (CALD)
• X-Linked Adrenoleukodystrophy (X-ALD)

Intervention

Genetic:
• Lenti-D Drug Product
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant.

Locations

Country	Name	City	State
Argentina	Instituto Neurogenia	Caba
Australia	Women's and Children's Hospital	North Adelaide
Brazil	Hospital das Clínicas da Universidade de São Paulo	São Paulo
France	Hôpital Bicêtre	Le Kremlin-Bicêtre	Cedex
Germany	Universitätsklinikum Leipzig AöR	Leipzig
Italy	Ospedale Pediatrico Bambino Gesù	Rome
Netherlands	Prinses Maxima Center	Utrecht
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Royal Free London Hospital	London	England
United States	Boston Children's Hospital/Massachusetts General Hospital	Boston	Massachusetts
United States	Mattel Children's Hospital-UCLA	Los Angeles	California
United States	University of Minnesota	Minneapolis	Minnesota
United States	Lucile Packard Children's Hospital - Stanford	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
bluebird bio

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Brazil,  France,  Germany,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	15 years post-drug-product infusion
Primary	Number of participants with malignancies		15 years post-drug-product infusion
Primary	Number of participants who experience graft versus host disease (GVHD)		15 years post-drug-product infusion
Primary	Number of participants with immune-related adverse events (AEs)		15 years post-drug-product infusion
Primary	Number of participants with new or worsening hematologic disorders		15 years post-drug-product infusion
Primary	Number of participants with new or worsening neurologic disorders		15 years post-drug-product infusion
Secondary	Number of participants who undergo subsequent stem cell transplantation		15 years post-drug-product infusion
Secondary	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.	15 years post-drug-product infusion
Secondary	Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)	GdE status means participants who will report GdE negative (-) or positive (+).	15 years post-drug-product infusion

External Links

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