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

Study Identifier:
rhLAMAN-03
ClinicalTrials.gov Identifier:
NCT01285700
EudraCT Identifier:
2010-022085-26
EU CT ID:
N/A
Study Contact Information:
N/A
Unmapped

Trial Documents

Clinical Study Report
Available Languages: English
Download document(s)

Study Details

Medical Condition
  • Alpha-mannosidosis
Study Drug
  • Drug: Lamazym
Date
Jan 2011 - Jan 2012
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5 - 21 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • The patient must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-mannosidase activity \< 10% of normal activity in blood leukocytes
  • The patient must have an age at the time of screening ≥ 5 year and ≤ 21 years
  • 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).
  • The patient must have the ability to mentally cooperate in the cognitive and motor function tests
  • The patient must have the ability to hear and follow a request. Hearing aids can be worn.
  • 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)
  • The patient and his/her guardian(s) must have the ability to comply with the protocol
Exclusion Criteria
  • The patient 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
  • Presence of an ECHO with abnormalities within half a year 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
  • Psychosis within the last 3 months
Healthy Volunteers
No

Protocol 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 Locations

Location
Status
Location
Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark, 2100
Status
N/A