A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis

• Conditions: Treatement of Alpha-Mannosidosis; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2012-000979-17-SE
• Lead Sponsor: Zymenex A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-06-07
• Last Update Posted: 4 August 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

1. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
2. The subject and his/her guardian(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 < 10% of normal activity (historical data)
4. The subject must have an age at the time of screening = 5 years and = 35 years
5. The subject must have the ability to physically and mentally cooperate in the tests
6. The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
Are the trial subjects under 18? yes
Number of subjects for this age range: 13
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. The subjects diagnosis cannot be confirmed by alpha-mannosidase activity < 10% of normal activity
2. The subject cannot walk without support
3. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis
4. History of BMT
5. 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
6. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
7. Pregnancy: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
8. Psychosis any psychotic disease, also in remission, is an exclusion criteria
9. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
10. Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
11. Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
12. Total IgE >800 IU/ml
13. Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis;Secondary Objective: PK evaluation;Primary end point(s): Primary Efficacy Endpoints is change from baseline in the active treated group versus the placebo group:<br>• The level of oligosaccharides in serum <br>• 3-minute stair climb test (3MSCT);Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site at baseline (prior to first dose), as midterm evaluation (after 26±3 weeks, referred to as midterm evaluation or visit 26a), and as end evaluation (after 52±3 weeks, referred to as end evaluation or visit 52a).