Clinical Trial: A Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Randomized, Multicenter, Open-label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophi
Brief Summary: This is a randomized, multicenter, open-label, Phase 3 clinical study in participants aged 12 years or older to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in participants with severe hemophilia A without inhibitors against FVIII.
Detailed Summary:
Sponsor: Hoffmann-La Roche
Current Primary Outcome: Number of bleeds over time [ Time Frame: 24 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Reduction in number of bleeds over time [ Time Frame: Baseline, 24 weeks ]
- Reduction in number of joint bleeds over time [ Time Frame: Baseline, 24 weeks ]
- Reduction in number of target joint bleeds over time [ Time Frame: Baseline, 24 weeks ]
- Health related quality of life scores [ Time Frame: 24 weeks ]
- Trough plasma concentration (Ctrough) of emicizumab [ Time Frame: (Pre-dose) Every week during Weeks 1-4, every 2 weeks during Weeks 5-8, every 4 weeks during Weeks 9-24, every 8 weeks during Weeks 25-48, every 12 weeks thereafter (maximum up to 2 years) ]
Original Secondary Outcome: Same as current
Information By: Hoffmann-La Roche
Dates:
Date Received: July 26, 2016
Date Started: September 27, 2016
Date Completion: March 2, 2018
Last Updated: March 30, 2017
Last Verified: March 2017
