Australasian Leukodystrophy Foundation | Incorporated No. IA41033

Gene Therapy Study Seeking Participants

ID-100200717

Gene Therapy Study Seeking Participants with Childhood Cerebral Adrenoleukodystrophy

An international clinical research study, called the Starbeam Study, is now enrolling boys, aged 17 and younger, who have been diagnosed with Childhood Cerebral Adrenoleukodystrophy (CCALD). CCALD symptoms usually occur in early childhood and progress rapidly, if untreated, ultimately leading to death.

CCALD is a rare, genetic brain disorder that occurs when a broken gene, the ABCD-1 gene, does not work properly. Boys with CCALD do not produce any or enough of the adrenoleukodystrophy protein, or ALDP. Without enough ALDP, cells in the brain collect and store too many very long chain fatty acids. These fatty acids are harmful to brain cells and can trigger inflammation, which is responsible for the breakdown of myelin, a protective sheath that surrounds and protects the nerve cells. As the myelin breaks down, the brain can no longer function properly. When untreated, this combination leads to the loss of function and, ultimately, death.

The Starbeam Study will assess the effectiveness and safety of an investigational gene therapy approach, known as gene transfer. The study involves transferring a new copy of the ABCD-1 gene into the patient’s own blood stem cells. The goal of the Starbeam Study is to determine if the one-time investigational gene therapy treatment can stop the progression of CCALD and if it is safe and well-tolerated.

Participation in the Starbeam Study will last approximately 26 months. At its completion, participants will be asked to participate in a separate, long-term follow-up study, as the long-term benefits and risks of gene therapy are unknown.

To learn more about the Starbeam Study, an investigational gene therapy for CCALD, please visit http://clinicaltrials.gov/ct2/show/NCT01896102?term=ALD-102&rank=1.

The Starbeam Study is sponsored by bluebird bio, a Cambridge, Mass., biotechnology company, that is dedicated to developing next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases.