A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

Study Identifier:
rhLAMAN-05
ClinicalTrials.gov Identifier:
NCT01681953
EudraCT Identifier:
2012-000979-17
EU CT ID:
N/A
Study Contact Information:
N/A
See Study Publication Below
Study Complete

Study Details

Medical Condition
  • Alphamanosidosis
Study Drug
  • Drug: Lamazym
  • Drug: Placebo
Date
Aug 2012 - May 2014
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5 - 35 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
  • The subject and his/her guardian(s) must have the ability to comply with the protocol
  • The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-Mannosidase activity \< 10% of normal activity (historical data)
  • The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
  • The subject must have the ability to physically and mentally cooperate in the tests
  • The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
  • Exclusion Criteria:
  • The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity \< 10% of normal activity
  • The subject cannot walk without support
  • Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
  • History of BMT
  • 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: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
  • Psychosis; any psychotic disease, also in remission, is an exclusion criteria
  • Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
  • Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
  • Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
  • Total IgE \>800 IU/ml
  • Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
Healthy Volunteers
No

Protocol Summary

The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis

Study Locations

Location
Status
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
Hôpital Femme Mère Enfant, Lyon, 59 boulevard Pinel
Bron, France, 69677
Status
N/A
Location
Hôpital Trousseau, Service de neuropédiatrie, Centre Référence des Maladies Lysosomales, 26 avenue du Docteur Arnold Netter
Paris, France, 75 571
Status
N/A
Location
Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin, Langenbeckstrasse 1
Mainz, Germany, 55131
Status
N/A
Location
The Children's Memorial Health Institute Warsaw, Department of Metabolic Diseases, Al Dzieci Polskich 20
Warsaw, Poland, 04 730
Status
N/A
Location
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, United Kingdom, M13 9WL
Status
N/A

Study Publications

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.Borgwardt L, Thuesen AM, Olsen KJ, Fogh J, Dali CI, Lund AM. Cognitive profile and activities of daily living: 35 patients with alpha-mannosidosis. J Inherit Metab Dis. 2015 Nov;38(6):1119-27. doi: 10.1007/s10545-015-9862-4. Epub 2015 May 28.Harmatz P, Cattaneo F, Ardigo D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis. Mol Genet Metab. 2018 Jun;124(2):152-160. doi: 10.1016/j.ymgme.2018.04.003. Epub 2018 Apr 18.Borgwardt L, Guffon N, Amraoui Y, Dali CI, De Meirleir L, Gil-Campos M, Heron B, Geraci S, Ardigo D, Cattaneo F, Fogh J, Van den Hout JMH, Beck M, Jones SA, Tylki-Szymanska A, Haugsted U, Lund AM. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial. J Inherit Metab Dis. 2018 Nov;41(6):1215-1223. doi: 10.1007/s10545-018-0185-0. Epub 2018 May 30.