Trial record 1 of 1 for:    ltf-304
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Long-term Follow-up of Subjects 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. Identifier: NCT02698579
Recruitment Status : Enrolling by invitation
First Posted : March 3, 2016
Last Update Posted : February 2, 2018
Information provided by (Responsible Party):
bluebird bio

Brief Summary:

This is a multi-center, long-term safety and efficacy follow-up study for subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in Study ALD-102.

After completing Study ALD-102 (approximately 2 years), eligible subjects 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) Other: Safety and efficacy assessments

Study Type : Observational
Estimated Enrollment : 25 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
Study Start Date : January 2016
Estimated Primary Completion Date : November 2033
Estimated Study Completion Date : November 2033

Group/Cohort Intervention/treatment
Subjects treated with Lenti-D
Subjects who have received Lenti-D Drug Product in Study ALD-102 (bluebird bio-sponsored clinical trial) and who meet the eligibility criteria for the study LTF-304
Other: Safety and efficacy assessments
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Primary Outcome Measures :
  1. Overall survival of subjects with cerebral adrenoleukodystrophy (CALD) treated with Lenti-D Drug Product [ Time Frame: 15 years post-drug-product infusion ]
  2. Proportion of subjects who experience new, persistent or worsening chronic GVHD [ Time Frame: 15 years post-drug-product infusion ]
  3. Proportion of subjects who undergo subsequent stem cell transplantation [ Time Frame: 15 years post-drug-product infusion ]
  4. All drug product-related AEs [ Time Frame: 15 years post-drug-product infusion ]
  5. All SAEs (regardless of relatedness to drug product) [ Time Frame: 15 years post-drug-product infusion ]
  6. Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. [ Time Frame: 5 years post-drug-product infusion ]
  7. Monitoring for clonal dominance using integration site analysis (ISA) [ Time Frame: 15 years post-drug-product infusion ]
  8. Monitoring for clinical signs of oncogenesis [ Time Frame: 15 years post-drug-product infusion ]
  9. MFD-free survival [ Time Frame: 15 years post-drug-product infusion ]

Secondary Outcome Measures :
  1. Change from baseline in Loes score [ 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 ]
  3. Monitoring of status of gadolinium enhancement (through MRI) from entry into Study LTF-304 [ Time Frame: 15 years post-drug-product infusion ]

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
Subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in Study ALD-102.

Inclusion Criteria:

  • Provision of written informed consent for this study by the subject or subject's parent(s)/ legal guardian(s) and written informed assent by subject, if applicable
  • Have received Lenti-D Drug Product in Study ALD-102
  • Able to comply with study requirements.

Exclusion Criteria:

  • Met the VCN discontinuation criterion from Study ALD-102, as follows: the subject has undetectable VCN (<0.0003 copies per cell) in peripheral blood cells for 2 consecutive measurements at least 1 month apart and at least 12 months after drug product infusion. This exclusion criterion does not apply to subjects who received allo-HSCT after Lenti-D Drug Product infusion in Study ALD-102.

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 identifier (NCT number): NCT02698579

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
Fundación Investigar
Buenos Aires, Argentina
Women's and Children's Hospital
North Adelaide, Australia
Hôpital Bicêtre
Le Kremlin-Bicêtre, Cedex, France, 94275
United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sponsors and Collaborators
bluebird bio
Study Director: Mohammed Asmal, MD, PhD bluebird bio, Inc.

Additional Information:
Responsible Party: bluebird bio Identifier: NCT02698579     History of Changes
Other Study ID Numbers: LTF-304
First Posted: March 3, 2016    Key Record Dates
Last Update Posted: February 2, 2018
Last Verified: December 2017

Keywords provided by bluebird bio:
X-linked Adrenoleukodystrophy
Hematopoietic Stem Cells

Additional relevant MeSH terms:
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