Adrenoleukodystrophy bronze Schilder disease
Conditions
Interventions
None listed
Sponsors
bluebird bio
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: Subjects must: 1. Provide informed consent from a competent custodial parents or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements. 2. Be male and
Exclusion criteria
Exclusion criteria: Subjects are excluded if they meet any of the following criteria. 1. Previous treatment with a gene therapy product. 2. Receipt of an experimental transplant procedure.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety Endpoints Assessed for all allo-HSC infusions (i.e. including successful and failed all-HSCTs) • Incidence of transplant-related mortality (TRM) as defined as death due to any trans-plantation-related cause other than disease relapse, through 100 and 365 days post-allo-HSC infusion • Incidence and timing of neutrophil engraftment • Incidence and timing of platelet engraftment • Incidence of engraftment failure or allograft rejection • Incidence of primary donor-derived chimerism of >=50% by 100 days post-allo-HSC infusion • Frequency and severity of National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) >=Grade 3 AEs, CTCAE >=Grade 3 infections, and all SAEs (Note: non-clinically significant laboratory AEs [i.e. do not require additional medical intervention] and hematological toxicities related to conditioning medication for first 30 days after any allo-HSC infusion will NOT be collected) • Proportion of subjects who experience either >=Grade II acute GVHD or chronic GVHD (Note: acute GVHD graded on the Acute GVHD Grading Scale (I-IV); chronic GVHD as assessed by the Investigator) • incidence of >=Grade 2 acute GVHD • incidence of chronic GVHD Efficacy Endpoints: Assessed from baseline up to and including the M24 and M48 Visits following the most recent allo-HSC infusion (i.e. from successful allo-HSCT) • Incidence of Major Functional Disabilities (MFDs); (defined as any of the following: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement) • Change from Baseline in Loes score • Change from Baseline in NFS • Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable • MFD-free survival • Overall survival | — |
Secondary
| Measure | Time frame |
|---|---|
| Exploratory Endpoints: Assessed from Baseline up to and including the M24 and the M48 Visits of the most recent allo-HSC infusion, and up to day of engraftment failure or allograft rejection for subjects with failed allo-HSCT: • IQ using the age-appropriate Wechsler test • VLCFA levels in fasting serum • Matrix metalloproteinases in cerebrospinal fluid, • chitotriosidase in plasma and cerebrospinal fluid, • overall QL score (PedsQL and UMN QL), including 30, 60 and 100 days post-allo-HSC infusion, and at the M6, M12, M24, M36 and M48 Visits | — |
Countries
Netherlands
Outcome results
None listed