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

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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.

Trial contacts and locations

2

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Data sourced from clinicaltrials.gov

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