Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Brief Title

Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Official Title

A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Brief Summary

      The overall objective is to evaluate the long-term efficacy, safety and tolerability of
      repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Reduction of Oligosaccharides in blood serum

Secondary Outcome

 The number of steps climbed in 3 minutes (3-minute stair climb)

Condition

Alpha-Mannosidosis

Intervention

Lamazym

Study Arms / Comparison Groups

 Lamazym
Description:  1 mg/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

10

Start Date

January 2012

Completion Date

September 2013

Primary Completion Date

August 2012

Eligibility 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

        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: Before the start of the treatment the investigators will decide whether or
             not there is a need for contraception. This assessment will be done through interviews
             with the patient and parents. The evaluation will be done continuously during the
             study

          -  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
      

Gender

All

Ages

5 Years - 21 Years

Accepts Healthy Volunteers

No

Contacts

Allan M Lund, MD, , 

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations


NCT ID

NCT01681940

Organization ID

rhLAMAN-04

Secondary IDs

2011-004355-40

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

March 2017