Disorders of fatty-acid metabolism
Conditions
Interventions
A group of approximately 50 subjects who received Lenti-D medication in a parent study that was not conducted in Brazil. Current study will last 13 years with visits every 6 months during the first 3
Other
E71.3
Sponsors
Blanchard y Asociados
HCFMUSP – Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
Eligibility
Sex/Gender
Male
Age
0 Years to 19 Years
Inclusion 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
Exclusion criteria: There are no exclusion criteria for this Study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluate proportion of subjects who experience graft versus host disease (GVHD); proportion of subjects who undergo subsequent stem cell transplantation; all drug product-related Adverse Events through 15 years post-drug product infusion; all serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product), including any new malignancy or new diagnosis of a neurologic, rheumatologic, or hematologic disorder that, in the Investigator’s opinion, is clinically significant and requires medical intervention; SAEs related to drug product would be collected through 15 years post-drug product infusion; incidence of vector-derived Replication Competent Lentivirus assessed from archived samples as clinically indicated; characterization of events of insertional mutagenesis leading to clonal dominance or leukemia; laboratory tests as specified. In addition, exposure to cytotoxic agents, radiotherapy, and other potentially mutagenic agents will be documented. The primary efficacy endpoint is evaluated by Major functional disability (MFD)-free survival | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary efficacy endpoints include evaluation of overall survival, of change from Baseline (defined in parent study) in neurologic function score (NFS) and of change in gadolinium enhancement (GdE) status from last magnetic resonance imaging (MRI) performed in parent study. | — |
Countries
Argentina, Australia, Brazil, France, United Kingdom, United States
Contacts
Public ContactElizabeth McNeil
bluebird bio, Inc
Outcome results
None listed