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

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.

Timeline

Start

2015-12

Primary completion

2018-10

Completion

2018-12-21

Outcome

Missed primary endpoint

Stopped: “Lack of efficacy”

registry LEAST_SQUARES_MEAN CER-001: -0.3 (-1.0 to 0.4) mm2 NCT02697136 ↗

registry analysis (other test); CER-001 vs Placebo; p = 0.185; Difference in LS Means -0.08 (95% CI -1.9 to 0.4); Mixed Models Analysis NCT02697136 ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CER-001	Protein / enzyme biologic	—	Intravenous

Indications