Lamazym Aftercare Study

Study Overview
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Brief Summary
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The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.

Detailed Description
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This protocol only concern subjects where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.

Official Title
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A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials

Conditions
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Alpha-Mannosidosis

Intervention / Treatment
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* Drug: Lamazym


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: the subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17 Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities The subject and his/her guardian(s) must have the ability to comply with the protocol Exclusion Criteria: 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 circumstances that, in the opinion of the Investigator, would preclude participation in the trial Pregnancy. Pregnant woman is excluded. Before start fot he treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception Psychosis; any psychotic disease, also in remission, is an exclusion criteria Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lamazym<br>1 mg Lamazym/kg body weight | Drug: Lamazym<br>* ERT, i.v. infusions weekly<br>* Other names: recombinant human alpha-mannosidase;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in Adverse events | AE documented at all visits | 3 year | 
| Change from baseline in Lamazym antibodies | AB measured every 12th week | 3 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| progress from baseline in number of steps climbed in 3 minutes |  | 1 year, 2 year and 3 year | 
| Progress from baseline in equivalent age |  | 1 year, 2 year and 3 year | 
| Progress from baseline in Forced Vital Capacity |  | 1 year, 2 year and 3 year | 
| Progress from baseline in distance walked in 6 minutes |  | 1 year, 2 year and 3 year |