A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Tr... | NCT01681953

NCT01681953

Protocol Section

Identification Module

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

ID	Type	Description	Link
2012-000979-17	EudraCT Number

Status Module

Name	Class
European Commission	OTHER

Oversight Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Label	Type	Description	Intervention Names
Lamazym	Active Comparator	1 mg Lamazym/kg body weight	Drug: Lamazym
Placebo	Placebo Comparator	Placebo is formulated as an isotonic phosphate buffer with glycine and mannitol	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Lamazym	Drug	ERT, i.v. infusions weekly	Lamazym	rhLAMAN

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Reduction of oligosaccharides in serum	Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group	Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks
The number of steps climbed in 3 minutes (3-minute stair climb test)	Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group	Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks

Secondary Outcomes

Measure	Description	Time Frame
Forced Vital Capacity	Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group	Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks
The distance walked in 6 minutes (6-minute walk test)

Other Outcomes

Measure	Description	Time Frame
Quantitative determination of rhLAMAN in plasma	Pharmacokinetic (PK) assessments. Blood samples are drawn pre-treatment and at various times post-treatment (see time frame above)	10 min, 60 min, 2 hours, 24 hours, 3 days, 7 days

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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)

Name	Affiliation	Role
Allan M Lund, MD	Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics	Principal Investigator
Jens Fogh	Zymenex A/S	Study Chair

Facility	Status	City	State	ZIP	Country	Contacts
Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9		Copenhagen		DK-2100	Denmark

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
26048034	Background	Borgwardt L, Stensland HM, Olsen KJ, Wibrand F, Klenow HB, Beck M, Amraoui Y, Arash L, Fogh J, Nilssen O, Dali CI, Lund AM. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation. Orphanet J Rare Dis. 2015 Jun 6;10:70. doi: 10.1186/s13023-015-0286-x.
26016802	Background

ID	Term
D044904	Mannosidase Deficiency Diseases
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn