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

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ClinicalTrials.gov Identifier: NCT02204904
Recruitment Status : Completed
First Posted : July 31, 2014
Last Update Posted : December 24, 2019
Sponsor:
Information provided by (Responsible Party):
bluebird bio

Study Description
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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.

Condition or disease Intervention/treatment
X-Linked Adrenoleukodystrophy (X-ALD) Cerebral Adrenoleukodystrophy (CALD) Adrenoleukodystrophy (ALD) Genetic: Allo-HSCT

Study Design
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Study Type : Observational
Actual Enrollment : 59 participants
Observational Model: Cohort
Time Perspective: Other
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
Actual Study Start Date : April 2015
Actual Primary Completion Date : December 6, 2019
Actual Study Completion Date : December 6, 2019

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Allo-HSCT prospective
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.
 Genetic: Allo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation
Allo-HSCT partial prospective/retrospective
Subjects who will be consented after they received an allo-HSC infusion but before they reach 24 months post-infusion on study. Subjects in this cohort will participate prospectively in at least the Month 24 Visit in order to obtain prospective on-study data for this and all visits after Month 24
 Genetic: Allo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation
Allo-HSCT retrospective
Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection.
 Genetic: Allo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of transplant-related mortality (TRM). [ Time Frame: Through 100 and 365 days post allo-HSC infusion ]
    TRM is defined as death due to any transplantation-related cause other than disease progression.

  2. Incidence and timing of neutrophil engraftment. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  3. Incidence and timing of platelet engraftment [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  4. Incidence of engraftment failure or allograft rejection. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  5. Incidence of primary donor-derived chimerism of ≥50%. [ Time Frame: by 100 days post allo-HSC infusion ]
  6. Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  7. Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  8. Incidence of ≥Grade II acute GVHD. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  9. Incidence of chronic GVHD. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  10. Number of emergency room visits. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  11. Number and duration of intensive care unit stay. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
  12. Number and duration of in-patient hospitalization. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]

Secondary Outcome Measures :
  1. Incidence of Major Functional Disabilities (MFDs). [ Time Frame: 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.

  2. Change from Baseline in Loes score [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
  3. Change from Baseline in Neurological Function Score (NFS) [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
  4. Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
  5. MFD-free survival [ Time Frame: 48 (± 2) months post allo-HSC infusion ]
  6. Overall survival [ Time Frame: 48 (± 2) months post allo-HSC infusion ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02204904


Locations
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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Stanford University
Palo Alto, California, United States, 94304
United States, Massachusetts
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States, 02141
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Argentina
Hospital Austral
Buenos Aires, Argentina
Canada, Quebec
McGill University Health Centre
Montréal, Quebec, Canada, H3H 2R9
Germany
University Hospital Leipzig
Leipzig, Germany
Italy
IRCCS Ospedale Pediatrico Bambine Gesú
Roma, Italy, 00165
Netherlands
Princess Maxima Center for Pediatric Oncology (PMC)
Utrecht, Netherlands
United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
bluebird bio
Investigators
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Study Director: Elizabeth McNeil, MD MSc bluebird bio, Inc.
More Information
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Responsible Party: bluebird bio
ClinicalTrials.gov Identifier: NCT02204904    
Other Study ID Numbers: ALD-103
First Posted: July 31, 2014    Key Record Dates
Last Update Posted: December 24, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Keywords provided by bluebird bio:
X-linked Adrenoleukodystrophy
Hematopoietic Stem Cells
Additional relevant MeSH terms:
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Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases