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

Cerenis Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Familial Hypoalphalipoproteinemia

Treatments: Drug: CER-001; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02697136

CER-001-CLIN-009

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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30 participants in 2 patient groups, including a placebo group

CER-001

Experimental group

Description:

CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions

Treatment:

Drug: CER-001

Placebo

Placebo Comparator group

Description:

Saline infusion; 9 weekly infusions followed by 20 biweekly infusions

Treatment:

Drug: Placebo