An observational study in X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD) and Adrenoleukodystrophy (ALD), sponsored by bluebird bio. Terminated at 13 sites in 7 countries. Open to male participants aged Up to 17 Years. Per ClinicalTrials.gov, last updated 2020-05-21.
Sponsored by bluebird bio · Observational
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 59 is below the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →bluebird bio is the lead sponsor of 12 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Depending on the cohort, the subject must:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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
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
Incidence of transplant-related mortality (TRM).
TRM is defined as death due to any transplantation-related cause other than disease progression.
Time frame: Through 100 and 365 days post allo-HSC infusion
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).
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.
Time frame: 1-48 (± 2) months post allo-HSC infusion
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
Plan to share: Yes
This study is terminated, as verified in Dec 2019. You cannot join it, but the record below documents what was studied.
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