A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
- Registration Number
- NCT01681953
- Lead Sponsor
- Zymenex A/S
- Brief Summary
The overall objective of this trial is to evaluate the efficacy and safety of repeated Lamazym i.v. treatment, compared with placebo, in subjects 5-35 years of age with alpha-Mannosidosis
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 25
- Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
- The subject and his/her guardian(s) must have the ability to comply with the protocol
- The subject must have a confirmed diagnosis of alpha-Mannosidosis as defined by alpha-Mannosidase activity < 10% of normal activity (historical data)
- The subject must have an age at the time of screening ≥ 5 years and ≤ 35 years
- The subject must have the ability to physically and mentally cooperate in the tests
- The subject must have an ECHO without abnormalities that, in the opinion of the Investigator, would preclude participation in the trial
- The subjects diagnosis cannot be confirmed by alpha-Mannosidase activity < 10% of normal activity
- The subject cannot walk without support
- Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-Mannosidosis
- History of BMT
- 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: Pregnant woman is excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
- Psychosis; any psychotic disease, also in remission, is an exclusion criteria
- Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
- Participation in other interventional trials testing IMP (including Lamazym) within the last 3 months
- Adult patients who, in the opinion of the Investigator, would be unable to give consent, and who does not have any legal protection or guardianship
- Total IgE >800 IU/ml
- Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Lamazym Lamazym 1 mg Lamazym/kg body weight Placebo Placebo Placebo is formulated as an isotonic phosphate buffer with glycine and mannitol
- Primary Outcome Measures
Name Time Method The number of steps climbed in 3 minutes (3-minute stair climb test) Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
Reduction of oligosaccharides in serum Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
- Secondary Outcome Measures
Name Time Method Development of clinically significant changes in vital signs and change in physical examination 1 week Safety endpoints assessed weekly throughout the trial
Clinical laboratory parameters (hematology, biochemistry and urinalysis) 1 week Safety endpoints assessed weekly throughout the trial
Forced Vital Capacity Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
The distance walked in 6 minutes (6-minute walk test) Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
Development of Lamazym antibodies and neutralizing/inhibitory antibodies 1 week Safety endpoints assessed weekly throughout the trial
Adverse Events 1 week Safety endpoint assessed weekly throughout the trial
Trial Locations
- Locations (6)
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9
🇩🇰Copenhagen, Denmark
Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin, Langenbeckstrasse 1
🇩🇪Mainz, Germany
Hôpital Femme Mère Enfant, Lyon, 59 boulevard Pinel
🇫🇷Bron, France
The Children's Memorial Health Institute Warsaw, Department of Metabolic Diseases, Al Dzieci Polskich 20
🇵🇱Warszawa, Poland
Genetic Medicine, 6th floor, St Mary's Hospital, Oxford Road,
🇬🇧Manchester, United Kingdom
Hôpital Trousseau, Service de neuropédiatrie, Centre Référence des Maladies Lysosomales, 26 avenue du Docteur Arnold Netter
🇫🇷PARIS Cedex 12, France