The Natural History of Alpha-Mannosidosis

Study Identifier:
rhLAMAN-01
ClinicalTrials.gov Identifier:
NCT00498420
EudraCT Identifier:
N/A
EU CT ID:
N/A
Study Contact Information:
N/A
See Study Publication Below
Study Complete

Trial Documents

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

Study Details

Medical Condition
  • Alpha-mannosidosis
Study Drug
    Date
    May 2007 - Sep 2009
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • The patient (or patient's legal guardian) must provide written informed consent prior to performing any survey-related procedures.
    • The patient must have a documented diagnosis of Alpha Mannosidosis, confirmed at screening by measurable clinical signs and symptoms of Alpha Mannosidosis
    • Documented deficiency of serum or leukocyte acid alpha-mannosidase enzyme activity level
    Exclusion Criteria
    • History of bone marrow transplantation.
    • Use of an investigational drug within 30 days prior to study enrollment.
    • Known medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly decrease study compliance.
    Healthy Volunteers
    No

    Protocol Summary

    The natural history study of the rare lysosomal disease alpha-mannosidosis will answer the question; why the rare disease develops as it does?

    Study Locations

    Location
    Status
    Location
    Department of Pediatrics, Charles University
    Prague, Czech Republic, 12000 Prague
    Status
    N/A
    Location
    University of Mainz
    Mainz, Germany, 55101
    Status
    N/A
    Location
    Department of Medicine, University of Tromsoe
    Tromsø, Norway, N-9038
    Status
    N/A
    Location
    Willink Biochemical Genetics Unit,. Royal Manchester Children's Hospital
    Manchester, United Kingdom, M27 4HA
    Status
    N/A

    Study Publications

    Beck M, Olsen KJ, Wraith JE, Zeman J, Michalski JC, Saftig P, Fogh J, Malm D. Natural history of alpha mannosidosis a longitudinal study. Orphanet J Rare Dis. 2013 Jun 20;8:88. doi: 10.1186/1750-1172-8-88.Borgwardt L, Stensland HM, Olsen KJ, Wibrand F, Klenow HB, Beck M, Amraoui Y, Arash L, Fogh J, Nilssen O, Dali CI, Lund AM. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation. Orphanet J Rare Dis. 2015 Jun 6;10:70. doi: 10.1186/s13023-015-0286-x.