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

Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Familial Primary Hypoalphalipoproteinemia

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.

Study Overview

• Status: Terminated
• Conditions: Familial Hypoalphalipoproteinemia
• Intervention / Treatment: Drug: CER-001; Drug: Placebo

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belgium
  • La Louvière, Belgium
    • Investigative Site
• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada
      • Investigative Site
  • Nova Scotia
    • Halifax, Nova Scotia, Canada
      • Investigative Site
  • Ontario
    • London, Ontario, Canada
      • Investigative Site
  • Quebec
    • Chicoutimi, Quebec, Canada
      • Investigative Site
    • Montreal, Quebec, Canada
      • Investigative Site
• France
  • Lille, France
    • Investigative Site
  • Montpellier, France
    • Investigative Site
  • Rouen, France
    • Investigative Site
  • Toulouse, France
    • Investigative Site
• Israel
  • Jerusalem, Israel
    • Investigative Site
  • Tel Aviv, Israel
    • Investigative Site
• Italy
  • Genoa, Italy
    • Investigative Site
  • Milan, Italy
    • Investigative Site
  • Pisa, Italy
    • Investigative Site
  • Rome, Italy
    • Investigative Site
• Netherlands
  • Amsterdam, Netherlands
    • Investigative Site
  • Utrecht, Netherlands
    • Investigative Site
• United States
  • Utah
    • Salt Lake City, Utah, United States, 84108
      • Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CER-001; CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions	Drug: CER-001; Recombinant human apoA-I/phospholipid complexes; Other Names: CAS 138-3435-67-3
Placebo Comparator: Placebo; Saline infusion; 9 weekly infusions followed by 20 biweekly infusions	Drug: Placebo; 0.9% Sodium Chloride Injection, USP; Other Names: Normal saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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	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	Baseline to Week 24

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Femoral MVWA	Assessed by 3TMRI; change from baseline; CER-001 versus placebo	Baseline, Weeks 8, 24 and 48

Collaborators and Investigators

• Sponsor: Cerenis Therapeutics, SA
• Investigators: Study Chair: Erik SG Stroes, MD PhD, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2015
• Primary Completion (Actual): October 1, 2018
• Study Completion (Actual): December 21, 2018

Study Registration Dates

• First Submitted: February 17, 2016
• First Submitted That Met QC Criteria: February 26, 2016
• First Posted (Estimated): March 3, 2016

Study Record Updates

• Last Update Posted (Actual): July 22, 2025
• Last Update Submitted That Met QC Criteria: July 2, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: Low HDL; Low apoA-I; ABCA-1 mutations; ApoA-I mutations
• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Hypolipoproteinemias; Hypoalphalipoproteinemias
• Other Study ID Numbers: CER-001-CLIN-009

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: To be determined