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.
Study Type
OBSERVATIONAL
Enrollment
59
Allogeneic Hematopoietic Stem Cell Transplantation
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, United States
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
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University of Minnesota
Minneapolis, Minnesota, United States
Duke University Medical Center
Durham, North Carolina, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Hospital Austral
Buenos Aires, Argentina
McGill University Health Centre
Montreal, Quebec, Canada
University Hospital Leipzig
Leipzig, Germany
IRCCS Ospedale Pediatrico Bambine Gesú
Roma, Italy
...and 3 more locations
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