An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Phase 2Not Yet RecruitingNCT07750990

Shionogi60 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pompe Disease

Interventions

S-606001DRUG

S-606001 will be administered orally.

ERTDRUG

ERT will be administered as an intravenous infusion.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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 exclusion criteria may apply.

Outcomes

Primary Outcomes

Number of Participants With Treatment-emergent Adverse Events

Time frame: Baseline through Week 96

Secondary Outcomes

Change from Baseline in Percent Forced Vital Capacity (%FVC)

Time frame: Baseline, Week 96

Change from Baseline in 6-minute Walk Test (6MWT)

Time frame: Baseline, Week 96

Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score

Time frame: Baseline, Week 96

Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator

Time frame: Baseline, Week 96

Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a)

Time frame: 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)

Time frame: Baseline, Week 96

Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea)

Time frame: Baseline, Week 96

Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain)

Time frame: Baseline, Week 96

Change from Baseline in 36-item Short Form Health Survey (SF-36)

Central Contacts

Shionogi Clinical Trials Administrator Clinical Support Help Line

CONTACT

1-800-849-9707 Shionogiclintrials-admin@shionogi.co.jp

Data from ClinicalTrials.gov

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