A Study of Repiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Status:	Terminated
Conditions:	Diabetes
Therapuetic Areas:	Endocrinology
Healthy:	No
Age Range:	18 - Any
Updated:	11/8/2017
Start Date:	September 2014

Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will
test respiratory muscle strength initially and again after 24 weeks in subjects treated or
not treated with BMN 701 .

Inclusion Criteria:

- Willing and able to provide written informed consent, after the nature of the study
has been explained, and prior to any study-related procedures

- Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study
entry

- Willing and able to comply with all study procedures

Exclusion Criteria:

- Requires ventilatory support while awake and in the upright position

- Concurrent disease, medical condition, or extenuating circumstance that, in the
opinion of the investigator, might compromise patient well-being, study completion, or
data collection

- Allergy to tools or procedures used for respiratory muscle testing

We found this trial at

sites

Royal Brompton Hospital

London, SW3 6LL

Principal Investigator: Michael Polkey, MD

Phone: 0207 352 8121

from

London,