TerminatedPhase 3

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

Stopped: Lack of efficacy

United States, Belgium, Canada30 participantsStarted 2015-12

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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)

What they're measuring

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

Timeframe: Baseline to Week 24

Trial details

NCT IDNCT02697136

SponsorCerenis Therapeutics, SA

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-10

Results submitted2024-09-19

View on ClinicalTrials.gov More Familial Hypoalphalipoproteinemia trials