Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
- Registration Number
- NCT01681940
- Lead Sponsor
- Zymenex A/S
- 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Lamazym Lamazym 1 mg/kg body weight
- Primary Outcome Measures
Name Time Method Reduction of Oligosaccharides in blood serum 6 months Efficacy endpoint evaluation as change from baseline
- Secondary Outcome Measures
Name Time Method Reduction of Oligosaccharides in CSF 6 months Efficacy endpoint evaluation as change from baseline
Adverse events 1 week Safety endpoint assesed weekly throughout the trial
Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) 1 week Safety endpoint assesed weekly throughout the trial
The number of steps climbed in 3 minutes (3-minute stair climb) 6 months Efficacy endpoint evaluation as change from baseline
The distance walked in 6 minutes (6-minute walk test) 6 months Efficacy endpoint evaluation as change from baseline
Development of clinically significant changes in vital signs and change in physical examination 1 week Safety endpoint assesed weekly throughout the trial
Pulmonary function 6 months Efficacy endpoint evaluation as change from baseline
Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies 1 week Safety endpoint assesed weekly throughout the trial
Trial Locations
- Locations (4)
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
🇩🇰Copenhagen, Denmark
Kinderneurologie Metabole Ziekten, UZ Brussel, Laarbeeklaan 101
🇧🇪Brussel, Belgium
Servicio de PediatrÃa, Hospital Materno Infantil, Reina SofÃa, Avda Menéndez Pidal sn
🇪🇸Córdoba, Spain
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
🇬🇧Manchester, United Kingdom