Late-onset Pompe disease.
Conditions
Brief summary
Change from baseline in %FVC at 52 weeks.
Detailed description
1. Assessment of adverse events and treatment-emergent adverse events. Physical examinations and vital sign measurements. Clinical laboratory evaluations including biochemistry, hematology, and urinalysis. Electrocardiograms., 2. Plasma concentration of S-606001., 3. Change from baseline in the parameters stated in the protocol., 4. Change from baseline in 6-minute walk test., 5. Change from baseline in pulmonary function parameters: maximal inspiratory pressure, maximal expiratory pressure. Change from baseline in motor function parameters: Gait, Stair, Gower’s Maneuver, Chair (GSGC) score, Daily Living Activity Levels. Change from baseline in the following parameters: PROMIS-Fatigue-8a, PROMIS-PF20, PROMIS-Dyspnea, PROMIS v2.0 PAIN, SF-36, PGI-S.
Interventions
DRUGS-606001
DRUGS-606001 matching placebo
Sponsors
Shionogi B.V.
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in %FVC at 52 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Assessment of adverse events and treatment-emergent adverse events. Physical examinations and vital sign measurements. Clinical laboratory evaluations including biochemistry, hematology, and urinalysis. Electrocardiograms., 2. Plasma concentration of S-606001., 3. Change from baseline in the parameters stated in the protocol., 4. Change from baseline in 6-minute walk test., 5. Change from baseline in pulmonary function parameters: maximal inspiratory pressure, maximal expiratory pressure. Change from baseline in motor function parameters: Gait, Stair, Gower’s Maneuver, Chair (GSGC) score, Daily Living Activity Levels. Change from baseline in the following parameters: PROMIS-Fatigue-8a, PROMIS-PF20, PROMIS-Dyspnea, PROMIS v2.0 PAIN, SF-36, PGI-S. | — |
Countries
Belgium, Denmark, France, Germany, Italy, Spain
Outcome results
None listed