Clinical Trial: Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
Brief Summary: The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
Detailed Summary:
Sponsor: Zymenex A/S
Current Primary Outcome: Reduction of Oligosaccharides in blood serum [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- The number of steps climbed in 3 minutes (3-minute stair climb) [ Time Frame: 6 months ]Efficacy endpoint evaluation as change from baseline
- Reduction of Oligosaccharides in CSF [ Time Frame: 6 months ]Efficacy endpoint evaluation as change from baseline
- The distance walked in 6 minutes (6-minute walk test) [ Time Frame: 6 months ]Efficacy endpoint evaluation as change from baseline
- Pulmonary function [ Time Frame: 6 months ]Efficacy endpoint evaluation as change from baseline
- Adverse events [ Time Frame: 1 week ]Safety endpoint assesed weekly throughout the trial
- Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ]Safety endpoint assesed weekly throughout the trial
- Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 1 week ]Safety endpoint assesed weekly throughout the trial
- Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies [ Time Frame: 1 week ]Safety endpoint assesed 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: January 2012
Date Completion:
Last Updated: March 28, 2017
Last Verified: March 2017