Clinical ALD Trials

A Prospective and Partially Retrospective Study to Characterize Safety and Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Childhood Cerebral Adrenoleukodystrophy

Funding Source:
bluebird bio

Cerebral ALD phenotype (cerALD)

Study Chair(s)/Principal Investigator(s):
Asif Paker, MD (bluebird bio)
Sandhya Kharbanda, MD
Keith van Haren, MD

Study Category:
Other: Observational Study

Study Status:
Active, currently recruiting

Not Applicable ID:

Study ID:

ALD Connect Affiliated Study

Observational Study

Study Summary:

Study ALD-103 will be a multi-site, global prospective and partially retrospective study that is designed to evaluate safety and efficacy of allo-HSCT in subjects with CCALD aged 17 or younger (i.e., <18 years of age). Suitability for allo-HSCT and the choice of the treatment
protocol utilized for these subjects will be determined by the subjects’ treating physicians as per their institutional policies/protocols and other local treatment guidelines. Procedures to be performed will be those according to institutional protocols and the accepted management of CCALD, including supportive care, choice of graft source, allo-HSCT protocol, prophylaxis and management of GVHD. This is a prospective and partially retrospective study that does not involve the use of an investigational drug, and describes the collection of a defined subset of the data from these procedures.

# of Subjects:

Enrollment Start Date:

Enrollment End Date:

Posting Last Modified Date:

Date Study Added to

Coordinating Center
Contact Information

bluebird bio, Inc.

bluebird bio, Inc.

150 Second Street
Cambridge, Massachusetts

Eligibility Criteria

Gender: Male

Maximum Age: 18

Time since Symptom Onset: Not applicable

Time since Diagnosis: Not applicable

Can participants use Riluzole? Yes

Other Eligibility Criteria:

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