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

NCT02191917 | Terminated Results

• 2 other identifiers: 701-2012014-002158-38
• Study Type: observational
• Target: 8
• Locations: 2 countries
• Sites: 2

Brief Summary

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 .

Conditions

Late-onset Pompe Disease

Outcome Measures

Primary Outcomes (1)

• Maximal Inspiratory Pressure (MIP)

  Pulmonary measure of change in maximal inspiratory pressure (MIP)

  Baseline, Week 28

Secondary Outcomes (1)

• Maximal Expiratory Pressure (MEP)

  Baseline, Week 28

Study Arms (1)

Measurement of Respiratory Muscle Strength

Procedure: Respiratory muscle strength measurements by different techniques.

Interventions

Respiratory muscle strength measurements by different techniques. PROCEDURE

Measurement of Respiratory Muscle Strength

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Fifteen subjects were planned for enrollment; there were no screening failures; 8 subjects were enrolled. Two subjects were enrolled from 701-301 and the remaining 6 subjects enrolled directly into 701-201 (no parent study)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (2)

University of Florida

Gainesville, Florida, 32610, United States

Location

Royal Brompton Hospital

London, SW36NP, United Kingdom

Location

Limitations and Caveats

The study was planned to have 15 participants. However, the study was terminated prematurely. Only 8 participants were enrolled. Seven participants had last assessment at Days 146, 168, 172, 174, 179, 188, 196, translating to one participant at Week 20 and six participants Weeks 24-28.

Results Point of Contact

• Title: Trial Specialist
• Organization: BioMarin Pharmacuetical Inc.

medinfo@bmrn.com

1-800-983-4587

Study Officials

• Medical Monitor, MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2014
• First Posted: July 16, 2014
• Study Start: October 21, 2014
• Primary Completion: June 22, 2016
• Study Completion: June 22, 2016
• Last Updated: January 9, 2023
• Results First Posted: January 9, 2023

Record last verified: 2022-04

Locations

US (1); GB (1)