Men and women who are at least 18 years old and have been diagnosed with late-onset Pompe disease are needed to participate in a research study for an investigational drug. Patients may participate if they have never received enzyme replacement therapy, and they may also participate if they have been receiving alglucosidase alfa for at least 9 months. Interested patients must also be able to walk at least 50 meters without stopping and without the use of an assistive device.
Participation in the study may least for up to 41 weeks with a 24-week treatment period. Participants will be given an infusion of the study drug, neoGAA, every other week during the treatment period. Infusions will take up to 4 hours each, and the treatment period will also involve 3 inpatient visits of 2 consecutive nights each. Enrolled participants will receive compensation for time and travel.
For more information, contact the Duke Clinical Research Unit at 919-613-6246 or firstname.lastname@example.org.