VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

This is an interventional treatment trial for Pompe Disease focused on measuring GSD-II, Pompe Disease, Glycogen Storage Diseases, GAA Read More

Inclusion Criteria:

• Participant is able and willing to provide informed consent prior to any study procedures are performed

• Diagnosis of GSDII based on one of the following:

  • Endogenous cultured skin fibroblast GAA activity less than (<) 40 percent (%) of adult normal level
  • Endogenous whole blood or dried blood spot GAA activity in deficiency range
  • Genetic analysis showing pathogenic variants in both alleles
• Onset of Pompe disease-related symptoms after 1 year of age
• Previously treated with Myozyme or Lumizyme for at least 12 months and on a stable regimen for the past 6 months

• Sexually active participants who are willing to use an acceptable method of contraception (abstinence, oral contraceptives, barrier method with spermicide, surgical sterilization, implanted or injectable contraceptives with a stable dose for at least 1 month prior to Baseline, hormonal intra-uterine device [IUD] inserted at least 1 month prior to Baseline) during the study and for 30 days after completion of treatment

  • If participant is female and not considered to be of childbearing potential, she is at least 2 years post-menopause, has undergone a tubal ligation, a total hysterectomy or bilateral oophorectomy
  • If participant is female and of childbearing potential, she has a negative serum pregnancy test during screening and Baseline and must be willing to undergo pregnancy testing at specific intervals during the study
• Participant meets at least one of the following criteria: greater than (>) 30% and <80% predicted upright forced volume capacity (FVC) or participant is able to walk >20% but <80% predicted normal on 6-minute walk test with or without use of assistive devices
• Able to comply with protocol requirements

Exclusion Criteria:

• Cardiac involvement in first year of life
• Anti-GAA antibody titers >1:51,200 at two time points
• Prior use of chaperone therapy for GSD-II within the last 12 months
• Use of immunosuppressive medication other than glucocorticoids within 6 months prior to study enrollment
• Use of invasive ventilatory assistance other than Bilevel Positive Airway Pressure (BiPAP) at night or during periods of rest
• Has received any investigational medication or has enrolled in any study involving investigational drugs or therapies within 30 days prior to first dose of study drug
• Start of or change in usual regimen of albuterol or respiratory muscle training within 30 days prior to first dose of study drug
• History of sensitivity to any of the constituents of the study drug
• Participant is breastfeeding or planning to become pregnant or to breastfeed during the study or is currently breastfeeding
• Participant has a medical condition or circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol or the participant's well-being or safety
• Participant has any condition that, in the view of the investigator, places the participant at high risk of poor treatment compliance or of not completing the study