A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis.

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2010-022084-36-DK
• Date of registration: 20/08/2010
• Prospective Registration: Yes
• Primary sponsor: Zymenex A/S
• Public title: A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis.
• Scientific title: A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis.
• Date of first enrolment: 05/10/2010
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-022084-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Denmark

Key inclusion & exclusion criteria

Inclusion criteria:
1. The patient must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes
2. The patient must have an age at the time of screening = 5 year and = 20 years
3. The patient must have physical ability to perform 6-minutes walk test (6MWT), 3 minute-stair climb test (3MSCT) and pulmonary lung function test (spirometry, body plethysmography).
4. The patient must have the ability to mentally cooperate in the cognitive and motor function tests
5. The patient must have the ability to hear and follow a request. Hearing aids can be worn.
6. Patient or patient’s legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
7. The patient 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:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Treatment of alpha-Mannosidosis

Intervention(s)

Product Name: Lamazym
Product Code: rhLAMAN
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: recombinant human alpha-mannosidase
Current Sponsor code: rhLAMAN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 1,8-2,2

Primary Outcome(s)

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

Secondary ID(s)

rhLAMAN-02