NCT02998879

Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis

A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years

Detailed description

The Primary endpoints of the study include: * Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis) * Detection of anti-velmanase alfa antibodies and neutralizing/inhibitory antibodies The Secondary endpoints include changes from baseline to 24 months for the following parameters. Efficacy outcomes: * Serum oligosaccharides * Functional capacity: Peabody Developmental Motor Scale - 2nd edition (PDMS-2) scores, Mullen's Scale of Early Learning (MSEL) scores, Bruininks-Oseretsky Test Of Motor Proficiency-2nd Edition (BOT-2), when applicable by age (from 4 years) or upon the judgment of the physician * Endurance: 3-Minute Stair Climb Test (3MSCT) and 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician, 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age, or when applicable according to the judgment of the physician * Hearing evaluation: Otoacoustic Emissions (OAE) testing, Automatic Auditory Brainstem Response (A-ABR) audiometry * Immunological profile, when applicable upon the judgment of the physician: * CSF biomarkers: Tau protein (Tau), Neurofilament Protein Light (NFL), Glial Fibrillary Acidic Protein (GFAp), Oligosaccharides * Assessment of quality of life via Questionnaire to parents * Assessment of mannose-rich oligosaccharides in brain tissue, MRI * Pharmacokinetic parameters

Conditions

• Alpha-Mannosidosis

Interventions

Type	Name	Description
DRUG	Velmanase Alfa (e.g. Lamazym)	iv infusion treatment

Timeline

• Start date: 2016-12-01
• Primary completion: 2020-07-01
• Completion: 2020-07-01
• First posted: 2016-12-21
• Last updated: 2021-10-26

Locations

6 sites across 5 countries: Austria, Denmark, France, Germany, Italy