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Not yet recruiting NCT07750990

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

Phase II Interventional Pompe Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: S-606001, ERT.
Who it may be relevant to
Registry conditions: Pompe Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2, Open-label, Multicenter, Extension Study to Investigate the Long-term Safety and Efficacy of S-606001 in Patients With Late-onset Pompe Disease

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.

Interventions

  • Drug S-606001
    S-606001 will be administered orally.
  • Drug ERT
    ERT will be administered as an intravenous infusion.

Primary outcome measures

  • Number of Participants With Treatment-emergent Adverse Events [Time frame: Baseline through Week 96]
Secondary outcome measures (10)
  • 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) [Time frame: Baseline, Week 96]
  • Change from Baseline in Patient Global Impression of Severity (PGI-S) [Time frame: Baseline, Week 96]

Eligibility criteria

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

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07750990 · 2507N1223 · 2026-525915-15

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗