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Trial record 5 of 19 for:    "alpha-Mannosidosis" OR "alpha-mannosidosis"

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

This study has been completed.
European Commission
Information provided by (Responsible Party):
Chiesi Farmaceutici S.p.A. ( Zymenex A/S ) Identifier:
First received: December 29, 2010
Last updated: March 28, 2017
Last verified: March 2017

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: No masking
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:

Further study details as provided by Chiesi Farmaceutici S.p.A.:

Primary Outcome Measures:
  • To evaluate the safety profile of rhLAMAN (Lamazym) [ Time Frame: 1-5 weeks ]

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 ]
  • To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03) [ Time Frame: 1 week ]

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


Ages Eligible for Study:   5 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its identifier: NCT01268358

Sponsors and Collaborators
Zymenex A/S
European Commission
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

Additional Information:
Responsible Party: Zymenex A/S Identifier: NCT01268358     History of Changes
Other Study ID Numbers: rhLAMAN-02
2010-022084-36 ( EudraCT Number )
Study First Received: December 29, 2010
Last Updated: March 28, 2017

Additional relevant MeSH terms:
Mannosidase Deficiency Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases processed this record on April 27, 2017