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

NCT02998879 | Completed Phase 2

• 2 other identifiers: CCD-LMZYMAA1-082016-001988-36
• Study Type: interventional
• Target: 5
• Locations: 5 countries
• Sites: 6

Brief 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

Conditions

Alpha-Mannosidosis

Outcome Measures

Primary Outcomes (6)

• Safety and tolerability of velmanase alfa as per Adverse events

  Safety and tolerability assessed as per AEs including infusion-related reactions \[IRRs\]

  From baseline throughout study completion, at least of 2 years

• Safety and tolerability of velmanase alfa as per vital signs

  From baseline throughout study completion, at least of 2 years

• Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per hematology

  From baseline throughout study completion, at least of 2 years

• Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per blood biochemistry

  From baseline throughout study completion, at least of 2 years

• Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per urinalysis

  From baseline throughout study completion, at least of 2 years

• Detection of anti-velmanase alfa-IgG antibodies (ADA) and neutralizing/inhibitory antibodies

  Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained

  From baseline throughout study completion, at least of 2 years

Secondary Outcomes (22)

• Evaluation of levels of Serum oligosaccharides

  From baseline throughout study completion, at least for 2 years

• Functional capacity: The Peabody Developmental Motor Scale test (PDMS-2)

  From baseline throughout study completion, at least for 2 years

• Functional capacity: Bruininks-Oseretsky test of Motor Proficiency (BOT-2) when applicable by age (from 4 years) or upon the judgment of the physician

  From baseline throughout study completion, at least for 2 years

• Functional capacity: Mullen Scales of Early Learning (MSEL)

  From baseline throughout study completion, at least for 2 years

• Endurance: 3-Minute Stair Climb Test (3MSCT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician

  From baseline throughout study completion, at least for 2 years

Study Arms (1)

Velmanase Alfa

EXPERIMENTAL

velmanase alfa 1mg/kg body weight infusion

Drug: Velmanase Alfa (e.g. Lamazym)

Interventions

Velmanase Alfa (e.g. Lamazym) DRUG

iv infusion treatment

Also known as: Lamazym

Velmanase Alfa

Eligibility Criteria

• Age: Up to 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patient's custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities
• The subject's custodial parent(s) must have the ability to comply with the protocol
• The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity in leukocytes or fibroblasts \< 10% of normal activity (historical data)
• The subject must have an age at the time of screening \< 6 years.

You may not qualify if:

• The subject's diagnosis cannot be confirmed by alpha-mannosidase activity \< 10% of normal activity
• Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
• History of BMT
• Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
• Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
• Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
• Participation in other interventional trials testing the IMP within the last 3 months.

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead
• Cromsource: collaborator

Study Sites (6)

Unknown Facility

Vienna, Austria

Location

Unknown Facility

Copenhagen, Denmark

Location

Unknown Facility

Lyon, France

Location

Unknown Facility

Hamburg, Germany

Location

Unknown Facility

Mainz, Germany

Location

Unknown Facility

Trieste, Italy

Location

Related Links

• Study Record on EU Clinical Trials Register including results

MeSH Terms

Conditions

alpha-Mannosidosis

Condition Hierarchy (Ancestors)

Mannosidase Deficiency Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2016
• First Posted: December 21, 2016
• Study Start: December 1, 2016
• Primary Completion: July 1, 2020
• Study Completion: July 1, 2020
• Last Updated: October 26, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); DK (1); IT (1); DE (2); AU (1)