Lamazym Aftercare Study

Phase 3

Completed

• Conditions: Alpha-Mannosidosis
• Interventions: Drug: Lamazym

• Registration Number: NCT01908725
• Lead Sponsor: 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.

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 Timeline

• Study Start: 2013-06
• Study First Submitted: 2013-06-12
• Study First Submitted QC: 2013-07-23
• Study First Posted: 2013-07-26
• Primary Completion: 2022-09-30
• Study Completion: 2022-09-30
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-26
• Last Update Posted: 2023-07-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Lamazym	Lamazym	1 mg Lamazym/kg body weight

Primary Outcome Measures

Name	Time	Method
Change from baseline in Adverse events	3 year	AE documented at all visits
Change from baseline in Lamazym antibodies	3 year	AB measured every 12th week

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9; 🇩🇰 Copenhagen, Denmark