Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
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ClinicalTrials.gov Identifier: NCT02204904 |
Recruitment Status :
Terminated
(Sufficient enrollment for Month 24 safety and efficacy endpoints)
First Posted : July 31, 2014
Last Update Posted : May 21, 2020
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Condition or disease | Intervention/treatment |
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X-Linked Adrenoleukodystrophy (X-ALD) Cerebral Adrenoleukodystrophy (CALD) Adrenoleukodystrophy (ALD) | Genetic: Allo-HSCT |
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 |

Group/Cohort | Intervention/treatment |
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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.
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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
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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.
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Genetic: Allo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation |
- 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.
- Incidence and timing of neutrophil engraftment. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Incidence and timing of platelet engraftment [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Incidence of engraftment failure or allograft rejection. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Incidence of primary donor-derived chimerism of ≥50%. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Incidence of ≥Grade II acute GVHD. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Incidence of chronic GVHD. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Number of emergency room visits. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Number and duration of intensive care unit stay. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- Number and duration of in-patient hospitalization. [ Time Frame: 1-48 (± 1) months post allo-HSC infusion ]
- 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.
- Change from Baseline in Loes score [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
- Change from Baseline in Neurological Function Score (NFS) [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
- Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable [ Time Frame: 1-48 (± 2) months post allo-HSC infusion ]
- MFD-free survival [ Time Frame: 48 (± 2) months post allo-HSC infusion ]
- Overall survival [ Time Frame: 48 (± 2) months post allo-HSC infusion ]

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

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
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 |
Study Director: | Elizabeth McNeil, MD MSc | bluebird bio, Inc. |
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: | May 21, 2020 |
Last Verified: | December 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
X-linked Adrenoleukodystrophy Hematopoietic Stem Cells |
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 |