Alpha-Mannosidosis
Conditions
Brief summary
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
Interventions
DRUGLamazym
ERT, i.v. infusions weekly
Sponsors
European Commission
Zymenex A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 21 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Reduction of Oligosaccharides in blood serum | 6 months | Efficacy endpoint evaluation as change from baseline |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Reduction of Oligosaccharides in CSF | 6 months | Efficacy endpoint evaluation as change from baseline |
| The distance walked in 6 minutes (6-minute walk test) | 6 months | Efficacy endpoint evaluation as change from baseline |
| Pulmonary function | 6 months | Efficacy endpoint evaluation as change from baseline |
| The number of steps climbed in 3 minutes (3-minute stair climb) | 6 months | Efficacy endpoint evaluation as change from baseline |
| Development of clinically significant changes in vital signs and change in physical examination | 1 week | Safety endpoint assesed weekly throughout the trial |
| Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) | 1 week | Safety endpoint assesed weekly throughout the trial |
| Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies | 1 week | Safety endpoint assesed weekly throughout the trial |
| Adverse events | 1 week | Safety endpoint assesed weekly throughout the trial |
Countries
Belgium, Denmark, Spain, United Kingdom
Outcome results
None listed