Long-term Follow-up of Participants 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 : August 12, 2022
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|
|Study Type :||Observational|
|Estimated Enrollment :||60 participants|
|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|
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.
Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant.
- 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.
- Percentage of Participants who Experience Graft Versus Host Disease (GVHD) [ Time Frame: 15 years post-drug-product infusion ]
- 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 ]
- Number of Participants with Drug Product-Related Adverse Events (AEs) [ Time Frame: 15 years post-drug-product infusion ]
- Number of Participants with Serious Adverse Events (SAEs) (Regardless of Relatedness to Drug Product) [ Time Frame: 15 years post-drug-product infusion ]
- 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 ]
- Incidence of Vector-derived Replication Competent Lentivirus (RCL), Assessed from Archived Samples as Clinically Indicated [ Time Frame: 5 years post-drug-product infusion ]
- Number of Participants with Insertional Oncogenesis [ Time Frame: 15 years post-drug-product infusion ]Insertional oncogenesis including Myelodysplasia, Leukemia, Lymphoma will be reported.
- Number of Participants with Clonal Predominance [ Time Frame: 15 years post-drug-product infusion ]
- Overall Survival [ Time Frame: 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.
- 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 (+).
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
|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|
|Women's and Children's Hospital|
|North Adelaide, Australia|
|Hospital das Clínicas da Universidade de São Paulo|
|São Paulo, Brazil, 05403-000|
|Le Kremlin-Bicêtre, Cedex, France, 94275|
|Universitätsklinikum Leipzig AöR|
|Leipzig, Germany, 04103|
|Prinses Maxima Center|
|Utrecht, Netherlands, 3584|
|Great Ormond Street Hospital|
|London, United Kingdom|
|Study Director:||Jakob Sieker, MD||bluebird bio, Inc.|