Familial HypoalphalipoproteinemiaPhase 3TANGONCT02697136
CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases
A Phase 3 study testing CER-001 in people with Familial Hypoalphalipoproteinemia, run by Cerenis Therapeutics, SA at 19 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Hypoalphalipoproteinemia
- What is being tested?
- CER-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 19 sitesin 7 countries
- Study dates
- Dec 2015to 21 Dec 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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)
Trial locations and contacts
19 sites in 7 countries.
19 sites in 7 countries
Canada5 sites
Investigative Site
Chicoutimi, Quebec
Investigative Site
Halifax, Nova Scotia
Investigative Site
London, Ontario
Investigative Site
Montreal, Quebec
Investigative Site
Vancouver, British Columbia
France4 sites
Investigative Site
Lille
Investigative Site
Montpellier
Investigative Site
Rouen
Investigative Site
Toulouse
Italy4 sites
Investigative Site
Genoa
Investigative Site
Milan
Investigative Site
Pisa
Investigative Site
Rome
About this study
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
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.
Read the 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.
- Conditions
- Familial Hypoalphalipoproteinemia
- Being tested
- CER-001, Placebo
- Keywords
- Low HDL, Low apoA-I, ABCA-1 mutations, ApoA-I mutations
- Medical subject headings
- Hypoalphalipoproteinemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CER-001Drug
Also called CAS 138-3435-67-3
Recombinant human apoA-I/phospholipid complexes
PlaceboDrug
Also called Normal saline
0.9% Sodium Chloride Injection, USP
| Study group | Role | What they receive |
|---|---|---|
| CER-001 | Experimental | CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions Receives: Drug: CER-001 |
| Placebo | Placebo comparator | Saline infusion; 9 weekly infusions followed by 20 biweekly infusions Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline to Week 24
Secondary outcomes (3)
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
Measured: 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
Measured: 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
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2016 (estimated)
- Study startDec 2015
- Primary completionOct 2018
- Study completion21 Dec 2018
Why it stopped: Lack of efficacy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Familial HypoalphalipoproteinemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cerenis Therapeutics, SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Erik SG Stroes, MD PhD, Study chair, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02697136
- Sponsor's protocol ID
- CER-001-CLIN-009
- Acronym
- TANGO
- Status last verified
- Jul 2025
- First submitted
- 17 Feb 2016
- Last updated
- 22 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02697136
- Acronym / sponsor ID
- TANGO / CER-001-CLIN-009
- Conditions
- Familial Hypoalphalipoproteinemia
- Keywords
- Low HDL, Low apoA-I, ABCA-1 mutations, ApoA-I mutations
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of efficacy
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Cerenis Therapeutics, SAIndustry (pharma, biotech, devices)
- Study start
- Dec 2015 (actual)
- Primary completion
- Oct 2018 (actual)
- Study completion
- 21 Dec 2018 (actual)
- First posted
- 3 Mar 2016 (estimated)
- Results
- Posted 22 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CER-001 | Experimental | Drug: CER-001CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions |
| Placebo | Placebo comparator | Drug: PlaceboSaline infusion; 9 weekly infusions followed by 20 biweekly infusions |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CER-001Also: CAS 138-3435-67-3Recombinant human apoA-I/phospholipid complexes | Drug | CER-001 |
| PlaceboAlso: Normal saline0.9% Sodium Chloride Injection, USP | Drug | Placebo |
Primary outcome
Change in Mean 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
3 secondary outcomes
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery
Time frame: Baseline to Week 8
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery
Time frame: Baseline to Week 48
Change in Target to Background Ratio (TBR) of the Carotid Artery
Time frame: Baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Familial Hypoalphalipoproteinemia
RecruitingObservational study
LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study
LCAT Deficiency
Recruiting
Atlantic Lipid Lowering Treatment Optimization Program
Familial Hypercholesterolemia, Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erik SG Stroes, MD PhD study chair
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
19 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CER-001 in people with Familial Hypoalphalipoproteinemia, run by Cerenis Therapeutics, SA at 19 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Canada, France, Italy, Israel, Netherlands and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.