Treatement of Alpha-Mannosidosis
Conditions
Interventions
Product Name: Lamazym
Product Code: rhLAMAN
Pharmaceutical Form: Powder for solution for infusion
Current Sponsor code: rhLAMAN
Other descriptive name: recombinant human alpha-mannosidase
Concentratio
Sponsors
Zymenex A/S
Eligibility
Sex/Gender
All
Inclusion criteria
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 Are the trial subjects under 18? yes Number of subjects for this age range: 8 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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 |
|---|---|
| Main Objective: The overall objective of this trial is the evaluation of long-term efficacy, safety and tolerability of Lamazym treatment in patients with alpha-Mannosidosis. The primary objectives of the trial are: Evaluation of long-term efficacy from baseline of Lamazym on reduction of the biomarker Oligosaccharides in blood and CSF and an improvement in the 3 minute stair climb, 6 minute walk test and pulmonary function from baseline. ;Secondary Objective: The secondary objectives of the trial are: To evaluate the long-term safety profile of Lamazym Evaluation of efficacy of Lamazym on: o The clinical parameters BOT2 and Hearing o The CSF biomarkers (Tau, NFLp, GFAp) o The cognitive development tested by Leiter R o The CNS improvement measured with MRI/MRS ;Primary end point(s): Primary Endpoint evaluation as change from baseline on Reduction of Oligosaccharides in blood serum and CSF and an improvement in the 3 minute stair climb, 6 minute walk test and pulmonary function ;Timepoint(s) of evaluation of this end point: Efficacy will be assessed at the Danish site as interim (January/February 2012) and as end evaluation (minimum 26 weeks post trial initiation). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety Endpoints measured throughout the trial of: Development of adverse events Development of clinically significant changes in vital signs and change in physical examination Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies Secondary Efficacy Endpoints evaluation as change from baseline on: CNS parameters: Reduced concentration of mannose-rich oligosaccharides in brain tissue as measured by MRS visual score, reduction of MRI diffusion coefficient in white matter, gray matter and centrum semi oval and Improved Cerebrospinal fluid neuro-degeneration biomarker (Tau, NFLp, GFAp) Clinical parameters: Improved functional capacity on the BOT2 and reduced hearing loss Cognitive ability: Improved age equivalence with Leiter R In addition, the CHAQ, filled by the subject’s guardian, will be evaluated ;Timepoint(s) of evaluation of this end point: Safety will be assessed at every visit. Efficacy will be assessed at the Danish site as interim (January/February 2012) and as end evaluation (minimum 26 weeks post trial initiation). | — |
Countries
Belgium, Denmark, Germany, Spain, United Kingdom
Contacts
Public ContactCEO
Zymenex A/S
Outcome results
None listed