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
- Conditions
- Alfa-mannosidosisMedDRA version: 20.0Level: LLTClassification code 10032658Term: Other specified disorders of carbohydrate transport and metabolismSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2016-001988-36-FR
- Lead Sponsor
- Chiesi Farmaceutici S.p.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 5
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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Objective: Not applicable;Timepoint(s) of evaluation of this end point: 24 months;Primary end point(s): -Safety and tolerability of velmanase alfa as per:<br>oAdverse events (AEs, including infusion-related reactions [IRRs])<br>oVital signs <br>oClinical laboratory parameters (hematology, biochemistry and urinalysis)<br>-Detection of anti-velmanase alfa-immunoglobulin G (IgG) antibodies and neutralizing/inhibitory antibodies <br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Efficacy outcomes:<br>-Serum oligosaccharides <br>-Functional capacity:<br>-Endurance:<br>-Hearing evaluation:<br>-Immunological profile, <br>-CSF biomarkers<br>-Assessment of quality of life via Questionnaire<br>-Assessment of mannose-rich oligosaccharides in brain tissue<br>-Magnetic Resonance Imaging (MRI)<br>- Pharmacokinetic parameters (if data allows)<br>;Timepoint(s) of evaluation of this end point: 24 months