Lamazym Aftercare Study (rhLAMAN-09)

July 26, 2023 updated by: Chiesi Farmaceutici S.p.A.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This protocol only concern subjects 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

Enrollment (Actual)

8

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Denmark
      • Copenhagen, Denmark, DK-2100
        • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in Adverse events
Time Frame: 3 year
AE documented at all visits
3 year
Change from baseline in Lamazym antibodies
Time Frame: 3 year
AB measured every 12th week
3 year

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chiesi Farmaceutici S.p.A.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2013

Primary Completion (Actual)

September 30, 2022

Study Completion (Actual)

September 30, 2022

Study Registration Dates

First Submitted

June 12, 2013

First Submitted That Met QC Criteria

July 23, 2013

First Posted (Estimated)

July 26, 2013

Study Record Updates

Last Update Posted (Actual)

July 28, 2023

Last Update Submitted That Met QC Criteria

July 26, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • rhLAMAN-09
  • 2013-000321-31 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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