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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
European Commission
Information provided by (Responsible Party):
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT01285700
First received: January 25, 2011
Last updated: September 25, 2012
Last verified: September 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Alpha Mannosidosis
 Drug: Lamazym
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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.

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Zymenex A/S:

Primary Outcome Measures:
  • Reduction of Oligosaccharides in urine [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline


Secondary Outcome Measures:
  • Reduction of Oligosaccharides in serum [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Reduction of Oligosaccharides in CSF [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • The distance walked in 6 minutes [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • The number of steps climbed in 3 minutes [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Pulmonary Function [ Time Frame: 3 months (interim evaluation) + 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Adverse events [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assessed weekly throughout the trial

  • Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assessed weekly throughout the trial

  • Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
    Safety endpoint assessed every 4th week throughout the trial

  • Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies [ Time Frame: 2 weeks ] [ Designated as safety issue: Yes ]
    Safety endpoint assessed every other week throughout the trial


Enrollment: 10
Study Start Date: January 2011
Estimated Study Completion Date: November 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Lamazym 25
25 U/kg
 Drug: Lamazym
ERT, infusion weekly
Other Name: rhLAMAN
Active Comparator: Lamazym 50
50 U/kg
 Drug: Lamazym
ERT, infusion weekly
Other Name: rhLAMAN

  Eligibility

Ages Eligible for Study:   5 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01285700

Locations
Denmark
Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Zymenex A/S
European Commission
Investigators
Principal Investigator: Allan M. Lund, MD Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark
Study Chair: Jens Fogh Zymenex A/S
  More Information

No publications provided

Responsible Party: Zymenex A/S
ClinicalTrials.gov Identifier: NCT01285700     History of Changes
Other Study ID Numbers: rhLAMAN-03, 2010-022085-26
Study First Received: January 25, 2011
Last Updated: September 25, 2012
Health Authority: Denmark: Danish Medicines Agency

Additional relevant MeSH terms:
Alpha-Mannosidosis
Mannosidase Deficiency Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013