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

NCT02898753 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-06-02

No results posted yet for this study

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)

Conditions

  • Pompe Disease

Interventions

DRUG

VAL-1221

VAL-1221 3, 10, or 30 mg/kg as per the dose and schedule specified in the arm group description

DRUG

RhGAA

Active comparator

Sponsors & Collaborators

  • Valerion Therapeutics, LLC

    lead INDUSTRY

Principal Investigators

  • Hal Landy, MD · Valerion Therapeutics, LLC

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-21
Primary Completion
2020-03-25
Completion
2020-03-25
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02898753 on ClinicalTrials.gov