A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Zymenex A/S4 sites in 4 countries10 target enrollmentJanuary 2012

ConditionsAlpha-Mannosidosis

InterventionsLamazym

DrugsLamazym

Overview

Phase: Phase 2
Intervention: Lamazym
Conditions: Alpha-Mannosidosis
Sponsor: Zymenex A/S
Enrollment: 10
Locations: 4
Primary Endpoint: Reduction of Oligosaccharides in blood serum
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis

Registry

clinicaltrials.gov

Start Date

January 2012

End Date

September 2013

Last Updated

9 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Zymenex A/S

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•The subject must have participated in the phase 1 trial (EudraCT number: 2010-022084-36) and phase 2a trial (EudraCT number: 2010-022085-26)
•Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
•The subject and his/her guardian(s) must have the ability to comply with the protocol

Exclusion Criteria

•The subject cannot walk without support
•Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
•History of bone marrow transplantation
•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 circumstance that, in the opinion of the investigator, would preclude participation in the trial.
•Pregnancy: Before the start of the treatment the investigators will decide whether or not there is a need for contraception. This assessment will be done through interviews with the patient and parents. The evaluation will be done continuously during the study
•Psychosis within the last 3 months
•Planned major surgery that, in the opinion of the investigator, would preclude participation in the trial
•Participation in other interventional trials testing IMP except for studies with Lamazym

Arms & Interventions

Lamazym

1 mg/kg body weight

Intervention: Lamazym

Outcomes

Primary Outcomes

Reduction of Oligosaccharides in blood serum

Time Frame: 6 months

Efficacy endpoint evaluation as change from baseline

Secondary Outcomes

Reduction of Oligosaccharides in CSF(6 months)
Adverse events(1 week)
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)(1 week)
Development of clinically significant changes in vital signs and change in physical examination(1 week)
The number of steps climbed in 3 minutes (3-minute stair climb)(6 months)
The distance walked in 6 minutes (6-minute walk test)(6 months)
Pulmonary function(6 months)
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies(1 week)