Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Genetix Biotherapeutics Inc.
Age
0–17
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
1. Provide informed consent from a competent custodial parent 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 institution's IRB/IEC and all other local requirements.
2. Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.
3. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
4. Depending on the cohort, the subject must:
* Be scheduled for allo-HSCT evaluation at a study site (prospective cohort only),
* Have received an allo-HSC infusion and be consented in time to complete the Month 24 Visit on study (partial prospective/retrospective cohort only), or
* Have received their most recent allo-HSC infusion on or after January 1, 2013 (retrospective cohort only).
You may not be if
1. Previous treatment with a gene therapy product.
2. Receipt of an experimental transplantation procedure.
