Clinical Trial: Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-This is a single-center, open-label, dose escalation study of patients with alpha-mannosidosis. 10 patients will be enrolled in this study receiving intravenous infusions of Lamazym. In order to avoid development of delayed hypersensitivity all patients will continue weekly treatment at the designated dose until the Safety Committee approves transfer to the rhLAMAN-03 protocol.
It is the hypothesis that Lamazym is safe to use.
Detailed Summary:
Sponsor: Zymenex A/S
Current Primary Outcome: To evaluate the safety profile of rhLAMAN (Lamazym) [ Time Frame: 1-5 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- To determine the PK profile of rhLAMAN (Lamazym) in patients with alpha-mannosidosis as measured by rhLAMAN levels in plasma [ Time Frame: 1 dosis ]
- To collect baseline measurements that are to be used for efficacy evaluation in the following trial (rhLAMAN-03) [ Time Frame: 1 week ]
Original Secondary Outcome: Same as current
Information By: Chiesi Farmaceutici S.p.A.
Dates:
Date Received: December 29, 2010
Date Started: October 2010
Date Completion:
Last Updated: March 28, 2017
Last Verified: March 2017
