NL-OMON43764
Not Yet Recruiting
Phase 4
A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease - BMN701-201
Overview
- Phase
- Phase 4
- Intervention
- Not specified
- Conditions
- Late-onset Pompe disease
- Sponsor
- BioMarin Pharmaceutical Inc.
- Enrollment
- 5
- Status
- Not Yet Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility 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 \<75% of the lower limit of the normal adult range reported by the testing laboratory, assessed by dried blood spot, whole\-blood, skin fibroblasts, or muscle biopsy
- •\* 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. 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.
Outcomes
Primary Outcomes
Not specified
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