Clinical Trial: A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects With Alpha-Man

Brief Summary: The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis

Detailed Summary:
Sponsor: Zymenex A/S

Current Primary Outcome:

• Reduction of oligosaccharides in serum [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ]
  Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
• The number of steps climbed in 3 minutes (3-minute stair climb test) [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ]
  Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Forced Vital Capacity [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ]
  Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
• The distance walked in 6 minutes (6-minute walk test) [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ]
  Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
• Adverse Events [ Time Frame: 1 week ]
  Safety endpoint assessed weekly throughout the trial
• Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ]
  Safety endpoints assessed weekly throughout the trial
• Clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 1 week ]
  Safety endpoints assessed weekly throughout the trial
• Development of Lamazym antibodies and neutralizing/inhibitory antibodies [ Time Frame: 1 week ]
  Safety endpoints assessed weekly throughout the trial

Original Secondary Outcome: Same as current

Information By: Chiesi Farmaceutici S.p.A.

Dates:
Date Received: August 22, 2012
Date Started: August 2012
Date Completion:
Last Updated: March 28, 2017
Last Verified: March 2017