Clinical trial NCT01681940: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alpha-Mannosidosis
Lead sponsor: Chiesi Farmaceutici S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: European Commission
First posted: 2012-09-10
Enrollment: 10
Sex: ALL
Minimum age (years): 5
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: Reduction of Oligosaccharides in blood serum
timeFrame: 6 months
Secondary outcomes:
measure: The number of steps climbed in 3 minutes (3-minute stair climb)
timeFrame: 6 months
measure: Reduction of Oligosaccharides in CSF
timeFrame: 6 months
measure: The distance walked in 6 minutes (6-minute walk test)
timeFrame: 6 months
measure: Pulmonary function
timeFrame: 6 months
measure: Adverse events
timeFrame: 1 week
measure: Development of clinically significant changes in vital signs and change in physical examination
timeFrame: 1 week
measure: Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis)
timeFrame: 1 week
measure: Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies
timeFrame: 1 week
Other outcomes:
Locations:
city: Brussel
country: Belgium
facility: Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
city: Copenhagen
country: Denmark
facility: Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
city: Córdoba
country: Spain
facility: Servicio de Pediatría, Hospital Materno Infantil, Reina Sofía, Avda Menéndez Pidal sn
city: Manchester
country: United Kingdom
facility: Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
Study officials:
affiliation: Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
name: Allan M Lund, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Zymenex A/S
name: Jens Fogh
role: STUDY_CHAIR
References: