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Heterozygous Familial HypercholesterolemiaPhase 2EXPRESSNCT01515241

Exploratory Study of Plaque Regression

A Phase 2 study testing CER-001 in people with Heterozygous Familial Hypercholesterolemia, run by Cerenis Therapeutics, SA at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Despite the availability of several classes of very effective drugs available to treat heterozygous Familial Hypercholesterolemia (HeFH), there remains a large unmet medical need for new, effective and well tolerated therapies. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CER-001
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteCanada
Study dates
Jan 2012to May 2012
Phase
Phase 2
Are there results?
None posted in the registry

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

Who may be able to join

  • Male or Female subjects at least 18 years old
  • Subject presents heterozygous FH, known CHD and receiving maximally tolerated lipid modifying therapy, at stable doses for at least 3 months
  • LDL-C of > 110 mg/dl
  • Angiographic evidence of coronary artery disease with suitable "target" coronary artery for IVUS

Who cannot join

  • Confirmed diagnosis of homozygous FH
  • Significant health problems (other than cardiovascular disease) in the recent past including blood disorders, cancer, or digestive problems
  • Female subjects not meeting the study definition of non child-bearing potential
  • Use of an investigational agent within 30 days of the first CER-001 dose
  • Receiving current lipid apheresis

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Montreal Heart Institute

    Montreal, Quebec, H1T1C8

Other trials in Canada

About this study

Official title: EXPLORATORY STUDY OF PLAQUE REGRESSION:A Phase II Single Center Open-Label Exploratory Trial of the Effect of CER 001 in Subjects With Familial Hypercholesterolemia

Despite the availability of several classes of very effective drugs available to treat heterozygous Familial Hypercholesterolemia (HeFH), there remains a large unmet medical need for new, effective and well tolerated therapies. There are a number of therapies given on a chronic basis to reduce long term risk, such as statins, fibrates, niacin, omega 3 fatty acids, resins, cholesterol absorption inhibitors and antiplatelet or anticoagulant drugs, but subjects with heterozygous Familial Hypercholesterolemia remain at high risk for cardiovascular events. There is still a need for acute therapies that can lead to rapid pacification of unstable plaque in order to reduce the risk of these events. This study will assess the effects of CER-001 , a recombinant human Apo-A-1 based HDL mimetic, on indices of atherosclerotic plaque progression and regression as assessed by 3Tesla MRI (3TMRI)and intravascular ultrasound (IVUS) evaluations in patients with HeFH.

Being tested
CER-001
Keywords
Heterozygous Familial Hypercholesterolemia, Familial Hypercholesterolemia, HDL mimetic, ApoA-1
Medical subject headings
Hyperlipoproteinemia Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CER-001Drug

    Weekly injection

Study groups in NCT01515241
Study groupRoleWhat they receive
Open label single arm study of CER-001OtherOpen label single arm study of CER-001
Receives: Drug: CER-001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Total Plaque Volume

    Nominal change in total plaque volume (ACTPV), as assessed by 3D IVUS, from the baseline measurement to the follow-up taken ~3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment)

    Measured: Baseline and 3 weeks post final dose

Secondary outcomes (2)

  1. Percent Change in Plaque Volume

    Percent change in total plaque volume (PCTPV), as assessed by IVUS, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication (approximately 10 weeks after the baseline assessment)

    Measured: Baseline and 3 weeks post final dose

  2. Change in carotid plaque volume

    Percent change in total carotid plaque volume, as assessed by 3TMRI, from the baseline measurement to the follow up taken approximately 3 weeks following the final dose of study medication

    Measured: Baseline and 3 weeks post final dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jan 2012 (estimated)
  2. Study startJan 2012
  3. Primary completionApr 2012
  4. Study completionMay 2012

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01515241
Sponsor's protocol ID
CER-001-CLIN-005
Acronym
EXPRESS
Status last verified
Jan 2014
First submitted
18 Jan 2012
Last updated
3 Mar 2014
Results posted
No
Data monitoring committee
No

Publications

  • Eriksson M, Carlson LA, Miettinen TA, Angelin B. Stimulation of fecal steroid excretion after infusion of recombinant proapolipoprotein A-I. Potential reverse cholesterol transport in humans. Circulation. 1999 Aug 10;100(6):594-8. doi: 10.1161/01.cir.100.6.594.

    PubMed 10441095

  • Nanjee MN, Doran JE, Lerch PG, Miller NE. Acute effects of intravenous infusion of ApoA1/phosphatidylcholine discs on plasma lipoproteins in humans. Arterioscler Thromb Vasc Biol. 1999 Apr;19(4):979-89. doi: 10.1161/01.atv.19.4.979.

    PubMed 10195926

  • Spieker LE, Sudano I, Hurlimann D, Lerch PG, Lang MG, Binggeli C, Corti R, Ruschitzka F, Luscher TF, Noll G. High-density lipoprotein restores endothelial function in hypercholesterolemic men. Circulation. 2002 Mar 26;105(12):1399-402. doi: 10.1161/01.cir.0000013424.28206.8f.

    PubMed 11914243

  • Nieuwdorp M, Vergeer M, Bisoendial RJ, op 't Roodt J, Levels H, Birjmohun RS, Kuivenhoven JA, Basser R, Rabelink TJ, Kastelein JJ, Stroes ES. Reconstituted HDL infusion restores endothelial function in patients with type 2 diabetes mellitus. Diabetologia. 2008 Jun;51(6):1081-4. doi: 10.1007/s00125-008-0975-2. Epub 2008 Apr 4. No abstract available.

    PubMed 18389214

  • Shaw JA, Bobik A, Murphy A, Kanellakis P, Blombery P, Mukhamedova N, Woollard K, Lyon S, Sviridov D, Dart AM. Infusion of reconstituted high-density lipoprotein leads to acute changes in human atherosclerotic plaque. Circ Res. 2008 Nov 7;103(10):1084-91. doi: 10.1161/CIRCRESAHA.108.182063. Epub 2008 Oct 2.

    PubMed 18832751

  • Nissen SE, Tsunoda T, Tuzcu EM, Schoenhagen P, Cooper CJ, Yasin M, Eaton GM, Lauer MA, Sheldon WS, Grines CL, Halpern S, Crowe T, Blankenship JC, Kerensky R. Effect of recombinant ApoA-I Milano on coronary atherosclerosis in patients with acute coronary syndromes: a randomized controlled trial. JAMA. 2003 Nov 5;290(17):2292-300. doi: 10.1001/jama.290.17.2292.

    PubMed 14600188

  • Tardif JC, Gregoire J, L'Allier PL, Ibrahim R, Lesperance J, Heinonen TM, Kouz S, Berry C, Basser R, Lavoie MA, Guertin MC, Rodes-Cabau J; Effect of rHDL on Atherosclerosis-Safety and Efficacy (ERASE) Investigators. Effects of reconstituted high-density lipoprotein infusions on coronary atherosclerosis: a randomized controlled trial. JAMA. 2007 Apr 18;297(15):1675-82. doi: 10.1001/jama.297.15.jpc70004. Epub 2007 Mar 26.

    PubMed 17387133

  • Waksman R, Torguson R, Kent KM, Pichard AD, Suddath WO, Satler LF, Martin BD, Perlman TJ, Maltais JA, Weissman NJ, Fitzgerald PJ, Brewer HB Jr. A first-in-man, randomized, placebo-controlled study to evaluate the safety and feasibility of autologous delipidated high-density lipoprotein plasma infusions in patients with acute coronary syndrome. J Am Coll Cardiol. 2010 Jun 15;55(24):2727-35. doi: 10.1016/j.jacc.2009.12.067.

    PubMed 20538165

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CER-001 in people with Heterozygous Familial Hypercholesterolemia, run by Cerenis Therapeutics, SA at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. 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: finished, as of the sponsor's last update on 3 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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 Total Plaque Volume (time frame: Baseline and 3 weeks post final dose). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01515241, which also lists 8 linked publications.

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 3 Mar 2014.

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