A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy

TerminadoClinicalTrials.gov

ID: NCT02204904Tipo: OBSERVATIONALInicio: 1 de abr de 2015Fin estimado: 6 de dic de 2019

Traducción no disponible, mostrando original

Resumen

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.

Elegibilidad

Edad máxima: 17 YearsSexo: MALE

Criterios de inclusión

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:
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).

Criterios de exclusión

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

Intervenciones

genetic

Allo-HSCT

Allogeneic Hematopoietic Stem Cell Transplantation

Ubicaciones

Hospital Austral

Buenos Aires, Argentina

Patrocinadores

PrincipalGenetix Biotherapeutics Inc.

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