X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Conditions
Keywords
X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
Brief summary
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
Interventions
GENETICAllo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation
Sponsors
Genetix Biotherapeutics Inc.
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
MALE
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
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).
Exclusion criteria
1. Previous treatment with a gene therapy product. 2. Receipt of an experimental transplantation procedure.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number and duration of in-patient hospitalization. | 1-48 (± 1) months post allo-HSC infusion | — |
| Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs. | 1-48 (± 1) months post allo-HSC infusion | — |
| Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD. | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence of ≥Grade II acute GVHD. | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence of chronic GVHD. | 1-48 (± 1) months post allo-HSC infusion | — |
| Number of emergency room visits. | 1-48 (± 1) months post allo-HSC infusion | — |
| Number and duration of intensive care unit stay. | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence and timing of platelet engraftment | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence of transplant-related mortality (TRM). | Through 100 and 365 days post allo-HSC infusion | TRM is defined as death due to any transplantation-related cause other than disease progression. |
| Incidence and timing of neutrophil engraftment. | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence of engraftment failure or allograft rejection. | 1-48 (± 1) months post allo-HSC infusion | — |
| Incidence of primary donor-derived chimerism of ≥50%. | by 100 days post allo-HSC infusion | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Loes score | 1-48 (± 2) months post allo-HSC infusion | — |
| Change from Baseline in Neurological Function Score (NFS) | 1-48 (± 2) months post allo-HSC infusion | — |
| Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable | 1-48 (± 2) months post allo-HSC infusion | — |
| MFD-free survival | 48 (± 2) months post allo-HSC infusion | — |
| Overall survival | 48 (± 2) months post allo-HSC infusion | — |
| Incidence of Major Functional Disabilities (MFDs). | 1-48 (± 2) months post allo-HSC infusion | MFDs is defined as any of the following: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. |
Countries
Argentina, Canada, Germany, Italy, Netherlands, United Kingdom, United States
Outcome results
None listed