Trial Outcomes & Findings for A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) (NCT NCT02191917)

NCT ID: NCT02191917

Last Updated: 2023-01-09

Results Overview

Pulmonary measure of change in maximal inspiratory pressure (MIP)

Recruitment status

TERMINATED

Target enrollment

8 participants

Primary outcome timeframe

Baseline, Week 28

Results posted on

2023-01-09

Participant Flow

Participant milestones

Participant milestones
Measure	Overall All enrolled subjects
Overall Study STARTED	8
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Overall n=8 Participants All subjects
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants
Race/Ethnicity, Customized White	8 Participants n=5 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants
Region of Enrollment United Kingdom	7 Participants n=5 Participants
Region of Enrollment United States	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Full Analysis Set (FAS) population includes all enrolled subjects who have at least one study assessment.

Pulmonary measure of change in maximal inspiratory pressure (MIP)

Outcome measures

Outcome measures
Measure	Overall n=8 Participants All participants
Maximal Inspiratory Pressure (MIP) Baseline	49.2 cm H2O Standard Deviation 21.7
Maximal Inspiratory Pressure (MIP) Last Visit	46.0 cm H2O Standard Deviation 20.93
Maximal Inspiratory Pressure (MIP) Change from Baseline	-5.5 cm H2O Standard Deviation 8.38

SECONDARY outcome

Timeframe: Baseline, Week 28

Population: Full Analysis Set (FAS) population includes all enrolled subjects who have at least one study assessment.

Pulmonary measure of change in maximal expiratory pressure (MEP)

Outcome measures

Outcome measures
Measure	Overall n=8 Participants All participants
Maximal Expiratory Pressure (MEP) Baseline	77.1 cm H2O Standard Deviation 32.39
Maximal Expiratory Pressure (MEP) Last Visit	69.0 cm H2O Standard Deviation 24.18
Maximal Expiratory Pressure (MEP) Change from Baseline	-11.6 cm H2O Standard Deviation 11.35

Adverse Events

Overall

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Overall n=8 participants at risk Overall
Injury, poisoning and procedural complications Post procedural haemorrhage	12.5% 1/8 • Number of events 1 • 28 weeks

Additional Information

Trial Specialist

BioMarin Pharmacuetical Inc.

Phone: 1-800-983-4587

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60