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

Stopped early · Phase 1/Phase 2

Conditions studied: Pompe Disease

In brief

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)

Key facts

Study ID

NCT02898753

Run by

Valerion Therapeutics, LLC

People needed

12

Starts

2017-06-21

Expected to finish

2020-03-25

Last updated by the study team

2020-06-02

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California, Irvine — Orange, California, United States
• Duke University Medical Center — Durham, North Carolina, United States
• National Hospital for Neurology and Neurosurgery — London, United Kingdom

Full record on ClinicalTrials.gov

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