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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02698579
Recruitment Status : Enrolling by invitation
First Posted : March 3, 2016
Last Update Posted : March 30, 2023
Information provided by (Responsible Party):
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.

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.

Condition or disease Intervention/treatment
Cerebral Adrenoleukodystrophy (CALD) Adrenoleukodystrophy (ALD) X-Linked Adrenoleukodystrophy (X-ALD) Genetic: Lenti-D Drug Product

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Study Type : Observational
Estimated Enrollment : 67 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Actual Study Start Date : January 22, 2016
Estimated Primary Completion Date : May 2037
Estimated Study Completion Date : May 2037

Group/Cohort Intervention/treatment
Lenti-D Drug Product
Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical trial) and who meet the eligibility criteria for the study LTF-304 will be followed in this observation study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent study).
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.
Other Names:
  • elivaldogene autotemcel
  • eli-cel

Primary Outcome Measures :
  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 Outcome Measures :
  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 ]
    GdE status means participants who will report GdE negative (-) or positive (+).

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   Male
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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02698579

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United States, California
Mattel Children's Hospital-UCLA
Los Angeles, California, United States, 90095
Lucile Packard Children's Hospital - Stanford
Palo Alto, California, United States, 94304
United States, Massachusetts
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Instituto Neurogenia
Caba, Argentina
Women's and Children's Hospital
North Adelaide, Australia
Hospital das Clínicas da Universidade de São Paulo
São Paulo, Brazil, 05403-000
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, France, 94275
Universitätsklinikum Leipzig AöR
Leipzig, Germany, 04103
Prinses Maxima Center
Utrecht, Netherlands, 3584
United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sponsors and Collaborators
bluebird bio
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Study Director: Himal Lal Thakar, MD bluebird bio, Inc.
Additional Information:
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Responsible Party: bluebird bio
ClinicalTrials.gov Identifier: NCT02698579    
Other Study ID Numbers: LTF-304
2015-002805-13 ( EudraCT Number )
First Posted: March 3, 2016    Key Record Dates
Last Update Posted: March 30, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Bluebird bio is committed to transparency and 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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by bluebird bio:
X-linked Adrenoleukodystrophy
Hematopoietic Stem Cells
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases