Lamazym Aftercare Study

Alpha-Mannosidosis Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01908725
• Other study ID #: rhLAMAN-09
• Secondary ID: 2013-000321-31
• Status: Completed
• Phase: Phase 3
• Start date: June 2013
• Est. completion date: September 30, 2022

Study information

• Verified date: July 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: September 30, 2022
• Est. primary completion date: September 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Alpha-Mannosidosis

Intervention

Drug:
• Lamazym
ERT, i.v. infusions weekly

Locations

Country	Name	City	State
Denmark	Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9	Copenhagen

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in Adverse events	AE documented at all visits	3 year
Primary	Change from baseline in Lamazym antibodies	AB measured every 12th week	3 year
Secondary	progress from baseline in number of steps climbed in 3 minutes		1 year, 2 year and 3 year
Secondary	Progress from baseline in equivalent age		1 year, 2 year and 3 year
Secondary	Progress from baseline in Forced Vital Capacity		1 year, 2 year and 3 year
Secondary	Progress from baseline in distance walked in 6 minutes		1 year, 2 year and 3 year

External Links

•   12th international symposium on MPS and related diseases
•   Results posted in EudraCT