Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
- Registration Number
- NCT07123155
- Lead Sponsor
- Shionogi
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 45
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Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.
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Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
- Deficiency of acid alpha-glucosidase (GAA) enzyme
- GAA genotype
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Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position.
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Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:
- Screening values of 6-minute walk distance (6MWD) are ≥75 meters
- Screening values of 6MWD are ≤90% of the predicted value for healthy adults
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Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months.
Key
- Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements.
- Has active infections at screening.
- Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
- Current or chronic history of liver disease.
- Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
- Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.
- Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
- Participant, if female, is pregnant or breastfeeding at screening.
- Participant, whether male or female, is planning to conceive a child during the study.
Note: Other protocol-specified inclusion and exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description S-606001 Low Dose S-606001 Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks. S-606001 High Dose S-606001 Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks. Placebo Placebo Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.
- Primary Outcome Measures
Name Time Method Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52 Baseline, Week 52
- Secondary Outcome Measures
Name Time Method Number of Participants With Treatment-emergent Adverse Events (TEAEs) Baseline up to Week 53 Change From Baseline in Pulmonary Function Parameter: Maximal Inspiratory Pressure (MIP) at Week 52 Baseline, Week 52 Change From Baseline in Pulmonary Function Parameter: Maximal Expiratory Pressure (MEP) at Week 52 Baseline, Week 52 Change From Baseline in Motor Function Parameter: Gait, Stair, Gower's Maneuver, Chair (GSGC) Score at Week 52 Baseline, Week 52 Change From Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS-Fatigue-8a) Score at Week 52 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 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 Baseline, Week 52 Change From Baseline in 36-item Short Form Health Survey (SF-36) Score at Week 52 Baseline, Week 52 Change From Baseline in Patient Global Impression of Severity (PGI-S) Score at Week 52 Baseline, Week 52 Plasma Concentration of S-606001 Up to Week 12 Change From Baseline in Serum Creatine Kinase Levels at Week 52 Baseline, Week 52 Change From Baseline in 6-minute Walk Test (6MWT) at Week 52 Baseline, Week 52