Summary

for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
estimated completion

Description

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)

Official Title

A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients

Details

The study will be conducted in two parts. Part 1 is a 3-month active control phase which comprises 3 sequential cohorts of 4 participants each randomized to treatment with either VAL-1221 (at 3, 10, or 30 milligrams/kilogram [mg/kg]) or positive control (recombinant human acid alpha-glucosidase [rhGAA]). Eligible participants who complete Part 1, including those maintained on rhGAA, will be offered inclusion in Part 2 of the study. Part 2 is a 9-month uncontrolled extension to evaluate long-term effects of VAL-1221 given by intravenous (IV) infusion once every other week at doses up to 30 mg/kg.

Keywords

Pompe Disease GSD-II Glycogen Storage Diseases GAA Glycogen Storage Disease Type II VAL-1221 RhGAA VAL-1221 3 mg/kg VAL-1221 10 mg/kg VAL-1221 30 mg/kg

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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 CAN'T join 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

Locations

  • University of California, Irvine
    Orange California 92868 United States
  • Duke University Medical Center
    Durham North Carolina 27710 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Valerion Therapeutics, LLC
ID
NCT02898753
Phase
Phase 1/2
Study Type
Interventional
Last Updated