A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
NCT ID: NCT01681953
Last Updated: 2020-08-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
25 participants
INTERVENTIONAL
2012-08-31
2014-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Lamazym
1 mg Lamazym/kg body weight
Lamazym
ERT, i.v. infusions weekly
Placebo
Placebo is formulated as an isotonic phosphate buffer with glycine and mannitol
Placebo
Infusions weekly
Interventions
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Lamazym
ERT, i.v. infusions weekly
Placebo
Infusions weekly
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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
* 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)
5 Years
35 Years
ALL
No
Sponsors
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European Commission
OTHER
Zymenex A/S
INDUSTRY
Responsible Party
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Principal Investigators
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Allan M Lund, MD
Role: PRINCIPAL_INVESTIGATOR
Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Jens Fogh
Role: STUDY_CHAIR
Zymenex A/S
Locations
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Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, , Denmark
Hôpital Femme Mère Enfant, Lyon, 59 boulevard Pinel
Bron, , France
Hôpital Trousseau, Service de neuropédiatrie, Centre Référence des Maladies Lysosomales, 26 avenue du Docteur Arnold Netter
Paris, , France
Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin, Langenbeckstrasse 1
Mainz, , Germany
The Children's Memorial Health Institute Warsaw, Department of Metabolic Diseases, Al Dzieci Polskich 20
Warsaw, , Poland
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, , United Kingdom
Countries
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References
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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.
Related Links
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Study Record on EU Clinical Trials Register including results
Other Identifiers
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2012-000979-17
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
rhLAMAN-05
Identifier Type: -
Identifier Source: org_study_id
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