Lamazym Aftercare Study FR (rhLAMAN-07)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT01908712
First received: July 16, 2013
Last updated: July 23, 2013
Last verified: July 2013
  Purpose

The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.


Condition Intervention Phase
Alpha-Mannosidosis
Drug: Lamazym
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials

Resource links provided by NLM:


Further study details as provided by Zymenex A/S:

Primary Outcome Measures:
  • Change from baseline in Adverse events [ Time Frame: 3 year ] [ Designated as safety issue: Yes ]
    AE documented at every visit

  • Change from baseline in Lamazym antibodies [ Time Frame: 3 year ] [ Designated as safety issue: Yes ]
    AB measured every 12th week


Secondary Outcome Measures:
  • progress from baseline in number of steps climbed in 3 minutes [ Time Frame: 1 year, 2 year and 3 year ] [ Designated as safety issue: No ]
  • Progress from baseline in equivalent age [ Time Frame: 1 year, 2 year and 3 year ] [ Designated as safety issue: No ]
  • Progress from baseline in Forced Vital Capacity [ Time Frame: 1 year, 2 year and 3 year ] [ Designated as safety issue: No ]
  • Progress from baseline in distance walked in 6 minutes [ Time Frame: 1 year, 2 year and 3 year ] [ Designated as safety issue: No ]

Enrollment: 7
Study Start Date: August 2013
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lamazym
1 mg Lamazym/kg body weight
Drug: Lamazym
ERT, i.v. infusions weekly
Other Names:
  • rhLAMAN
  • recombinant human alpha-mannosidase

Detailed Description:

This trial provide aftercare treatment with Lamazym to French patients.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • the subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
  • 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

Exclusion Criteria:

  • 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 circumstances that, in the opinion of the Investigator, would preclude participation in the trial
  • Pregnancy. Pregnant woman is excluded. Before start fot he 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
  • 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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01908712

Locations
France
Hôpital Femme Mére Enfant - CHU de Lyon
Bron, France, F-69677
Sponsors and Collaborators
Zymenex A/S
Investigators
Principal Investigator: Allan M Lund, MD Copenhagen University Hospital, Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre
  More Information

Additional Information:
No publications provided

Responsible Party: Zymenex A/S
ClinicalTrials.gov Identifier: NCT01908712     History of Changes
Other Study ID Numbers: rhLAMAN-07, 2013-000336-97
Study First Received: July 16, 2013
Last Updated: July 23, 2013
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

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 September 18, 2014