Trial record 7 of 15 for:    "alpha-Mannosidosis" OR "alpha-mannosidosis"

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
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 April 21, 2014