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

Phase 3TerminatedNCT02697136

Cerenis Therapeutics, SA30 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Familial Hypoalphalipoproteinemia

Interventions

CER-001DRUG

Recombinant human apoA-I/phospholipid complexes

PlaceboDRUG

0.9% Sodium Chloride Injection, USP

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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)

Locations (19)

Investigative Site

Salt Lake City, Utah, United States

Investigative Site

La Louvière, Belgium

Investigative Site

Vancouver, British Columbia, Canada

Outcomes

Primary Outcomes

Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery

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

Time frame: Baseline to Week 24

Secondary Outcomes

Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery

Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Time frame: Baseline to Week 8

Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery

Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Time frame: Baseline to Week 48

Change in Target to Background Ratio (TBR) of the Carotid Artery

Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET

Time frame: Baseline to Week 24

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.