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

This study has been completed.
Sponsor:
Collaborator:
European Commission
Information provided by:
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT01268358
First received: December 29, 2010
Last updated: May 6, 2011
Last verified: May 2011
History of Changes
  Purpose

This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.

It is the hypothesis that Lamazym is safe to use.


Condition Intervention Phase
Alpha Mannosidosis
 Drug: Lamazym
 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics 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:
  • To evaluate the safety profile of rhLAMAN (Lamazym) [ Time Frame: 1-5 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma [ Time Frame: 1 dosis ] [ Designated as safety issue: No ]
  • To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03) [ Time Frame: 1 week ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: October 2010
Study Completion Date: January 2011
Primary Completion Date: January 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lamazym 6.25 Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Name: rhLAMAN
Experimental: Lamazym 12.5 Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Name: rhLAMAN
Experimental: Lamazym 25 Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Name: rhLAMAN
Experimental: Lamazym 50 Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Name: rhLAMAN
Experimental: Lamazym 100 Drug: Lamazym
Lamazym, ERT, infusion weekly
Other Name: rhLAMAN

  Eligibility

Ages Eligible for Study:   5 Years to 20 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 ≤ 20 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: NCT01268358

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: Allan Meldgaard lund, MD, DMSc, M, Department of Clinical Genetics, Juliane Marie Centre, Region Hovedstaden, Copenhagen University hospital, Denmark
ClinicalTrials.gov Identifier: NCT01268358     History of Changes
Other Study ID Numbers: rhLAMAN-02, 2010-022084-36
Study First Received: December 29, 2010
Last Updated: May 6, 2011
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