Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Sufficient enrollment for Month 24 safety and efficacy endpoints
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
| |
| 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 |
| |
| Allo-HSCT retrospective | Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allo-HSCT | Genetic | Allogeneic Hematopoietic Stem Cell Transplantation |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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%. | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 1-48 (± 2) months post allo-HSC infusion |
| Change from Baseline in Loes score |
Not provided
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.
Depending on the cohort, the subject must:
Exclusion Criteria:
Not provided
Not provided
Not provided
Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Elizabeth McNeil, MD MSc | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States | ||
| Stanford University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34781360 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Type | Date | Date Unknown |
|---|---|---|
| Release | Mar 4, 2022 | |
| Reset | May 23, 2022 |
Not provided
Not provided
Not provided
Not provided
| 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 |
| 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 |
| Overall survival | 48 (± 2) months post allo-HSC infusion |
| 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 | 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 |
Not provided
| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Mar 4, 2022 | May 23, 2022 |
| ID | Term |
|---|---|
| D000326 | Adrenoleukodystrophy |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D018901 | Peroxisomal Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
Not provided
Not provided