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VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

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

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

NCT02898753•Valerion Therapeutics, LLC

View on ClinicalTrials.gov

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)

Detailed Description

Part 1 comprises 3 sequential cohorts of 4 patients each randomized to treatment with either VAL-1221 (at 3, 10, or 30 mg/kg) or positive control (rhGAA). Patients randomized to VAL-1221 will receive 7 intravenous (IV) infusions of VAL-1221 (one infusion every other week) over 12 weeks. Control patients will continue receiving their accustomed dose and regimen of Myozyme®. Part 2 is an uncontrolled extension to evaluate long-term effects of VAL-1221 given by IV infusion once every other week at doses up to 40 mg/kg.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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
+2 more criteria

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
+1 more criteria

Locations (3)

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

Key Dates

Start Date

June 21, 2017

Primary Completion Date

March 25, 2020

Completion Date

March 25, 2020

Last Updated

June 2, 2020

Enrollment

ACTUAL participants

Conditions

Pompe Disease

Interventions

VAL-1221

DRUG

RhGAA

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 2, 2020Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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