Clinical Trial: CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Fa

Brief Summary: The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when administered to patients with familial primary hypoalphalipoproteinemia with proven CVD and appropriate background lipid-lowering therapy.

Detailed Summary: Subjects will be required to have acceptable baseline 3TMRI imaging of carotid arteries. Subjects meeting all eligibility criteria will be randomized to receive CER-001 or placebo (2:1 randomization scheme). Randomized subjects will return weekly for a total of 9 infusions and then biweekly for an additional 20 infusions. 3TMRI imaging of the carotid and femoral arteries will be performed at Week 8, Week 24 (primary endpoint) and Week 48. The total study duration from randomization can range from 50 to 54 weeks for patients completing the study as designed.
Sponsor: Cerenis Therapeutics, SA

Current Primary Outcome: Change in mmean vessel wall area (MVWA) of the carotid artery [ Time Frame: Baseline to Week 24 ]

Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in mean vessel wall area (MVWA) of the carotid artery [ Time Frame: Baseline to Week 8 ]
    Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
  • Change in mean vessel wall area (MVWA) of the carotid artery [ Time Frame: Baseline to Week 48 ]
    Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
  • Change in Target to Background Ratio (TBR) of the carotid artery [ Time Frame: Baseline to Week 24 ]
    Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET


Original Secondary Outcome: Same as current

Information By: Cerenis Therapeutics, SA

Dates:
Date Received: February 17, 2016
Date Started: December 2015
Date Completion: December 2017
Last Updated: March 16, 2017
Last Verified: March 2017