A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

This study is enrolling participants by invitation only.
Sponsor:
Collaborator:
European Commission
Information provided by (Responsible Party):
Zymenex A/S
ClinicalTrials.gov Identifier:
NCT01681953
First received: August 22, 2012
Last updated: September 5, 2012
Last verified: September 2012

August 22, 2012
September 5, 2012
August 2012
October 2013   (final data collection date for primary outcome measure)
  • Reduction of oligosaccharides in serum [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ] [ Designated as safety issue: No ]
    Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
  • The number of steps climbed in 3 minutes (3-minute stair climb test) [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ] [ Designated as safety issue: No ]
    Primary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
Same as current
Complete list of historical versions of study NCT01681953 on ClinicalTrials.gov Archive Site
  • Forced Vital Capacity [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline evaluation prior to first dose, midterm evaluation after 26 weeks, and end evaluation after 52 weeks ] [ Designated as safety issue: No ]
    Secondary efficacy endpoint evaluated as change from baseline in the active group versus the placebo group
  • Adverse Events [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoint assessed weekly throughout the trial
  • Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoints assessed weekly throughout the trial
  • Clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoints assessed weekly throughout the trial
  • Development of Lamazym antibodies and neutralizing/inhibitory antibodies [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Safety endpoints assessed weekly throughout the trial
Same as current
Quantitative determination of rhLAMAN in plasma [ Time Frame: 10 min, 60 min, 2 hours, 24 hours, 3 days, 7 days ] [ Designated as safety issue: No ]
Pharmacokinetic (PK) assessments. Blood samples are drawn pre-treatment and at various times post-treatment (see time frame above)
Same as current
 
A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis
A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis.

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

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Alpha-Mannosidosis
  • Drug: Lamazym
    ERT, i.v. infusions weekly
    Other Names:
    • rhLAMAN
    • recombinant human alpha-mannosidase
  • Drug: Placebo
  • Active Comparator: Lamazym
    1 mg Lamazym/kg body weight
    Intervention: Drug: Lamazym
  • Placebo Comparator: Placebo
    Placebo is formulated as an isotonic phosphate buffer with glycine and mannitol
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Enrolling by invitation
20
March 2014
October 2013   (final data collection date for primary outcome measure)

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)
Both
5 Years to 35 Years
No
Contact information is only displayed when the study is recruiting subjects
Denmark,   United Kingdom,   France,   Poland,   Germany
 
NCT01681953
rhLAMAN-05, 2012-000979-17
Yes
Zymenex A/S
Zymenex A/S
European Commission
Principal Investigator: Allan M Lund, MD Copenhagen University Hospital, Center for Metabolic Diseases, Department for Clinical Genetics
Study Chair: Jens Fogh Zymenex A/S
Zymenex A/S
September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP