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

Phase 3

Terminated

• Conditions: Familial Hypoalphalipoproteinemia
• Interventions: Drug: Placebo; Drug: CER-001

• Registration Number: NCT02697136
• Lead Sponsor: Cerenis Therapeutics, SA

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 Description

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.

Study Timeline

• Study Start: 2015-12
• Study First Submitted: 2016-02-17
• Study First Submitted QC: 2016-02-26
• Study First Posted: 2016-03-03
• Primary Completion: 2018-10
• Study Completion: 2018-12-21
• Status Verified: 2019-02
• Disposition First Submitted: 2019-02-06
• Disposition First Submitted QC: 2019-02-06
• Last Update Submitted: 2019-02-06
• Disposition First Posted: 2019-02-11
• Last Update Posted: 2019-02-11

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 30

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Saline infusion; 9 weekly infusions followed by 20 biweekly infusions
CER-001	CER-001	CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions

Primary Outcome Measures

Name	Time	Method
Change in mmean vessel wall area (MVWA) of the carotid artery	Baseline to Week 24	Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Secondary Outcome Measures

Name	Time	Method
Change in mean vessel wall area (MVWA) of the carotid artery	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	Baseline to Week 24	Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET

Trial Locations

Locations (1)

Investigative Site; 🇳🇱 Utrecht, Netherlands