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

• Status: Terminated
• Trial end date: 2020-03-25
• Target enrollment: 0
• Participant gender: All

Summary

This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Valerion Therapeutics, LLC

Criteria

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