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

February 19, 2025 updated by: bluebird bio

Long-term Follow-up of Subjects 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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

64

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Caba, Argentina
        • Instituto Neurogenia
  • Australia
      • North Adelaide, Australia
        • Women's and Children's Hospital
  • Brazil
      • São Paulo, Brazil, 05403-000
        • Hospital das Clínicas da Universidade de São Paulo
  • France
    • Cedex
      • Le Kremlin-Bicêtre, Cedex, France, 94275
        • Hôpital Bicêtre
  • Germany
      • Leipzig, Germany, 04103
        • Universitätsklinikum Leipzig AöR
  • Italy
      • Rome, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù
  • Netherlands
      • Utrecht, Netherlands, 3584
        • Prinses Maxima Center
  • United Kingdom
      • London, United Kingdom
        • Great Ormond Street Hospital
    • England
      • London, England, United Kingdom, NW3 2QG
        • Royal Free London Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Mattel Children's Hospital-UCLA
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital - Stanford
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital/Massachusetts General Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 17 years (Child, Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study will be expected to participate in this long-term follow-up study.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Long-term followup
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).
Genetic: No 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
15 years post-drug-product infusion
Number of participants with malignancies
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion
Number of participants with new or worsening neurologic disorders
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants who undergo subsequent stem cell transplantation
Time Frame: 15 years post-drug-product infusion
15 years post-drug-product infusion
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.
15 years post-drug-product infusion
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.
15 years post-drug-product infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

bluebird bio

Investigators

  • Study Director: Vinod K Prasad, MD, FRCP, bluebird bio, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 22, 2016

Primary Completion (Estimated)

August 1, 2038

Study Completion (Estimated)

August 1, 2038

Study Registration Dates

First Submitted

February 17, 2016

First Submitted That Met QC Criteria

March 2, 2016

First Posted (Estimated)

March 3, 2016

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 19, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LTF-304
  • 2015-002805-13 (EudraCT Number)
  • 2024-513904-33-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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