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

BioMarin Pharmaceutical

Status and phase: Terminated; Phase 2

Conditions: Late-onset Pompe Disease

Treatments: Procedure: Respiratory muscle strength measurements by different techniques.

Study type: Observational

Funder types: Industry

Identifiers

NCT02191917

2014-002158-38 (EudraCT Number)

701-201

Details and patient eligibility

About

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 .

Enrollment: 8 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

8 participants in 1 patient group

Measurement of Respiratory Muscle Strength

Treatment:

Procedure: Respiratory muscle strength measurements by different techniques.