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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objectives of the study are: • To evaluate the safety profile of rhLAMAN (Lamazym) • To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma ;Secondary Objective: The secondary objectives are to collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03);Primary end point(s): safety endpoints: Adverse events Vital signs Urinalysis Hematology and blood chemistry rhLAMAN antibody and inhibitory antibody | — |
Countries
Denmark
Outcome results
None listed