A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age

• Register: EUCTR
• Last refreshed on: 28 September 2020
• Main ID: EUCTR2016-001988-36-AT
• Date of registration: 22/02/2017
• Prospective Registration: Yes
• Primary sponsor: Chiesi Farmaceutici S.p.A.
• Public title: A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age
• Scientific title: 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
• Date of first enrolment: 10/04/2017
• Target sample size: 3
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-001988-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Denmark, Germany

Contacts

• Name: Clinical Project Manager
• Affiliation: Chiesi Farmaceutici S.p.A.

• Name: Clinical Project Manager
• Affiliation: Chiesi Farmaceutici S.p.A.

Key inclusion & exclusion criteria

Inclusion criteria:
1) Patient’s custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities;
2) The subject’s custodial parent(s) must have the ability to comply with the protocol;
3) 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);
4) The subject must have an age at the time of screening < 6 years.

Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
The subject’s diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity;
2) Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis;
3) History of Bone Marrow Transplantation (BMT);
4) 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;
5) Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial;
6) Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial;
7) Participation in other interventional trials testing the IMP within the last 3 months.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Alfa-mannosidosis
MedDRA version: 20.0 Level: LLT Classification code 10032658 Term: Other specified disorders of carbohydrate transport and metabolism System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Intervention(s)

Product Name: Velmanase alfa
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: VELMANASE ALFA
CAS Number: 1492823-75-2
Other descriptive name: recombinant human alpha-mannosidase
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Primary end point(s): -Safety and tolerability of velmanase alfa as per:
oAdverse events (AEs, including infusion-related reactions [IRRs])
oVital signs
oClinical laboratory parameters (hematology, biochemistry and urinalysis)
-Detection of anti-velmanase alfa-immunoglobulin G (IgG) antibodies

Secondary Objective: Not applicable

Timepoint(s) of evaluation of this end point: 24 months

Main Objective: The main objectives of this trial are to evaluate safety and efficacy of repeated velmanase alfa i.v. infusions in pediatric alpha-mannosidosis patients aged less than 6 years" as per section 2 of the study protocol

Secondary Outcome(s)

Secondary end point(s): Efficacy outcomes:
-Serum oligosaccharides
-Functional capacity:
-Endurance:
-Hearing evaluation:
-Immunological profile,
-CSF biomarkers
-Assessment of quality of life via Questionnaire
-Assessment of mannose-rich oligosaccharides in brain tissue
-Magnetic Resonance Imaging (MRI)
- Pharmacokinetic parameters (if data allows)

Timepoint(s) of evaluation of this end point: 24 months

Secondary ID(s)

2016-001988-36-DK

CCD-LMZYMAA1-08

Source(s) of Monetary Support

Chiesi Farmaceutici S.p.A.

Ethics review

Status: Approved
Approval date: 28/02/2017
Contact: