Clinical trial NCT02697136: CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Familial Hypoalphalipoproteinemia
Keywords: Low HDL
Low apoA-I
ABCA-1 mutations
ApoA-I mutations
Lead sponsor: Cerenis Therapeutics, SA
Sponsor class: INDUSTRY
First posted: 2016-03-03
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility 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)

Primary outcomes:
measure: Change in mmean vessel wall area (MVWA) of the carotid artery
timeFrame: Baseline to Week 24
Secondary outcomes:
measure: Change in mean vessel wall area (MVWA) of the carotid artery
timeFrame: Baseline to Week 8
measure: Change in mean vessel wall area (MVWA) of the carotid artery
timeFrame: Baseline to Week 48
measure: Change in Target to Background Ratio (TBR) of the carotid artery
timeFrame: Baseline to Week 24
Other outcomes:
measure: Change in femoral MVWA
timeFrame: Baseline, Weeks 8, 24 and 48
Locations:
city: Salt Lake City
country: United States
facility: Investigative Site
state: Utah
city: La Louvière
country: Belgium
facility: Investigative Site
city: Vancouver
country: Canada
facility: Investigative Site
state: British Columbia
city: Halifax
country: Canada
facility: Investigative Site
state: Nova Scotia
city: London
country: Canada
facility: Investigative Site
state: Ontario
city: Chicoutimi
country: Canada
facility: Investigative Site
state: Quebec
city: Montreal
country: Canada
facility: Investigative Site
state: Quebec
city: Lille
country: France
facility: Investigative Site
city: Montpellier
country: France
facility: Investigative Site
city: Rouen
country: France
facility: Investigative Site
city: Toulouse
country: France
facility: Investigative Site
city: Jerusalem
country: Israel
facility: Investigative Site
city: Tel Aviv
country: Israel
facility: Investigative Site
city: Genoa
country: Italy
facility: Investigative Site
city: Milan
country: Italy
facility: Investigative Site
city: Pisa
country: Italy
facility: Investigative Site
city: Rome
country: Italy
facility: Investigative Site
city: Amsterdam
country: Netherlands
facility: Investigative Site
city: Utrecht
country: Netherlands
facility: Investigative Site
Study officials:
affiliation: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
name: Erik SG Stroes, MD PhD
role: STUDY_CHAIR
References: