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Shionogi 2405N1221 (Esprit)

  • Status
    Accepting Candidates
  • Age
    18 Years - N/A
  • Sexes
    All
  • Healthy Volunteers
    No
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Objective

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.

Details

Full study title A phase 2, multicenter, randomized, placebo-controlled, double-blind study to investigate the safety, pharmacodynamics, and preliminary efficacy of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease
Protocol number OCR49044
ClinicalTrials.gov ID NCT07123155
Phase Phase 2

Eligibility

Key Inclusion Criteria:

  • Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.

  • Participant must have a diagnosis of LOPD based on documentation of 1 of the

Following:

  1. Deficiency of acid alpha-glucosidase (GAA) enzyme

  2. GAA genotype

  • Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥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:

  1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters

  2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults

  • Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months.

Key Exclusion Criteria:

  • Has a medical condition or any other extenuating circumstance that may 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.

  • Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .

  • Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.

  • Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.

  • Participant, if female, is pregnant or breastfeeding at screening.

  • Participant, whether male or female, is planning to conceive a child during the study.

Note: Other protocol-specified inclusion and exclusion criteria may apply.

Lead researcher

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  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.