The purpose of this research study is to compare the safety and effectiveness of two enzyme replacement therapies (the investigational medication neoGAA and the currently approved therapy alglucosidase alfa) in patients with late-onset Pompe disease who have not tried prior treatment.
Who may qualify.
The Comet Study may be appropriate for those who meet the following criteria:
- Three years of age or older
- Able to walk 40 meters (approximately 130 feet) without stopping and without an assistive device
- Has not had previous treatment with immune tolerance induction therapy, alglucosidase alfa, or any investigational therapy for Pompe disease
After you or your loved one gives consent to participate in this research study, there will be an initial screening visit to determine your or your loved one’s eligibility for the Comet Study. If this study is a good fit, you or your loved one will receive either the investigational enzyme replacement therapy or the currently approved enzyme replacement therapy (alglucosidase alfa) by IV infusion approximately every two weeks for approximately three years (149 weeks).
See If You May Qualify
Learn more about Pompe disease.