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Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis

NCT04031066 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2020-12-08

No results posted yet for this study

Summary

Randomized, double-blind, placebo-controlled, parallel group study where subjects will receive velmanase alfa or placebo for 24 weeks.

Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and functional assessments. Study treatment is administered weekly through i.v. infusions

Conditions

  • Alpha-Mannosidosis

Interventions

DRUG

Velmanase Alfa

infusion i.v. treatment

DRUG

Placebo

infusion i.v. treatment

Sponsors & Collaborators

  • Chiesi Farmaceutici S.p.A.

    lead INDUSTRY

Principal Investigators

  • Paul Harmatz, MD · UCSF Benioff Children's Hospital Oakland

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-01-11
Primary Completion
2021-10-27
Completion
2021-12-29
FDA Drug
Yes

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Drugs

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 NCT04031066 on ClinicalTrials.gov