Treatment of alpha-Mannosidosis
Conditions
Sponsors
Zymenex A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. The patient cannot walk without support. 2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis 3. History of bone marrow transplantation 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. Presence of an ECHO with abnormalities within half a year 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 8. Psychosis within the last 3 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • Define effective dose based on evaluation of efficacy of rhLAMAN (Lamazym) from baseline on: The biochemical markers , The clinical parameters • To evaluate the long-term safety profile of rhLAMAN (Lamazym) ;Secondary Objective: • To determine the PK profile after repeated dose of rhLAMAN (Lamazym) in patients with alpha-Mannosidosis as measured by rhLAMAN levels in plasma ;Primary end point(s): Efficacy endpoints, measured at interim (3 months) and 6 months evaluation: Change from baseline of Oligosaccharide in serum-, urine- and cerebrospinal fluid Change from baseline in cerebrospinal fluid neurodegeneration biomarkers Change from baseline of MRS (white matter and mannose level) Change from baseline of functional capacity (6MWT, 3MSC, BOT2, Gait) Change from baseline of cognitive development (Leiter R) Change from baseline of Pulmonary function (FVC) Change from baseline of Hearing Safety endpoints: Adverse events Vital signs Urinalysis Hematology and blood chemistry rhLAMAN antibody and inhibitory antibody | — |
Countries
Denmark
Outcome results
None listed