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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
European Commission
Information provided by (Responsible Party):
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT01681940
First received: August 22, 2012
Last updated: September 5, 2012
Last verified: September 2012
History of Changes
  Purpose

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


Condition Intervention Phase
Alpha-Mannosidosis
 Drug: Lamazym
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Zymenex A/S:

Primary Outcome Measures:
  • Reduction of Oligosaccharides in blood serum [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline


Secondary Outcome Measures:
  • The number of steps climbed in 3 minutes (3-minute stair climb) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Reduction of Oligosaccharides in CSF [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • The distance walked in 6 minutes (6-minute walk test) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Pulmonary function [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Efficacy endpoint evaluation as change from baseline

  • Adverse events [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assesed weekly throughout the trial

  • Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assesed weekly throughout the trial

  • Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assesed weekly throughout the trial

  • Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assesed weekly throughout the trial


Enrollment: 9
Study Start Date: January 2012
Estimated Study Completion Date: April 2013
Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lamazym
1 mg/kg body weight
 Drug: Lamazym
ERT, i.v. infusions weekly
Other Names:
  • rhLAMAN
  • recombinant human alpha-mannosidase

  Eligibility

Ages Eligible for Study:   5 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01681940

Locations
Belgium
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
Brussel, Belgium, 1090
Denmark
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark, DK-2100
Spain
Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
Córdoba, Spain, 14004
United Kingdom
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Zymenex A/S
European Commission
Investigators
Principal Investigator: Allan M Lund, MD Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Study Chair: Jens Fogh Zymenex A/S
  More Information

No publications provided

Responsible Party: Zymenex A/S
ClinicalTrials.gov Identifier: NCT01681940     History of Changes
Other Study ID Numbers: rhLAMAN-04, 2011-004355-40
Study First Received: August 22, 2012
Last Updated: September 5, 2012
Health Authority: Denmark: Danish Medicines Agency
Germany: Federal Institute for Drugs and Medical Devices
United Kingdom: Medicines and Healthcare Products Regulatory Agency
France: L’Agence nationale de sécurité du médicament et des produits de santé
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Belgium: Federal Agency for Medicinal Products and Health Products

Additional relevant MeSH terms:
Alpha-Mannosidosis
Mannosidase Deficiency Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 22, 2013