Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Study Identifier:
rhLAMAN-04
ClinicalTrials.gov Identifier:
NCT01681940
EudraCT Identifier:
2011-004355-40
EU CT ID:
N/A
Study Contact Information:
N/A
Study Complete

Trial Documents

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

Study Details

Medical Condition
  • Alpha-mannosidosis
Study Drug
  • Drug: Lamazym
Date
Jan 2012 - Aug 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 subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
  • Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
  • The subject and his/her guardian(s) must have the ability to comply with the protocol
Exclusion Criteria
  • The subject 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
  • 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: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study
  • Psychosis within the last 3 months
  • Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
  • Participation in other interventional trials testing IMP except for studies with Lamazym
Healthy Volunteers
No

Protocol Summary

The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis

Study Locations

Location
Status
Location
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
Brussels, Belgium, 1090
Status
N/A
Location
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark, DK-2100
Status
N/A
Location
Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
Córdoba, Spain, 14004
Status
N/A
Location
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, United Kingdom, M13 9WL
Status
N/A