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
Shionogi Clinical Trials Administrator Clinical Support Help Line
CONTACT
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of California - Irvine Medical Center
Irvine, California, United States
RECRUITINGUniversity of Florida (UF) - Gainesville
Gainesville, Florida, United States
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
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
RECRUITINGUniversity of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States
RECRUITINGLysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, United States
RECRUITINGUZ Leuven
Leuven, Belgium
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