Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

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