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

• Status: Terminated
• Trial end date: 2018-12-21
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cerenis Therapeutics, SA

Criteria

Main Inclusion Criteria:

• Male and female patients, aged 18 and above.

• ApoA-I < 70 mg/dL

• Symptomatic or asymptomatic cardiovascular disease

• Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for ABCA1 and/or ApoA-1

• Stable doses of lipid lowering therapies for at least 6 weeks prior to baseline procedures

Main Exclusion Criteria:

• Females of childbearing potential

• Patients with LCAT mutations

• Patients who experienced recent cardiovascular or cerebrovascular events

• Hypertriglyceridemia (>500 mg/dL)

• Severe anemia (Hgb < 10 g/dL)

• Uncontrolled diabetes (HbA1c >10%)

• Congestive heart failure (NYHA class II or higher)

• Contraindication for MRI scanning (e.g., implanted metal objects, claustrophobia)