Sufficient enrollment for Month 24 safety and efficacy endpoints
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
Sponsor study identifier
observational
59
7 countries
13
Study Start
April 2015
Actual (reported)Primary Completion
December 6, 2019
Actual (reported)Study Completion
December 6, 2019
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 6, 2019
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
July 28, 2014
Actual (reported)July 31, 2014
Actual (reported)April 2015
Actual (reported)December 6, 2019
Actual (reported)December 6, 2019
Actual (reported)Incidence of transplant-related mortality (TRM).
TRM is defined as death due to any transplantation-related cause other than disease progression.
Through 100 and 365 days post allo-HSC infusion
Incidence and timing of neutrophil engraftment.
1-48 (± 1) months post allo-HSC infusion
Incidence and timing of platelet engraftment
1-48 (± 1) months post allo-HSC infusion
Incidence of engraftment failure or allograft rejection.
1-48 (± 1) months post allo-HSC infusion
Incidence of primary donor-derived chimerism of ≥50%.
Incidence of Major Functional Disabilities (MFDs).
1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Loes score
1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Neurological Function Score (NFS)
1-48 (± 2) months post allo-HSC infusion
Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable
1-48 (± 2) months post allo-HSC infusion
MFD-free survival
48 (± 2) months post allo-HSC infusion
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.
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
Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection.
Allogeneic Hematopoietic Stem Cell Transplantation
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Stanford University
Palo Alto, California, 94304, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02141, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Hospital Austral
Chiesa R, Boelens JJ, Duncan CN, Kuhl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294.
Elizabeth McNeil, MD MSc
bluebird bio, Inc.
59 participants (actual enrollment reported by the registry).
First Submitted
July 28, 2014
First Posted
July 31, 2014
Study Start
April 2015
Actual (reported)Primary Completion
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.
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.
1-48 (± 1) months post allo-HSC infusion
Incidence of ≥Grade II acute GVHD.
1-48 (± 1) months post allo-HSC infusion
Incidence of chronic GVHD.
1-48 (± 1) months post allo-HSC infusion
Number of emergency room visits.
1-48 (± 1) months post allo-HSC infusion
Number and duration of intensive care unit stay.
1-48 (± 1) months post allo-HSC infusion
Number and duration of in-patient hospitalization.
1-48 (± 1) months post allo-HSC infusion
Contact the study team to confirm eligibility.
Buenos Aires, Argentina
McGill University Health Centre
Montreal, Quebec, H3H 2R9, Canada
University Hospital Leipzig
Leipzig, Germany
IRCCS Ospedale Pediatrico Bambine Gesú
Roma, 00165, Italy
Princess Maxima Center for Pediatric Oncology (PMC)
Utrecht, Netherlands
Great Ormond Street Hospital
London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
December 6, 2019
Actual (reported)Study Completion
December 6, 2019
Actual (reported)Last Updated
May 21, 2020
Record last verified: 2019-12
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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