Krabbe Disease Progression Study

The Natural History of Infantile Globoid Cell Leukodystrophy

Protocol Description

Girl in a pumpkin patchThis study is being done to help researchers better understand infantile globoid leukodystrophy (GLD), or Krabbe disease, a rare, inherited degenerative disorder that affects the nervous system and for which there is presently no cure. The study will gather information about participants over 18 months to document the progression of the disease. Relatively little is known about infantile GLD and no prior studies have examined its natural history, or progression, over an 18-month period. By better understanding parameters and progression of infantile GLD, researchers will lay a foundation for evaluation of potential future treatments developed for the disease.

Eligibility Criteria

Participants in this study include children of both genders, ages infant to 2 years, who have been diagnosed with GLD.
Boys and Girls: Ages infant to 24 months

Requirements

The study involves a series of evaluations at the NDRD clinic at Children’s Hospital of Pittsburgh of UPMC, including brain imaging and other tests, all of which are considered to be standard care for patients with Krabbe disease.
Visits: 7, each of which will occur over 1 or 2 days
Duration: 18 months

Leaflet for Study Candidates (PDF)

Status: Closed to Enrollment

Source(s) of Support

ACE BioSciences A/S, subsidiary of Zymenex Holding A/S

Primary Investigator

Maria L. Escolar, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Program Coordinator Mary Brannaman, MPA, BS
412-692-6350