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Evaluation of Long-term Efficacy of Treatment With Lamazym — rhLAMAN-10  

Alpha-Mannosidosis Clinical Trial

Official title:
A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials

Clinical Trial Summary

The overall objective is to evaluate the long-term efficacy of Lamazym i.v. treatment in patients with alpha-Mannosidosis previously enrolled in Lamazym trials and currently receiving the treatment according to the AfterCare Program.

Clinical Trial Description

The primary objective of the trial is to evaluate the impact of the long-term treatment with Lamazym upon the level of biomarker oligosaccharides in serum and upon the endurance as measured by the change from baseline in the number of steps climbed in 3 minutes (3MSCT). As secondary objectives, the long term efficacy of Lamazym will be investigated upon endurance as measured by the change from baseline in the number of meters walked in six minutes (6MWT), upon pulmonary function, motor proficiency by BOT-2 and hearing capability by audiometry. In addition, cognitive development will be assessed by Leiter-R test. CNS involvement will be evaluated with MRI/MRS (for patients who previously participated in rhLAMAN-02 trial), CSF biomarkers (Tau, NFL, GFAp) and CSF biomarkers oligosaccharides. Clearance of oligosaccharides in urine will be measured. Long-term safety and Pharmaco-Kinetic (PK) profile after long-term treatment as measured by rhLAMAN levels in plasma will be assessed as well. Quality of life will be assessed by questionnaires (CHAQ and EQ-5D-5L). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02478840
Study type Interventional
Source Chiesi Farmaceutici S.p.A.
Contact
Status Completed
Phase Phase 3
Start date February 2015
Completion date June 2015
View Details
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