Health TrialsbyHealthPanel

A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

ConditionPompe Disease
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorShionogi

About this trial

The primary purpose of this study is to evaluate the safety and tolerability profile of S-606001 in participants with LOPD.

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 form

Participant must have a diagnosis of LOPD based on documentation of the following: deficiency of acid alpha-glucosidase (GAA) enzyme; GAA genotype

Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening or participant has >80% FVC in upright position and ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position

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

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

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

Trial design

Design model

Single group

Treatments tested in this trial

  • S-606001

    Drug

    S-606001 will be administered orally as a tablet.

Treatment groups

15 Participants
are divided into 1 treatment group
Group A: S-606001Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants with Treatment-emergent Adverse Events

Time frame
Baseline (Day 1) through Week 49

Secondary outcomes

1

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

Time frame
Baseline (Day 1), Week 48
2

Change From Baseline in Maximum Expiratory Capacity (MEP)

Time frame
Baseline (Day 1), Week 48
3

Change From Baseline in Minimum Inspiratory Capacity (MIP)

Time frame
Baseline (Day 1), Week 48
4

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

Time frame
Baseline (Day 1), Week 48

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: