TerminatedNot Applicable

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Stopped: Sufficient enrollment for Month 24 safety and efficacy endpoints

United States59 participantsStarted 2015-04

Plain-language 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.

Who can participate

Age range17 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.
✓. 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.
✓. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
✓. Depending on the cohort, the subject must:

Exclusion criteria

✕. Previous treatment with a gene therapy product.
✕. Receipt of an experimental transplantation procedure.

What they're measuring

Incidence of transplant-related mortality (TRM).

Timeframe: Through 100 and 365 days post allo-HSC infusion

Incidence and timing of neutrophil engraftment.

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence and timing of platelet engraftment

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence of engraftment failure or allograft rejection.

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence of primary donor-derived chimerism of ≥50%.

Timeframe: by 100 days post allo-HSC infusion

Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.

Timeframe: 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.

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Trial details

NCT IDNCT02204904

SponsorGenetix Biotherapeutics Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2019-12-06

View on ClinicalTrials.gov More X-Linked Adrenoleukodystrophy (X-ALD) trials

Who can participate

Age range17 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.
✓. 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.
✓. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
✓. Depending on the cohort, the subject must:

Exclusion criteria

✕. Previous treatment with a gene therapy product.
✕. Receipt of an experimental transplantation procedure.

What they're measuring

Incidence of transplant-related mortality (TRM).

Timeframe: Through 100 and 365 days post allo-HSC infusion

Incidence and timing of neutrophil engraftment.

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence and timing of platelet engraftment

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence of engraftment failure or allograft rejection.

Timeframe: 1-48 (± 1) months post allo-HSC infusion

Incidence of primary donor-derived chimerism of ≥50%.

Timeframe: by 100 days post allo-HSC infusion

Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.

Timeframe: 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.

Timeframe: 1-48 (± 1) months post allo-HSC infusion