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

• Sponsor: Cerenis Therapeutics, SA (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02697136
• Collaborator: (none)
• Enrollment: 30
• Locations: 19
• Arms: 2
• Actual Duration (Months): 36.7
• Patients Per Site: 1.6
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Familial Hypoalphalipoproteinemia	Drug: CER-001; Drug: Placebo	Phase 3

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 Design

• Study Type: Interventional
• Actual Enrollment: 30 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Dec 1, 2015
• Actual Primary Completion Date: Oct 1, 2018
• Actual Study Completion Date: Dec 21, 2018

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Change in mmean vessel wall area (MVWA) of the carotid artery [Baseline to Week 24]

   Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Secondary Outcome Measures

1. Change in mean vessel wall area (MVWA) of the carotid artery [Baseline to Week 8]

   Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

2. Change in mean vessel wall area (MVWA) of the carotid artery [Baseline to Week 48]

   Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

3. Change in Target to Background Ratio (TBR) of the carotid artery [Baseline to Week 24]

   Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET

Other Outcome Measures

1. Change in femoral MVWA [Baseline, Weeks 8, 24 and 48]

   Assessed by 3TMRI; change from baseline; CER-001 versus placebo

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigative Site	Salt Lake City	Utah	United States
2	Investigative Site	La Louvière		Belgium
3	Investigative Site	Vancouver	British Columbia	Canada
4	Investigative Site	Halifax	Nova Scotia	Canada
5	Investigative Site	London	Ontario	Canada
6	Investigative Site	Chicoutimi	Quebec	Canada
7	Investigative Site	Montreal	Quebec	Canada
8	Investigative Site	Lille		France
9	Investigative Site	Montpellier		France
10	Investigative Site	Rouen		France
11	Investigative Site	Toulouse		France
12	Investigative Site	Jerusalem		Israel
13	Investigative Site	Tel Aviv		Israel
14	Investigative Site	Genoa		Italy
15	Investigative Site	Milan		Italy
16	Investigative Site	Pisa		Italy
17	Investigative Site	Rome		Italy
18	Investigative Site	Amsterdam		Netherlands
19	Investigative Site	Utrecht		Netherlands

Sponsors and Collaborators

• Cerenis Therapeutics, SA

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Cerenis Therapeutics, SA
• ClinicalTrials.gov Identifier: NCT02697136
• Other Study ID Numbers: CER-001-CLIN-009
• First Posted: Mar 3, 2016
• Last Update Posted: Feb 11, 2019
• Last Verified: Feb 1, 2019

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Keywords provided by Cerenis Therapeutics, SA: Low HDL; Low apoA-I; ABCA-1 mutations; ApoA-I mutations
• Additional relevant MeSH terms: Hypoalphalipoproteinemias

Study Results

No Results Posted as of Feb 11, 2019