Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02204904
Recruitment Status : Recruiting
First Posted : July 31, 2014
Last Update Posted : July 19, 2019
Information provided by (Responsible Party):
bluebird bio

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

Layout table for study information
Study Type : Observational
Estimated Enrollment : 60 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
Estimated Primary Completion Date : May 2021
Estimated Study Completion Date : May 2021

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

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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
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

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.

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 identifier (NCT number): NCT02204904

Layout table for location contacts
Contact: bluebird bio 339-499-9300

Layout table for location information
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Principal Investigator: Neena Kapoor, MD         
Stanford University Recruiting
Palo Alto, California, United States, 94304
Principal Investigator: Ami Shah, MD         
United States, Massachusetts
Boston Children's Hospital/Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02141
Principal Investigator: Christine Duncan, MD         
Principal Investigator: Florian S Eichler, MD         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Principal Investigator: Paul Orchard, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Principal Investigator: Vinod Prasad, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Nancy Bunin, MD         
Principal Investigator: Nancy Bunin, MD         
United States, Utah
University of Utah School of Medicine Withdrawn
Salt Lake City, Utah, United States, 84108
Hospital Austral Recruiting
Buenos Aires, Argentina
Principal Investigator: Hernan Amartino, MD         
Canada, Quebec
McGill University Health Centre Recruiting
Montréal, Quebec, Canada, H3H 2R9
Contact: Genevieve Bernard, MD         
Principal Investigator: Genevieve Bernard, MD         
University Hospital Leipzig Recruiting
Leipzig, Germany
Principal Investigator: Jörn-Sven Kühl , MD         
IRCCS Ospedale Pediatrico Bambine Gesú Recruiting
Roma, Italy, 00165
Contact: Francesco Locatelli, MD         
Principal Investigator: Francesco Locatelli, MD         
Princess Maxima Center for Pediatric Oncology (PMC) Recruiting
Utrecht, Netherlands
Principal Investigator: Caroline Lindemans, MD         
United Kingdom
Great Ormond Street Hospital Recruiting
London, United Kingdom
Principal Investigator: Robert Chiesa, MD         
Central Manchester University Hospitals NHS Foundation Trust Recruiting
Manchester, United Kingdom, M13 9WL
Principal Investigator: Simon Jones, MD         
Sponsors and Collaborators
bluebird bio
Layout table for investigator information
Study Director: Elizabeth McNeil, MD MSc bluebird bio, Inc.

Layout table for additonal information
Responsible Party: bluebird bio Identifier: NCT02204904     History of Changes
Other Study ID Numbers: ALD-103
First Posted: July 31, 2014    Key Record Dates
Last Update Posted: July 19, 2019
Last Verified: July 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:
Layout table for MeSH terms
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
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
Molecular Mechanisms of Pharmacological Action
Enzyme Inhibitors
Gout Suppressants
Antirheumatic Agents
Free Radical Scavengers