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

Alpha-Mannosidosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01681940
• Other study ID #: rhLAMAN-04
• Secondary ID: 2011-004355-40
• Status: Completed
• Phase: Phase 2
• First received: August 22, 2012
• Last updated: March 28, 2017
• Start date: January 2012
• Est. completion date: September 2013

Study information

• Verified date: March 2017
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: September 2013
• Est. primary completion date: August 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Alpha-Mannosidosis

Intervention

Drug:
• Lamazym
ERT, i.v. infusions weekly

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
Zymenex A/S	European Commission

Countries where clinical trial is conducted

Belgium,  Denmark,  Spain,  United Kingdom, 

Outcome

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

External Links

•   Study Record on EU Clinical Trials Register including results