Actively Recruiting
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Led by Shionogi · Updated on 2026-04-30
45
Participants Needed
28
Research Sites
92 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
CONDITIONS
Official Title
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must be 18 years of age or older and weigh at least 40 kilograms at consent.
- Diagnosis of late-onset Pompe disease confirmed by acid alpha-glucosidase enzyme deficiency or GAA genotype.
- Forced vital capacity (%FVC) between 30% and 80% upright without mechanical ventilation, or at least 10% drop in %FVC from upright to supine with supine %FVC at least 20%.
- Ability to perform a 6-minute walk test with a distance of at least 75 meters and at least 90% of predicted value for healthy adults.
- Currently receiving enzyme replacement therapy for at least 24 months with no changes in regimen in the last 6 months.
You will not qualify if you...
- Medical conditions or circumstances posing undue safety risk or impacting protocol compliance.
- Active infections at screening.
- Malignancy within the past 5 years except for certain skin cancers resected without metastatic disease for 3 years.
- Current or chronic liver disease.
- Known biallelic loss of function mutations in glycogenin gene (GYG) or glycogen phosphorylase muscle-associated gene (PYGM).
- Receipt of investigational therapy or pharmacological treatment for Pompe disease within 30 days or 5 half-lives before day 1 or planned during the study.
- Prior gene therapy or RNA therapy for Pompe disease.
- Pregnancy or breastfeeding at screening.
- Planning to conceive a child during the study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 28 locations
1
University of California - Irvine Medical Center
Irvine, California, United States, 92868
Actively Recruiting
2
University of Florida (UF) - Gainesville
Gainesville, Florida, United States, 32611
Actively Recruiting
3
Emory University Hospital
Atlanta, Georgia, United States, 30322
Not Yet Recruiting
4
Washington University in St. Louis
St Louis, Missouri, United States, 63130
Actively Recruiting
5
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
9
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, United States, 22030
Actively Recruiting
10
UZ Leuven
Leuven, Belgium
Actively Recruiting
11
Aarhus University Hospital
Aarhus, Denmark
Actively Recruiting
12
HLC Hopital Pierre Wertheimer
Bron, France
Actively Recruiting
13
AP-HP Hopital Raymond Poincare
Garches, France
Actively Recruiting
14
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, France
Actively Recruiting
15
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, France
Not Yet Recruiting
16
Universitaetsklinikum Halle (Saale)
Halle, Germany
Actively Recruiting
17
SphinCS GmbH
Höchheim, Germany
Actively Recruiting
18
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, Germany
Actively Recruiting
19
A.O.U. Policlinico "G. Martino"
Messina, Italy
Not Yet Recruiting
20
AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino, Italy
Actively Recruiting
21
Erasmus MC
GE Rotterdam, GE Rotterdam, Netherlands
Actively Recruiting
22
Hospital Universitario 12 de Octubre
Madrid, Spain
Actively Recruiting
23
Hospital Universitari i Politecnic La Fe
Valencia, Spain
Actively Recruiting
24
Salford Royal Hospital
Statford, Statford, United Kingdom
Actively Recruiting
25
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Not Yet Recruiting
26
National Hospital for Neurology & Neurosurgery
London, United Kingdom
Not Yet Recruiting
27
Royal Free London NHS Foundation Trust
London, United Kingdom
Actively Recruiting
28
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Actively Recruiting
Research Team
S
Shionogi Clinical Trials Administrator Clinical Support Help Line
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here