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

ConditionPompe Disease
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorShionogi

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • S-606001

    Drug

    S-606001 will be administered orally.

  • ERT

    Drug

    ERT will be administered as an intravenous infusion.

Treatment groups

60 Participants
are divided into 1 treatment group
Group A: S-606001Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Number of Participants With Treatment-emergent Adverse Events

Time frame
Baseline through Week 96

Secondary outcomes

1

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

Time frame
Baseline, Week 96
2

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

Time frame
Baseline, Week 96
3

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

Time frame
Baseline, Week 96
4

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

Time frame
Baseline, Week 96

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shionogi

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: