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Lamazym Aftercare Study (rhLAMAN-09)

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ClinicalTrials.gov Identifier: NCT01908725
Recruitment Status : Active, not recruiting
First Posted : July 26, 2013
Last Update Posted : December 22, 2017
Sponsor:
Information provided by (Responsible Party):
Chiesi Farmaceutici S.p.A.

Brief Summary:
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 or disease Intervention/treatment Phase
Alpha-Mannosidosis Drug: Lamazym Phase 3

Detailed Description:
This protocol only concern subjects from Poland, where compassionate use program was not accepted. Efficacy will be evaluated once yearly to follow the subject's progress in clinical parameters.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : June 2013
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Lamazym
1 mg Lamazym/kg body weight
Drug: Lamazym
ERT, i.v. infusions weekly
Other Names:
  • rhLAMAN
  • recombinant human alpha-mannosidase



Primary Outcome Measures :
  1. Change from baseline in Adverse events [ Time Frame: 3 year ]
    AE documented at all visits

  2. Change from baseline in Lamazym antibodies [ Time Frame: 3 year ]
    AB measured every 12th week


Secondary Outcome Measures :
  1. progress from baseline in number of steps climbed in 3 minutes [ Time Frame: 1 year, 2 year and 3 year ]
  2. Progress from baseline in equivalent age [ Time Frame: 1 year, 2 year and 3 year ]
  3. Progress from baseline in Forced Vital Capacity [ Time Frame: 1 year, 2 year and 3 year ]
  4. Progress from baseline in distance walked in 6 minutes [ Time Frame: 1 year, 2 year and 3 year ]


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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01908725


Locations
Denmark
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
Copenhagen, Denmark, DK-2100
Sponsors and Collaborators
Chiesi Farmaceutici S.p.A.

Additional Information:
Responsible Party: Chiesi Farmaceutici S.p.A.
ClinicalTrials.gov Identifier: NCT01908725     History of Changes
Other Study ID Numbers: rhLAMAN-09
2013-000321-31 ( EudraCT Number )
First Posted: July 26, 2013    Key Record Dates
Last Update Posted: December 22, 2017
Last Verified: December 2017

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