A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

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