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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-12-31

Study Completion Date

2018-12-21

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Familial Hypoalphalipoproteinemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CER-001

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

Group Type EXPERIMENTAL

CER-001

Intervention Type DRUG

Recombinant human apoA-I/phospholipid complexes

Placebo

Saline infusion; 9 weekly infusions followed by 20 biweekly infusions

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

0.9% Sodium Chloride Injection, USP

Interventions

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CER-001

Recombinant human apoA-I/phospholipid complexes

Intervention Type DRUG

Placebo

0.9% Sodium Chloride Injection, USP

Intervention Type DRUG

Other Intervention Names

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CAS 138-3435-67-3 Normal saline

Eligibility Criteria

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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

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)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No