A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease

A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease - BMN701-201

Status

Active, not recruiting

Phases

Phase 4

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON43764

Enrollment

Registered

2016-05-24

Start date

2015-03-31

Completion date

Unknown

Last updated

2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Late-onset Pompe disease

Interventions

Late-onset Pompe disease

Respiratory Muscle Strength

Eligibility

Age

18 Years to 64 Years

Inclusion criteria

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 based on GAA gene mutations and/or endogenous GAA activity

Exclusion criteria

Exclusion criteria: * Requires ventilatory support while awake and in the upright position. Subjects who require night time ventilator support may only enroll upon obtaining approval from the sponsor. * Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise patient wellbeing, study completion, or data collection. - subjects with a diagnosis of asthma, chronic obstructive pulmonary disease, emphysema, or any other condition that may manifest with airway obstruction should be excluded, unless the subject has an FEV1/FVC ratio > 0.80 on spirometry (in another study or elsewhere) performed within 3 months in this study. * Swallowing difficulty precluding balloon catheter placement (eg, esophageal strictures) * Allergy to tools or procedures used for respiratory muscle testing * Implanted ferrous metals (eg, cardiac pacemakers) * Subjects taking medication for the treatment of obstructive lung disease (including, but not limited to, systemic or inhaled beta agonists, inhaled corticosteroids, or inhaled anticholinergic/anti-muscarinic agents) may not be enrolled in this study. * Subjects who are pregnant, planning to become pregnant, or unable/unwilling to use effective contraception (as evaluated by the principal investigator) for the duration of the study.

Design outcomes

Primary

Measure	Time frame
The primary objective of this study is to compare respiratory muscle strength values obtained using volitional techniques with values obtained using non-volitional techniques	—

Secondary

Measure	Time frame
The secondary objective is to compare the change for these two sets of values after 24 weeks in subjects treated or not treated with BMN 701.	—

Countries

The Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)