The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
45
Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52
Time frame: Baseline, Week 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline up to Week 53
Plasma Concentration of S-606001
Time frame: Up to Week 12
Change From Baseline in Serum Creatine Kinase Levels at Week 52
Time frame: Baseline, Week 52
Change From Baseline in 6-minute Walk Test (6MWT) at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Pulmonary Function Parameter: Maximal Inspiratory Pressure (MIP) at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Pulmonary Function Parameter: Maximal Expiratory Pressure (MEP) at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Motor Function Parameter: Gait, Stair, Gower's Maneuver, Chair (GSGC) Score at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS-Fatigue-8a) Score at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Patient-Reported Outcomes Measurement Information System Physical Function 20-item short form (PROMIS-PF-20) Score at Week 52
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University of California - Irvine Medical Center
Irvine, California, United States
RECRUITINGUniversity of Florida (UF) - Gainesville
Gainesville, Florida, United States
NOT_YET_RECRUITINGEmory University Hospital
Atlanta, Georgia, United States
NOT_YET_RECRUITINGWashington University in St. Louis
St Louis, Missouri, United States
RECRUITINGDuke University Medical Center
Durham, North Carolina, United States
NOT_YET_RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
NOT_YET_RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGUniversity of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States
NOT_YET_RECRUITINGLysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, United States
RECRUITINGUZ Leuven
Leuven, Belgium
NOT_YET_RECRUITING...and 18 more locations
Time frame: Baseline, Week 52
Change From Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 PAIN) Score at Week 52
Time frame: Baseline, Week 52
Change From Baseline in 36-item Short Form Health Survey (SF-36) Score at Week 52
Time frame: Baseline, Week 52
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score at Week 52
Time frame: Baseline, Week 52