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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02697136
Recruitment Status : Terminated (Lack of efficacy)
First Posted : March 3, 2016
Last Update Posted : February 11, 2019
Sponsor:
Information provided by (Responsible Party):
Cerenis Therapeutics, SA

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.

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Study Type : Interventional  (Clinical Trial)
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 : December 2015
Actual Primary Completion Date : October 2018
Actual Study Completion Date : December 21, 2018

Resource links provided by the National Library of Medicine


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 Name: 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 Name: Normal saline




Primary Outcome Measures :
  1. Change in mmean vessel wall area (MVWA) of the carotid artery [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Baseline, Weeks 8, 24 and 48 ]
    Assessed by 3TMRI; change from baseline; CER-001 versus placebo



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02697136


Locations
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United States, Utah
Investigative Site
Salt Lake City, Utah, United States
Belgium
Investigative Site
La Louvière, Belgium
Canada, British Columbia
Investigative Site
Vancouver, British Columbia, Canada
Canada, Nova Scotia
Investigative Site
Halifax, Nova Scotia, Canada
Canada, Ontario
Investigative Site
London, Ontario, Canada
Canada, Quebec
Investigative Site
Chicoutimi, Quebec, Canada
Investigative Site
Montreal, Quebec, Canada
France
Investigative Site
Lille, France
Investigative Site
Montpellier, France
Investigative Site
Rouen, France
Investigative Site
Toulouse, France
Israel
Investigative Site
Jerusalem, Israel
Investigative Site
Tel Aviv, Israel
Italy
Investigative Site
Genoa, Italy
Investigative Site
Milan, Italy
Investigative Site
Pisa, Italy
Investigative Site
Rome, Italy
Netherlands
Investigative Site
Amsterdam, Netherlands
Investigative Site
Utrecht, Netherlands
Sponsors and Collaborators
Cerenis Therapeutics, SA
Investigators
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Study Chair: Erik SG Stroes, MD PhD Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

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Responsible Party: Cerenis Therapeutics, SA
ClinicalTrials.gov Identifier: NCT02697136    
Other Study ID Numbers: CER-001-CLIN-009
First Posted: March 3, 2016    Key Record Dates
Last Update Posted: February 11, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: To be determined
Keywords provided by Cerenis Therapeutics, SA:
Low HDL
Low apoA-I
ABCA-1 mutations
ApoA-I mutations
Additional relevant MeSH terms:
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Hypoalphalipoproteinemias
Hypolipoproteinemias
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases