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A phase 2, randomized, double-blind study of S-606001 as an add-on to enzyme replacement therapy (E

UCI Specialty Areas: Genetics, Neurology
Principal Investigator: Tahseen Mozaffar
Official 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
A Study On:
Brain - Neurologic/ Psychologic
Neuromuscular Diseases
Pompe Disease

Study Description

Eligibility

  1. Participant must be greater than or equal to 18 years of age and greater than or equal to 40 kilograms (kg) of body weight at the time of signing the informed consent.
  2. Participant must have a diagnosis of LOPD based on documentation of 1 of the following:

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

b. GAA genotype

  1. Participant has a %FVC greater than or equal to 30% and less than or equal to 80% in an upright position without mechanical ventilation at screening; or Participant has greater than or equal to 10% %FVC drop from upright position to supine position and %FVC greater than or equal to 20% in a supine position.
  2. Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:

a. Screening values of 6-minute walk distance (6MWD) are &greater than or equal to 75 meters

b. Screening values of 6MWD are less than or equal to 90% of the predicted value for healthy adults

  1. Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for greater than or equal to 24 months, with no regimen change in the last 6 months.

Key

  1. 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.
  2. Has active infections at screening.
  3. 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.
  4. Current or chronic history of liver disease.
  5. Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
  6. 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.
  7. Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
  8. Participant, if female, is pregnant or breastfeeding at screening.
  9. Participant, whether male or female, is planning to conceive a child during the study.

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

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Privacy Policy

University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

In the instance of parental permission, “You” refers to “Your child.”

  • Please read the information below and ask questions about anything that you do not understand. A
    researcher listed above will be available to answer your questions.
  • You are being asked to participate in a clinical research potential participant database to identify
    potential clinical research studies you may be eligible for. Participation in this database is voluntary.
    You may choose to skip any question. You may refuse to participate or discontinue your involvement
    at any time without penalty or loss of benefits. You are free to withdraw from this database at any
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    immediately    .
  • You are eligible to participate in this study if you may be interested in participating in a research trial at UCI Health
  • We would like to interview you to learn more about your eligibility for current clinical research studies at UCI. The interview will last about 5-30 minutes, depending on the extent of your medical history.
  • Possible risks/discomforts associated with the database are hearing about potential clinical research studies that may not be of interest to you, invasion of your privacy, or a potential breach of confidentiality.
  • There are no direct benefits from participation in the database. However, this database may help inform you on clinical research studies that you may be eligible for. Participation in UCI clinical research studies help us gain important data and knowledge that may lead to improved treatment options for people with similar conditions to you in the future.
  • There are no alternative procedures available. The only alternative is not to participate in this study.
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  • All information collected will be stored confidentially in a world-class securely-hosted application, “RealTime CTMS,” with robust data protections and encryption. This application has HIPAA compliant architecture and application rules to ensure data privacy and regulatory compliance.
  • Researchers will use your information to assess your eligibility for current and future clinical research studies at UCI. You may opt out at any time.
  • The research team, authorized UCI personnel, and regulatory entities, may have access to your study records to protect your safety and welfare.
  • While the research team will make every effort to keep your personal information confidential, it is possible that an unauthorized person might see it. We cannot guarantee total privacy.
  • Future Research Use
    Researchers will use your information to conduct this study. Once the study is done using your information, we may share them with other researchers so they can use them for other studies in the future. We will not share your name or any other private identifiable information that would let the researchers know who you are. We will not ask you for additional permission to share this de-identified information
  • Data Retention - In accordance with UC Office of the President policy, information will be retained for 10 years after the end of the calendar year in which the research is completed.
  • If, during the course of this study, significant new information becomes available that may relate to your willingness to continue to participate, this information will be provided to you by the research team listed at the top of the form.
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  • Please contact the UCI Institutional Review Board by phone, (949) 824-8170, by e-mail at IRB@research.uci.edu or at 160 Aldrich Hall, Irvine, CA 92697-7600 if you are unable to reach the researchers listed at the top of the form and have general questions; have concerns or complaints about the research; have questions about your rights as a research subject; or have general comments or suggestions.
What is an IRB? An Institutional Review Board (IRB) is a committee made up of scientists and nonscientists. The IRB’s role is to protect the rights and welfare of human subjects involved in research. The IRB also assures that the research complies with applicable regulations, laws, and institutional policies. UCI