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Clinical Trial 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


Clinical Trial 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 ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02998879
Study type Interventional
Source Chiesi Farmaceutici S.p.A.
Contact
Status Completed
Phase Phase 2
Start date December 2016
Completion date July 2020

See also
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Completed NCT01681940 - Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis Phase 2
Withdrawn NCT04031066 - Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis Phase 3
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Enrolling by invitation NCT01908712 - Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients Phase 3
Completed NCT01681953 - A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis Phase 3
Active, not recruiting NCT01372228 - Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Phase 1/Phase 2
Available NCT04959240 - Expanded Access to Velmanase Alfa