Pompe Disease
Conditions
Keywords
S-606001, Late-onset Pompe Disease, LOPD, ERT, Enzyme Replacement Therapy, NCT07123155, Muscle glycogen synthase, Liver glycogen synthase, Rare disease, Autosomal disease, Acid alpha-glucosidase, GAA, Glycogen storage disorder, GSD
Brief summary
The primary purpose of this study is to evaluate the long-term safety and tolerability profile of S-606001 in participants with LOPD. Participants who completed other S-606001 studies (for example, NCT07123155), including participants in a S-606001 study whose allocated arm was terminated by the sponsor and who are willing to participate in this study, will be enrolled globally.
Interventions
Sponsors
Shionogi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Participant must be ≥40 kilograms of body weight at the time of signing the informed consent * Participants who previously completed a S-606001 Pompe treatment study, including participants whose assigned treatment arm was terminated by the sponsor Key
Exclusion criteria
* Has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements * Current enrollment in any other clinical study involving an investigational intervention or any other type of medical research * Participant, if female, is pregnant or breastfeeding at visit 1 * Participant, whether male or female, is planning to conceive a child during the study Note: Other protocol-specified inclusion and
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Treatment-emergent Adverse Events | Baseline through Week 96 |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in Percent Forced Vital Capacity (%FVC) | Baseline, Week 96 |
| Change from Baseline in 6-minute Walk Test (6MWT) | Baseline, Week 96 |
| Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score | Baseline, Week 96 |
| Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator | Baseline, Week 96 |
| Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a) | Baseline, Week 96 |
| Change from Baseline in Patient-Reported Outcomes Measurement Information System Item Bank v2.0 - Physical Function - Short Form 20a (PROMIS-Item Bank-PF-20a) | Baseline, Week 96 |
| Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea) | Baseline, Week 96 |
| Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain) | Baseline, Week 96 |
| Change from Baseline in 36-item Short Form Health Survey (SF-36) | Baseline, Week 96 |
| Change from Baseline in Patient Global Impression of Severity (PGI-S) | Baseline, Week 96 |
Contacts
CONTACTShionogi Clinical Trials Administrator Clinical Support Help Line
STUDY_DIRECTORMedical Director
Shionogi Inc.
Outcome results
None listed