Clinical Trial: Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-<

Brief Summary: This is a single-center, open-label, multiple-dose study of the efficacy and long-term safety of Lamazym for the treatment of patients with alpha-mannosidosis.

Detailed Summary:
Sponsor: Zymenex A/S

Current Primary Outcome: Reduction of Oligosaccharides in urine [ Time Frame: 3 months (interim evaluation) + 6 months ]

Efficacy endpoint evaluation as change from baseline


Original Primary Outcome:

  • To evaluate the efficacy endpoints [ Time Frame: 6 months ]
    The efficacy endpoints (Oligosaccharide level in urine, serum and CSF, Gait, 6MWT, 3MSC, FVC, MRS) are evaluated to identify which endpoint will be used in following clinical studies as the primary endpoint
  • Dose finding study of repeated dose i.v. treatment of Lamazym [ Time Frame: 6 ]
    To select the optimal dose for repeated dose i.v. treatment of Lamazym


Current Secondary Outcome:

  • Reduction of Oligosaccharides in serum [ Time Frame: 3 months (interim evaluation) + 6 months ]
    Efficacy endpoint evaluation as change from baseline
  • Reduction of Oligosaccharides in CSF [ Time Frame: 3 months (interim evaluation) + 6 months ]
    Efficacy endpoint evaluation as change from baseline
  • The distance walked in 6 minutes [ Time Frame: 3 months (interim evaluation) + 6 months ]
    Efficacy endpoint evaluation as change from baseline
  • The number of steps climbed in 3 minutes [ Time Frame: 3 months (interim evaluation) + 6 months ]
    Efficacy endpoint evaluation as change from baseline
  • Pulmonary Function [ Time Frame: 3 months (interim evaluation) + 6 months ]
    Efficacy endpoint evaluation as change from baseline
  • Adverse events [ Time Frame: 1 week ]
    Safety endpoint assessed weekly throughout the trial
  • Development of clinically significant changes in vital signs and change in physical examination [ Time Frame: 1 week ]
    Safety endpoint assessed weekly throughout the trial
  • Development of clinically significant changes in the clinical laboratory parameters (hematology, biochemistry and urinalysis) [ Time Frame: 4 weeks ]
    Safety endpoint assessed every 4th week throughout the trial
  • Development of rhLAMAN antibodies and neutralizing/inhibitory antibodies [ Time Frame: 2 weeks ]
    Safety endpoint assessed every other week throughout the trial


Original Secondary Outcome: To evaluate the long-term safety and tolerability of repeated dose i.v. treatment of Lamazym [ Time Frame: 6 months ]

Information By: Zymenex A/S

Dates:
Date Received: January 25, 2011
Date Started: January 2011
Date Completion: November 2012
Last Updated: September 25, 2012
Last Verified: September 2012