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

NCT02697136 | Terminated Phase 3 Results DMC

• 1 other identifier: CER-001-CLIN-009
• Study Type: interventional
• Target: 30
• Locations: 7 countries
• Sites: 19

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.

Conditions

Familial Hypoalphalipoproteinemia

Keywords

Low HDL; Low apoA-I; ABCA-1 mutations; ApoA-I mutations

Outcome Measures

Primary Outcomes (1)

• 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

  Baseline to Week 24

Secondary Outcomes (3)

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

  Baseline to Week 8

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

  Baseline to Week 48

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

  Baseline to Week 24

Other Outcomes (1)

• Change in Femoral MVWA

  Baseline, Weeks 8, 24 and 48

Study Arms (2)

CER-001

EXPERIMENTAL

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

Drug: CER-001

Placebo

PLACEBO COMPARATOR

Saline infusion; 9 weekly infusions followed by 20 biweekly infusions

Drug: Placebo

Interventions

CER-001 DRUG

Recombinant human apoA-I/phospholipid complexes

Also known as: CAS 138-3435-67-3

CER-001

Placebo DRUG

0.9% Sodium Chloride Injection, USP

Also known as: Normal saline

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Cerenis Therapeutics, SA: lead

Study Sites (19)

Investigative Site

Salt Lake City, Utah, 84108, United States

Location

Investigative Site

La Louvière, Belgium

Location

Investigative Site

Vancouver, British Columbia, Canada

Location

Investigative Site

Halifax, Nova Scotia, Canada

Location

Investigative Site

London, Ontario, Canada

Location

Investigative Site

Chicoutimi, Quebec, Canada

Location

Investigative Site

Montreal, Quebec, Canada

Location

Investigative Site

Lille, France

Location

Investigative Site

Montpellier, France

Location

Investigative Site

Rouen, France

Location

Investigative Site

Toulouse, France

Location

Investigative Site

Jerusalem, Israel

Location

Investigative Site

Tel Aviv, Israel

Location

Investigative Site

Genoa, Italy

Location

Investigative Site

Milan, Italy

Location

Investigative Site

Pisa, Italy

Location

Investigative Site

Rome, Italy

Location

Investigative Site

Amsterdam, Netherlands

Location

Investigative Site

Utrecht, Netherlands

Location

MeSH Terms

Conditions

Hypoalphalipoproteinemias

Interventions

CER-001; Saline Solution

Condition Hierarchy (Ancestors)

Hypolipoproteinemias; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Constance Peyrottes
• Organization: Abionyx Pharma

cpeyrottes@abionyx.com

+33673045380

Study Officials

• Erik SG Stroes, MD PhD

  Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2016
• First Posted: March 3, 2016
• Study Start: December 1, 2015
• Primary Completion: October 1, 2018
• Study Completion: December 21, 2018
• Last Updated: July 22, 2025
• Results First Posted: July 22, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (5); IT (4); NL (2); IL (2); BE (1); FR (4); US (1)