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Homozygous Familial HypercholesterolemiaPhase 2MODENCT01412034

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

A Phase 2 study testing CER-001 in people with Homozygous Familial Hypercholesterolemia, run by Cerenis Therapeutics, SA at 9 sites in 5 countries.

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

The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress existing disease due to years of cholesterol accumulation in the vessel walls and therefore… Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Homozygous Familial Hypercholesterolemia
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
23 participants
Where
9 sitesin 5 countries
Study dates
Nov 2011to Aug 2014
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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male or female subject 12 years or older
  • Subject presents with Homozygous FH

Who cannot join

  • Weight >100 kg
  • Subjects with significant health problems in the recent past including blood disorders, cancer, or digestive problems
  • Female subjects of child-bearing potential
  • Known major hematologic, renal , hepatic, metabolic, gastrointestinal or endocrine dysfunction
  • Contraindication to MRI scanning that would preclude the use of contrast-enhanced 3TMRI

Trial locations and contacts

9 sites in 5 countries.

9 sites in 5 countries

Netherlands3 sites

  • Clinical Research Facility

    Amsterdam, 1105AZ

  • Clinical Research Facility

    Maastricht, 6229 HX

  • Clinical Research Facility

    Nijmegen, 6500 HB

Other trials in Netherlands

United States2 sites

  • Clinical Research Facility

    Hartford, Connecticut, 06102

  • Clinical Research Facility

    North Massapequa, New York, 11758

Other trials in United States

Canada2 sites

  • Clinical Research Facility

    Chicoutimi, Quebec, G7H 7P2

  • Clinical Research Facility

    Québec, Quebec, G1V4M6

Other trials in Canada

Italy1 site

  • Clinical Research Facility

    Rome, 100161

Other trials in Italy

United Kingdom1 site

  • Clinical Research Facility

    Manchester, M13 9WL

Other trials in United Kingdom

About this study

Official title: Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)

The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress existing disease due to years of cholesterol accumulation in the vessel walls and therefore cannot adequately reduce the existing risk for an ischemic event. HDL has multiple actions that could lead to plaque stabilization and regression, such as rapid removal of large quantities of cholesterol from the vasculature, improvement in endothelial function, protection against oxidative damage and reduction in inflammation. 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 measurements in patients with HoFH.

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

What participants receive

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

  • CER-001Drug

    Biweekly infusion

Study groups in NCT01412034
Study groupRoleWhat they receive
CER-001ExperimentalOpen 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. Percent change from baseline to follow-up in carotid mean vessel wall area

    Percent change from baseline to follow-up in carotid mean vessel wall area

    Measured: Baseline then 6 months and/or ~2 weeks post final dose

Secondary outcomes (1)

  1. Change in carotid vessel wall volume

    Percent change in carotid vessel wall volume , as assessed by 3TMRI, from the baseline measurement to the follow up taken ~2 weeks following the final dose of study medication.

    Measured: Baseline then 6 months and/or ~2 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 posted8 Aug 2011 (estimated)
  2. Study startNov 2011
  3. Primary completionOct 2013
  4. Study completionAug 2014

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
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01412034
Sponsor's protocol ID
CER-001-CLIN-003
Acronym
MODE
Status last verified
Jul 2015
First submitted
5 Aug 2011
Last updated
18 Aug 2015
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

  • 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

  • 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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CER-001 in people with Homozygous Familial Hypercholesterolemia, run by Cerenis Therapeutics, SA at 9 sites in 5 countries.

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 2 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 18 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in Netherlands, United States, Canada, Italy, United Kingdom. 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?

Percent change from baseline to follow-up in carotid mean vessel wall area (time frame: Baseline then 6 months and/or ~2 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 NCT01412034, 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 18 Aug 2015.

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