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

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ClinicalTrials.gov Identifier: NCT02698579
Recruitment Status : Enrolling by invitation
First Posted : March 3, 2016
Last Update Posted : February 19, 2019
Sponsor:
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 a parent clinical study.

After completing a parent clinical study (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

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Study Type : Observational
Estimated Enrollment : 50 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 : March 2036
Estimated Study Completion Date : March 2036


Group/Cohort Intervention/treatment
Subjects treated with Lenti-D
Subjects 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.
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. Proportion of subjects who experience graft versus host disease (GVHD) [ Time Frame: 15 years post-drug-product infusion ]
  2. Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) [ Time Frame: 15 years post-drug-product infusion ]
  3. All drug product-related AEs [ Time Frame: 15 years post-drug-product infusion ]
  4. All SAEs (regardless of relatedness to drug product) [ Time Frame: 15 years post-drug-product infusion ]
  5. Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. [ Time Frame: 5 years post-drug-product infusion ]
  6. Monitoring for clonal dominance using integration site analysis (ISA) [ Time Frame: 15 years post-drug-product infusion ]
  7. Monitoring for clinical signs of oncogenesis [ Time Frame: 15 years post-drug-product infusion ]
  8. MFD-free survival [ Time Frame: 15 years post-drug-product infusion ]

Secondary Outcome Measures :
  1. Overall Survival of subjects with cerebral adrenoleukodystrophy (CALD) treated with Lenti-D [ 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. Change in gadolinium enhancement (GdE) status from last MRI performed in parent study. [ 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 a parent clinical study.
Criteria

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 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
Algeria
University Hospital Ben Badis
Constantine, Algeria
Argentina
Medeos SRL
Buenos Aires, Argentina
Australia
Women's and Children's Hospital
North Adelaide, Australia
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: Elizabeth McNeil, MD bluebird bio, Inc.

Additional Information:
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Responsible Party: bluebird bio
ClinicalTrials.gov Identifier: NCT02698579     History of Changes
Other Study ID Numbers: LTF-304
First Posted: March 3, 2016    Key Record Dates
Last Update Posted: February 19, 2019
Last Verified: February 2019

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