Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD) Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02204904
• Other study ID #: ALD-103
• Status: Terminated
• Start date: April 2015
• Est. completion date: December 6, 2019

Study information

• Verified date: December 2019
• Source: bluebird bio
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 59
• Est. completion date: December 6, 2019
• Est. primary completion date: December 6, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 17 Years

Eligibility

Inclusion Criteria:

1. 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.

2. 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.

3. Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.

4. 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:

1. Previous treatment with a gene therapy product.

2. Receipt of an experimental transplantation procedure.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenoleukodystrophy (ALD)
• Cerebral Adrenoleukodystrophy (CALD)
• X-Linked Adrenoleukodystrophy (X-ALD)

Intervention

Genetic:
• Allo-HSCT
Allogeneic Hematopoietic Stem Cell Transplantation

Locations

Country	Name	City	State
Argentina	Hospital Austral	Buenos Aires
Canada	McGill University Health Centre	Montréal	Quebec
Germany	University Hospital Leipzig	Leipzig
Italy	IRCCS Ospedale Pediatrico Bambine Gesú	Roma
Netherlands	Princess Maxima Center for Pediatric Oncology (PMC)	Utrecht
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Central Manchester University Hospitals NHS Foundation Trust	Manchester
United States	Boston Children's Hospital/Massachusetts General Hospital	Boston	Massachusetts
United States	Duke University Medical Center	Durham	North Carolina
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	University of Minnesota	Minneapolis	Minnesota
United States	Stanford University	Palo Alto	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
bluebird bio

Countries where clinical trial is conducted

United States,  Argentina,  Canada,  Germany,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Incidence and timing of neutrophil engraftment.		1-48 (± 1) months post allo-HSC infusion
Primary	Incidence and timing of platelet engraftment		1-48 (± 1) months post allo-HSC infusion
Primary	Incidence of engraftment failure or allograft rejection.		1-48 (± 1) months post allo-HSC infusion
Primary	Incidence of primary donor-derived chimerism of =50%.		by 100 days post allo-HSC infusion
Primary	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
Primary	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
Primary	Incidence of =Grade II acute GVHD.		1-48 (± 1) months post allo-HSC infusion
Primary	Incidence of chronic GVHD.		1-48 (± 1) months post allo-HSC infusion
Primary	Number of emergency room visits.		1-48 (± 1) months post allo-HSC infusion
Primary	Number and duration of intensive care unit stay.		1-48 (± 1) months post allo-HSC infusion
Primary	Number and duration of in-patient hospitalization.		1-48 (± 1) months post allo-HSC infusion
Secondary	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
Secondary	Change from Baseline in Loes score		1-48 (± 2) months post allo-HSC infusion
Secondary	Change from Baseline in Neurological Function Score (NFS)		1-48 (± 2) months post allo-HSC infusion
Secondary	Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable		1-48 (± 2) months post allo-HSC infusion
Secondary	MFD-free survival		48 (± 2) months post allo-HSC infusion
Secondary	Overall survival		48 (± 2) months post allo-HSC infusion