Brief Title
Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
Official Title
A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis.
Brief Summary
This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety
of Lamazym for the treatment of patients with alpha-mannosidosis.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Reduction of Oligosaccharides in urine
Secondary Outcome
Reduction of Oligosaccharides in serum
Condition
Alpha Mannosidosis
Intervention
Lamazym
Study Arms / Comparison Groups
Lamazym 25
Description: 25 U/kg
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
10
Start Date
January 2011
Completion Date
November 2012
Primary Completion Date
January 2012
Eligibility 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 ≤ 21 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
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
Gender
All
Ages
5 Years - 21 Years
Accepts Healthy Volunteers
No
Contacts
Allan M. Lund, MD, ,
Location Countries
Denmark
Location Countries
Denmark
Administrative Informations
NCT ID
NCT01285700
Organization ID
rhLAMAN-03
Secondary IDs
2010-022085-26
Responsible Party
Sponsor
Study Sponsor
Zymenex A/S
Collaborators
European Commission
Study Sponsor
Allan M. Lund, MD, Principal Investigator, Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark
Verification Date
September 2012