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Lamazym Aftercare Study (rhLAMAN-09)

Primary Purpose

Alpha-Mannosidosis

Status
Completed
Phase
Phase 3
Locations
Denmark
Study Type
Interventional
Intervention
Lamazym
Sponsored by
Chiesi Farmaceutici S.p.A.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha-Mannosidosis

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lamazym

Arm Description

1 mg Lamazym/kg body weight

Outcomes

Primary Outcome Measures

Change from baseline in Adverse events
AE documented at all visits
Change from baseline in Lamazym antibodies
AB measured every 12th week

Secondary Outcome Measures

progress from baseline in number of steps climbed in 3 minutes
Progress from baseline in equivalent age
Progress from baseline in Forced Vital Capacity
Progress from baseline in distance walked in 6 minutes

Full Information

First Posted
June 12, 2013
Last Updated
July 26, 2023
Sponsor
Chiesi Farmaceutici S.p.A.
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1. Study Identification

Unique Protocol Identification Number
NCT01908725
Brief Title
Lamazym Aftercare Study
Acronym
rhLAMAN-09
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 Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
June 2013 (undefined)
Primary Completion Date
September 30, 2022 (Actual)
Study Completion Date
September 30, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chiesi Farmaceutici S.p.A.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha-Mannosidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Lamazym
Arm Type
Experimental
Arm Description
1 mg Lamazym/kg body weight
Intervention Type
Drug
Intervention Name(s)
Lamazym
Other Intervention Name(s)
rhLAMAN, recombinant human alpha-mannosidase
Intervention Description
ERT, i.v. infusions weekly
Primary Outcome Measure Information:
Title
Change from baseline in Adverse events
Description
AE documented at all visits
Time Frame
3 year
Title
Change from baseline in Lamazym antibodies
Description
AB measured every 12th week
Time Frame
3 year
Secondary Outcome Measure Information:
Title
progress from baseline in number of steps climbed in 3 minutes
Time Frame
1 year, 2 year and 3 year
Title
Progress from baseline in equivalent age
Time Frame
1 year, 2 year and 3 year
Title
Progress from baseline in Forced Vital Capacity
Time Frame
1 year, 2 year and 3 year
Title
Progress from baseline in distance walked in 6 minutes
Time Frame
1 year, 2 year and 3 year

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
City
Copenhagen
ZIP/Postal Code
DK-2100
Country
Denmark

12. IPD Sharing Statement

Links:
URL
http://www.google.dk/url?sa=t&rct=j&q=line%20borgwardt&source=web&cd=1&cad=rja&sqi=2&ved=0CCsQtwIwAA&url=http%3A%2F%2Fwww.youtube.com%2Fwatch%3Fv%3DZGgqK7oJQFQ&ei=4TG4UejWD8SSO7TqgbAI&usg=AFQjCNG4azVFYvyGp-yNBeoP4tG6YQQ1Tw&bvm=bv.47810305,d.ZWU
Description
12th international symposium on MPS and related diseases
URL
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-000321-31/results
Description
Results posted in EudraCT

Learn more about this trial

Lamazym Aftercare Study

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