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



Status:Recruiting
Conditions:Endocrine
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:Any - 17
Updated:1/20/2019
Start Date:April 2015
End Date:May 2021
Contact:bluebird bio
Email:clinicaltrials@bluebirdbio.com
Phone:339-499-9300

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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

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.


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.
We found this trial at
7
sites
Boston, Massachusetts 02115
Principal Investigator: Christine Duncan, MD
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Neena Kapoor, MD
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Paul Orchard, MD
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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Minneapolis, MN
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Buenos Aires,
Principal Investigator: Hernan Amartino, MD
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Vinod Prasad, MD
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Durham, NC
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Palo Alto, California 94304
Principal Investigator: Ami Shah, MD
Phone: 650-724-9179
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Palo Alto, CA
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Salt Lake City, Utah 84132
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Salt Lake City, UT
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