Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Study Details
Study Description
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 | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | 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:
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Outcome Measures
Primary Outcome Measures
- Major Functional Disability (MFD)-Free Survival [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) [15 years post-drug-product infusion]
- Percentage of Participants who Undergo Subsequent Stem Cell Transplantation (i.e. Second Hematopoietic Stem Cell [HSC] Infusion) [15 years post-drug-product infusion]
- Number of Participants with Drug Product-Related Adverse Events (AEs) [15 years post-drug-product infusion]
- Number of Participants with Serious Adverse Events (SAEs) (Regardless of Relatedness to Drug Product) [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 [15 years post-drug-product infusion]
- Incidence of Vector-derived Replication Competent Lentivirus (RCL), Assessed from Archived Samples as Clinically Indicated [5 years post-drug-product infusion]
- Number of Participants with Insertional Oncogenesis [15 years post-drug-product infusion]
Insertional oncogenesis including Myelodysplasia, Leukemia, Lymphoma will be reported.
- Number of Participants with Clonal Predominance [15 years post-drug-product infusion]
Secondary Outcome Measures
- Overall Survival [15 years post-drug-product infusion]
- Change from Baseline in Neurological Function Score (NFS) [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 [15 years post-drug-product infusion]
GdE status means participants who will report GdE negative (-) or positive (+).
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Mattel Children's Hospital-UCLA | Los Angeles | California | United States | 90095 |
| 2 | Boston Children's Hospital/Massachusetts General Hospital | Boston | Massachusetts | United States | 02115 |
| 3 | University of Minnesota | Minneapolis | Minnesota | United States | 55455 |
| 4 | Instituto Neurogenia | Caba | Argentina | ||
| 5 | Women's and Children's Hospital | North Adelaide | Australia | ||
| 6 | Hospital das Clínicas da Universidade de São Paulo | São Paulo | Brazil | 05403-000 | |
| 7 | Hôpital Bicêtre | Le Kremlin-Bicêtre | Cedex | France | 94275 |
| 8 | Universitätsklinikum Leipzig AöR | Leipzig | Germany | 04103 | |
| 9 | Prinses Maxima Center | Utrecht | Netherlands | 3584 | |
| 10 | Great Ormond Street Hospital | London | United Kingdom |
Sponsors and Collaborators
- bluebird bio
Investigators
- Study Director: Jakob Sieker, MD, bluebird bio, Inc.
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- LTF-304
- 2015-002805-13