Alpha-Mannosidosis
Conditions
Brief summary
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
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.
Interventions
DRUGLamazym
ERT, i.v. infusions weekly
Sponsors
Chiesi Farmaceutici S.p.A.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Adverse events | 3 year | AE documented at all visits |
| Change from baseline in Lamazym antibodies | 3 year | AB measured every 12th week |
Secondary
| Measure | 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 |
Countries
Denmark
Outcome results
None listed