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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 : January 19, 2021
Sponsor:
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 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

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Study Type : Observational
Estimated Enrollment : 60 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
Participants treated with Lenti-D
Partcipants 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.
Genetic: Lenti-D
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. Percentage of Participants who Experience Graft Versus Host Disease (GVHD) [ Time Frame: 15 years post-drug-product infusion ]
  3. Percentage of Participants who Undergo Subsequent Stem Cell Transplantation (i.e. Second Hematopoietic Stem Cell [HSC] Infusion) [ Time Frame: 15 years post-drug-product infusion ]
  4. Number of Participants with Drug Product-Related Adverse Events (AEs) [ Time Frame: 15 years post-drug-product infusion ]
  5. Number of Participants with Serious Adverse Events (SAEs) (Regardless of Relatedness to Drug Product) [ Time Frame: 15 years post-drug-product infusion ]
  6. Number of Participants with Serious or non-serious Immune-related Adverse Events (AEs) and New or Worsening Hematologic or Neurologic Disorders or Malignancies [ Time Frame: 15 years post-drug-product infusion ]
  7. Incidence of Vector-derived Replication Competent Lentivirus (RCL), Assessed from Archived Samples as Clinically Indicated [ Time Frame: 5 years post-drug-product infusion ]
  8. Number of Participants with Insertional Oncogenesis [ Time Frame: 15 years post-drug-product infusion ]
    Insertional oncogenesis including Myelodysplasia, Leukemia, Lymphoma will be reported.

  9. Number of Participants with Clonal Predominance [ Time Frame: 15 years post-drug-product infusion ]

Secondary Outcome Measures :
  1. Overall Survival [ 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. Percentage of Participants with Gadolinium Enhancement (GdE) Status [ 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

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Ages Eligible for Study:   Child, Adult, Older Adult
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.
Criteria

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 Lenti-D Drug Product in a parent clinical study
  • Able to comply with study requirements

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


Locations
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United States, California
Mattel Children's Hospital-UCLA
Los Angeles, California, United States, 90095
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
Argentina
Medeos SRL
Buenos Aires, Argentina
Australia
Women's and Children's Hospital
North Adelaide, Australia
Brazil
Hospital das Clínicas da Universidade de São Paulo
São Paulo, Brazil, 05403-000
France
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, France, 94275
United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sponsors and Collaborators
bluebird bio
Investigators
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Study Director: Andrew Dietz, 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: January 19, 2021
Last Verified: January 2021

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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:
Adrenoleukodystrophy
X-linked Adrenoleukodystrophy
Hematopoietic Stem Cells
Additional relevant MeSH terms:
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Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
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