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

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X-linked adrenoleukodystrophy
Related Access Program
University of Minnesota – Clinical and Translational Science Institute – X-linked Adrenoleukodystrophy
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Brief Title

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

Official Title

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

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.

Study Type

Observational

Primary Outcome

Incidence of transplant-related mortality (TRM).

Secondary Outcome

 Incidence of Major Functional Disabilities (MFDs).

Condition

X-Linked Adrenoleukodystrophy (X-ALD)

Intervention

Allo-HSCT

Study Arms / Comparison Groups

 Allo-HSCT prospective
Description:  Subjects who will be consented before they received an allo-HSC infusion. They will be consented and enrolled on the study during the Screening Period.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Genetic

Estimated Enrollment

59

Start Date

April 2015

Completion Date

December 6, 2019

Primary Completion Date

December 6, 2019

Eligibility Criteria

        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.

Gender

Male

Ages

N/A - 17 Years

Accepts Healthy Volunteers

No

Contacts

Elizabeth McNeil, MD MSc, ,

Location Countries

Argentina

Location Countries

Argentina

Administrative Informations

NCT ID

NCT02204904

Organization ID

ALD-103

Responsible Party

Sponsor

Study Sponsor

bluebird bio

Study Sponsor

Elizabeth McNeil, MD MSc, Study Director, bluebird bio, Inc.

Verification Date

December 2019