Brief Title
A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
Official Title
A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis.
Brief Summary
The overall objective of this trial is to evaluate the efficacy and safety of repeated
Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with
alpha-Mannosidosis
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Reduction of oligosaccharides in serum
Secondary Outcome
Forced Vital Capacity
Condition
Alpha-Mannosidosis
Intervention
Lamazym
Study Arms / Comparison Groups
Lamazym
Description: 1 mg Lamazym/kg body weight
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
25
Start Date
August 2012
Completion Date
May 2014
Primary Completion Date
May 2014
Eligibility Criteria
Inclusion Criteria:
- 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
- The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by
alpha-Mannosidase activity < 10% of normal activity (historical data)
- The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
- The subject must have the ability to physically and mentally cooperate in the tests
- The subject must have an ECHO without abnormalities that, in the opinion of the
Investigator, would preclude participation in the trial
Exclusion Criteria:
- The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity < 10% of
normal activity
- The subject cannot walk without support
- Presence of known chromosomal abnormality and syndromes affecting psychomotor
development, other than alpha-Mannosidosis
- History of BMT
- 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: Pregnant woman is excluded. Before start of the 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 exclusion criteria
- Planned major surgery that, in the opinion of the Investigator, would preclude
participation in the trial
- Participation in other interventional trials testing IMP (including Lamazym) within
the last 3 months
- Adult patients who, in the opinion of the Investigator, would be unable to give
consent, and who does not have any legal protection or guardianship
- Total IgE >800 IU/ml
- Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
Gender
All
Ages
5 Years - 35 Years
Accepts Healthy Volunteers
No
Contacts
Allan M Lund, MD, ,
Location Countries
Denmark
Location Countries
Denmark
Administrative Informations
NCT ID
NCT01681953
Organization ID
rhLAMAN-05
Secondary IDs
2012-000979-17
Responsible Party
Sponsor
Study Sponsor
Zymenex A/S
Collaborators
European Commission
Study Sponsor
Allan M Lund, MD, Principal Investigator, Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Verification Date
July 2020