Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Study Overview
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Brief Summary
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The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis

Official Title
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A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

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 the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26) Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject) The subject and his/her guardian(s) must have the ability to comply with the protocol Exclusion Criteria: The subject cannot walk without support Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis History of bone marrow transplantation 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. Pregnancy: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study Psychosis within the last 3 months Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial Participation in other interventional trials testing IMP except for studies with Lamazym

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 21 Years

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/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 |
| --- | --- | --- |
| Reduction of Oligosaccharides in blood serum | Efficacy endpoint evaluation as change from baseline | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of steps climbed in 3 minutes (3-minute stair climb) | Efficacy endpoint evaluation as change from baseline | 6 months | 
| Reduction of Oligosaccharides in CSF | Efficacy endpoint evaluation as change from baseline | 6 months | 
| The distance walked in 6 minutes (6-minute walk test) | Efficacy endpoint evaluation as change from baseline | 6 months | 
| Pulmonary function | Efficacy endpoint evaluation as change from baseline | 6 months | 
| Adverse events | Safety endpoint assesed weekly throughout the trial | 1 week | 
| Development of clinically significant changes in vital signs and change in physical examination | Safety endpoint assesed weekly throughout the trial | 1 week | 
| Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) | Safety endpoint assesed weekly throughout the trial | 1 week | 
| Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies | Safety endpoint assesed weekly throughout the trial | 1 week |