Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

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

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

Study Overview

• Status: Completed
• Conditions: Alpha-Mannosidosis
• Intervention / Treatment: Drug: Lamazym

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brussel, Belgium, 1090
    • Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
• Denmark
  • Copenhagen, Denmark, DK-2100
    • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
• Spain
  • Córdoba, Spain, 14004
    • Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
• United Kingdom
  • Manchester, United Kingdom, M13 9WL
    • Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Lamazym; 1 mg/kg body weight	Drug: Lamazym; ERT, i.v. infusions weekly; Other Names: rhLAMAN; recombinant human alpha-mannosidase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction of Oligosaccharides in blood serum	Efficacy endpoint evaluation as change from baseline	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of steps climbed in 3 minutes (3-minute stair climb)	Efficacy endpoint evaluation as change from baseline	6 months
Reduction of Oligosaccharides in CSF	Efficacy endpoint evaluation as change from baseline	6 months
The distance walked in 6 minutes (6-minute walk test)	Efficacy endpoint evaluation as change from baseline	6 months
Pulmonary function	Efficacy endpoint evaluation as change from baseline	6 months
Adverse events	Safety endpoint assesed weekly throughout the trial	1 week
Development of clinically significant changes in vital signs and change in physical examination	Safety endpoint assesed weekly throughout the trial	1 week
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)	Safety endpoint assesed weekly throughout the trial	1 week
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies	Safety endpoint assesed weekly throughout the trial	1 week

Collaborators and Investigators

• Sponsor: Zymenex A/S
• Collaborators: European Commission
• Investigators: Principal Investigator: Allan M Lund, MD, Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics; Study Chair: Jens Fogh, Zymenex A/S

Publications and helpful links

Helpful Links

• Study Record on EU Clinical Trials Register including results

Study record dates

Study Major Dates

• Study Start: January 1, 2012
• Primary Completion (Actual): August 1, 2012
• Study Completion (Actual): September 1, 2013

Study Registration Dates

• First Submitted: August 22, 2012
• First Submitted That Met QC Criteria: September 5, 2012
• First Posted (Estimate): September 10, 2012

Study Record Updates

• Last Update Posted (Actual): March 29, 2017
• Last Update Submitted That Met QC Criteria: March 28, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mannosidase Deficiency Diseases; alpha-Mannosidosis
• Other Study ID Numbers: rhLAMAN-04; 2011-004355-40 (EudraCT Number)