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

Primary Purpose

Alpha-Mannosidosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Lamazym
Sponsored by
Zymenex A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alpha-Mannosidosis

Eligibility Criteria

5 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
  • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
  • Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
  • Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lamazym

Arm Description

1 mg/kg body weight

Outcomes

Primary Outcome Measures

Reduction of Oligosaccharides in blood serum
Efficacy endpoint evaluation as change from baseline

Secondary Outcome Measures

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

Full Information

First Posted
August 22, 2012
Last Updated
March 28, 2017
Sponsor
Zymenex A/S
Collaborators
European Commission
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1. Study Identification

Unique Protocol Identification Number
NCT01681940
Brief Title
Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
Official Title
A Multi-Center, Open-Label Trial of the Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
Study Type
Interventional

2. Study Status

Record Verification Date
March 2017
Overall Recruitment Status
Completed
Study Start Date
January 2012 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
September 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zymenex A/S
Collaborators
European Commission

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha-Mannosidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Lamazym
Arm Type
Experimental
Arm Description
1 mg/kg body weight
Intervention Type
Drug
Intervention Name(s)
Lamazym
Other Intervention Name(s)
rhLAMAN, recombinant human alpha-mannosidase
Intervention Description
ERT, i.v. infusions weekly
Primary Outcome Measure Information:
Title
Reduction of Oligosaccharides in blood serum
Description
Efficacy endpoint evaluation as change from baseline
Time Frame
6 months
Secondary Outcome Measure Information:
Title
The number of steps climbed in 3 minutes (3-minute stair climb)
Description
Efficacy endpoint evaluation as change from baseline
Time Frame
6 months
Title
Reduction of Oligosaccharides in CSF
Description
Efficacy endpoint evaluation as change from baseline
Time Frame
6 months
Title
The distance walked in 6 minutes (6-minute walk test)
Description
Efficacy endpoint evaluation as change from baseline
Time Frame
6 months
Title
Pulmonary function
Description
Efficacy endpoint evaluation as change from baseline
Time Frame
6 months
Title
Adverse events
Description
Safety endpoint assesed weekly throughout the trial
Time Frame
1 week
Title
Development of clinically significant changes in vital signs and change in physical examination
Description
Safety endpoint assesed weekly throughout the trial
Time Frame
1 week
Title
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
Description
Safety endpoint assesed weekly throughout the trial
Time Frame
1 week
Title
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
Description
Safety endpoint assesed weekly throughout the trial
Time Frame
1 week

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Allan M Lund, MD
Organizational Affiliation
Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Jens Fogh
Organizational Affiliation
Zymenex A/S
Official's Role
Study Chair
Facility Information:
Facility Name
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
City
Brussel
ZIP/Postal Code
1090
Country
Belgium
Facility Name
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
City
Copenhagen
ZIP/Postal Code
DK-2100
Country
Denmark
Facility Name
Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
City
Córdoba
ZIP/Postal Code
14004
Country
Spain
Facility Name
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Links:
URL
https://www.clinicaltrialsregister.eu/ctr-search/search?query=2011-004355-40
Description
Study Record on EU Clinical Trials Register including results

Learn more about this trial

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

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