Overview

This trial is active, not recruiting.

Condition alpha-mannosidosis
Treatment lamazym
Phase phase 3
Sponsor Zymenex A/S
Start date June 2013
End date December 2018
Trial size 5 participants
Trial identifier NCT01908725, 2013-000321-31, rhLAMAN-09

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
1 mg Lamazym/kg body weight
lamazym rhLAMAN
ERT, i.v. infusions weekly

Primary Outcomes

Measure
Change from baseline in Adverse events
time frame: 3 year
Change from baseline in Lamazym antibodies
time frame: 3 year

Secondary Outcomes

Measure
progress from baseline in number of steps climbed in 3 minutes
time frame: 1 year, 2 year and 3 year
Progress from baseline in equivalent age
time frame: 1 year, 2 year and 3 year
Progress from baseline in Forced Vital Capacity
time frame: 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

Eligibility Criteria

Male or female participants of any age.

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

Additional Information

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
Principal investigator Allan M Lund, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Chiesi Farmaceutici S.p.A..