AzTrials
AzTrials

Pulmonary Arterial HypertensionEarly Phase 1APPRoAcH-pNCT03655704

Apabetalone for Pulmonary Arterial Hypertension: a Pilot Study

A Early Phase 1 study testing Apabetalone in people with Pulmonary Arterial Hypertension, run by Steeve Provencher at 2 sites in Canada.

Completed

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

Official record
Search all trials

Trial summary

The main OBJECTIVE of this proposal is to extend the investigator's preclinical findings on the role of epigenetics and DNA damage and Bromodomain-Containing Protein 4 (BRD4) inhibition as a therapy for a devastating disease, pulmonary arterial hypertension (PAH). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Pulmonary Arterial Hypertension
What is being tested?
Apabetalone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
2 sitesCanada
Study dates
22 Aug 2019to 13 Dec 2021
Phase
Early Phase 1
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adults (18-75 yrs) with PAH of idiopathic or hereditary origin, associated with connective tissue diseases, or anorexigen use.
  • Mean PA pressure ≥25mmHg, with pulmonary artery wedge pressure ≤15mmHg. In addition, subjects will be required meet the following hemodynamic criteria:
  • PVR >480 dyn.s.cm-5
  • Negative vasoreactivity test mandatory in idiopathic, heritable, and drug/toxin induced PAH (at baseline or during previous RHC).
  • World Health Organization functional class (WHO FC) II or III.
  • Appropriate stable therapy for PAH for ≥4 months before screening, including endothelin receptor antagonists (ERAs) other than bosentan and/or phosphodiesterase type 5 (PDE-5) inhibitors and/or prostanoids.
Show all 10Show fewer
  • Two 6-min walk tests of 150-550m inclusive and within ±15% of each other (the latter being used as baseline value).
  • Patients must be able to understand the study procedures and agree to participate in the study by providing written informed consent.
  • Patients of childbearing potential must have a negative serum pregnancy test (β-hCG) within 72 hours prior to receiving the first dose of study treatment.
  • Patients must be postmenopausal, free from menses for >1 year, surgically sterilized, willing to use adequate contraception to prevent pregnancy, or agree to abstain from activities that could result in pregnancy, from enrollment through 3 months after the last dose of study treatment.

Who cannot join

  • PAH related to HIV infection, portal hypertension or congenital heart disease.
  • Pulmonary hypertension due to left heart disease (WHO PH group 2), lung disease and/or hypoxia (WHO PH group 3), chronic thromboembolic pulmonary hypertension (WHO PH group 4), or unclear multifactorial mechanisms (WHO PH group 5).
  • Known or suspected pulmonary veno-occlusive disease (PVOD).
  • Severe restrictive lung disease (Total Lung Capacity <60% predicted)
  • Severe obstructive lung disease (FEV1/FVC < 60% after a bronchodilator)
  • DLCO <40%
Show all 34Show fewer
  • Systolic blood pressure <90 mmHg
  • Resting heart rate in the awake patient <50 BPM or >110 BPM
  • Clinically unstable right heart failure within the last 3 months or are WHO FC IV.
  • Received any investigational drug within 30 days of screening.
  • Body mass index (BMI) <18 or >40 kg/m2 at screening.
  • Patients must not be pregnant, breastfeeding, or expecting to conceive children while receiving study treatment and for 3 months after the last dose of study treatment.
  • Cardiopulmonary rehabilitation program based on exercise (planned or started ≤12 weeks prior to Day 1).
  • Presence of ≥3 of the following risk factors for heart failure with preserved ejection fraction at screening:
  • BMI >30 kg/m2.
  • Diabetes mellitus of any type.
  • Essential hypertension.
  • Coronary artery disease, i.e., any of the following:
  • History of stable angina ii. More than 50% stenosis in a coronary artery (by coronary angiography) iii. History of myocardial infarction iv. History of or planned coronary artery bypass grafting and/or coronary artery stenting.
  • A ventilation-perfusion lung scan or pulmonary angiography indicative of thromboembolic disease.
  • Evidence of organ dysfunction other than right heart failure, including:
  • Creatinine clearance <45 ml/min (using the Cockroft-Gault formula).
  • Serum AST or ALT >3 x ULN.
  • Total bilirubin > 1.5 x ULN.
  • Childs-Pugh class B-C liver cirrhosis.
  • Hemoglobin <100 g/L.
  • Absolute neutrophil count < 1,500/μL .
  • Platelets < 150,000/μL .
  • Anticipated survival less than 1 year due to concomitant disease.
  • History of cancer in the past 5 years (except for low grade and fully resolved non-melanoma skin cancer).
  • Hypersensitivity to the components of apabetalone or any excipient of their formulations.
  • Forbidden concomitant therapy:
  • Any investigational drug other than the study treatment.
  • Based on in vitro data and clinical exposure data, apabetalone is considered unlikely to cause clinically significant drug interactions through inhibition or induction of cytochrome P450 enzyme activity. Nonetheless, as the contribution of metabolic clearance to total drug clearance in man is unknown, potent inhibitors (ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, telithromycin, clarithromycin and nelfanavir) or inducers (Phenytoin, rifampicin, carbamazepine and phenobarbitone, nevirapine, modafinil and St John's Wort) of CYP3A4 must not be used during this study for any patient receiving apabetalone to ensure patient safety. Moreover, bosentan has been associated with a 5-10% risk or reversible raised in LFTs. Although there is no evidence of increased risk of apabetalone-related increases in LFTs amongst bosentan users, the use of bosentan will be forbidden during this study.

Trial locations and contacts

2 sites in 1 country.

Canada2 sites
  • Peter Lougheed Center

    Calgary, Alberta, T1Y 6J4

  • IUCPQ-UL

    Québec, Quebec, G1V 4G5

Other trials in Canada

About this study

The main OBJECTIVE of this proposal is to extend the investigator's preclinical findings on the role of epigenetics and DNA damage and Bromodomain-Containing Protein 4 (BRD4) inhibition as a therapy for a devastating disease, pulmonary arterial hypertension (PAH).

There is strong evidence that BRD4 plays a key role in the pathological phenotype in PAH accounting for disease progression and that BRD4 inhibition can reverse PAH in several animal models. Intriguingly, coronary artery disease (CAD) and metabolic syndrome are more prevalent in PAH compared with the global population, suggesting a link between these diseases. Interestingly, BRD4 is also a trigger for calcification and remodeling processes and regulates transcription of lipoprotein and inflammatory factors, all of which are important in PAH and CAD. Apabetalone, an orally available BRD4 inhibitor, is now in a clinical development stage with a good safety profile.

At this stage, the investigators propose a pilot study to assess the feasibility of a Phase 2 clinical trial assessing apabetalone in the PAH population. The overall HYPOTHESIS is that BRD4 inhibition with apabetalone is a safe and effective therapy for PAH.

Read the detailed description

In line with most pilot and safety studies, this is a two-centre (Quebec and Calgary) open-label trial. A 4-week pre-treatment phase will allow ensuring that patients are on stable doses of medication. Patients will be given doses of apabetalone 100mg BID for 16 weeks. Patients will be regularly followed. At baseline and week 16, a cardiac catheterization and MRI will assess changes in pulmonary hemodynamics and RV function.

Being tested
Apabetalone
Keywords
Pulmonary Arterial Hypertension, Apabetalone, Bromodomain-Containing Protein 4 (BRD4), BRD4 inhibitor, Feasibility pilot study, Phase 2 clinical trial, Open-label, Two-centre
Medical subject headings
Pulmonary Arterial Hypertension

What participants receive

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

  • ApabetaloneDrug

    Also called BRD4 inhibitor

    A 4-week pre-treatment phase will allow ensuring that patients are on stable doses of medication. Patients will be given doses of apabetalone 100mg BID for 16 weeks. Patients will be regularly followed (Fig.1). At baseline and week 16, a cardiac catheterization and MRI will assess changes in pulmonary hemodynamics and RV function.

Study groups in NCT03655704
Study groupRoleWhat they receive
ApabetaloneExperimental100mg BID for 16 weeks.
Receives: Drug: Apabetalone

What is being measured

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

  1. Change in Pulmonary Vascular Resistance (PVR), dyn·s·cm-5

    Right heart catheterization: Measuring PVR is performed in a standardized manner in catheterization laboratories of the participating centres, according to recommendations. Printed copies of waveforms will be kept for monitoring visits and documentation of the accuracy of the pressures and calculations.

    Measured: Baseline,and 16 weeks later

Timeline

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

  1. First posted31 Aug 2018
  2. Study start22 Aug 2019
  3. Primary completion15 Oct 2021
  4. Study completion13 Dec 2021

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
Early Phase 1
Primary purpose
Screening
Allocation
Not applicable
Intervention model
Single group
Model description
Pilot study to assess the feasibility of a Phase 2 clinical trial assessing apabetalone in 10 PAH patients
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03655704
Sponsor's protocol ID
CER-21723
Acronym
APPRoAcH-p
Status last verified
Apr 2022
First submitted
28 Aug 2018
Last updated
25 Apr 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Meloche J, Potus F, Vaillancourt M, Bourgeois A, Johnson I, Deschamps L, Chabot S, Ruffenach G, Henry S, Breuils-Bonnet S, Tremblay E, Nadeau V, Lambert C, Paradis R, Provencher S, Bonnet S. Bromodomain-Containing Protein 4: The Epigenetic Origin of Pulmonary Arterial Hypertension. Circ Res. 2015 Aug 28;117(6):525-35. doi: 10.1161/CIRCRESAHA.115.307004. Epub 2015 Jul 29.

    PubMed 26224795
  • Meloche J, Lampron MC, Nadeau V, Maltais M, Potus F, Lambert C, Tremblay E, Vitry G, Breuils-Bonnet S, Boucherat O, Charbonneau E, Provencher S, Paulin R, Bonnet S. Implication of Inflammation and Epigenetic Readers in Coronary Artery Remodeling in Patients With Pulmonary Arterial Hypertension. Arterioscler Thromb Vasc Biol. 2017 Aug;37(8):1513-1523. doi: 10.1161/ATVBAHA.117.309156. Epub 2017 May 4.

    PubMed 28473439
  • Paulin R, Michelakis ED. The metabolic theory of pulmonary arterial hypertension. Circ Res. 2014 Jun 20;115(1):148-64. doi: 10.1161/CIRCRESAHA.115.301130.

    PubMed 24951764
  • Zamanian RT, Hansmann G, Snook S, Lilienfeld D, Rappaport KM, Reaven GM, Rabinovitch M, Doyle RL. Insulin resistance in pulmonary arterial hypertension. Eur Respir J. 2009 Feb;33(2):318-24. doi: 10.1183/09031936.00000508. Epub 2008 Dec 1.

    PubMed 19047320
  • Ruffenach G, Chabot S, Tanguay VF, Courboulin A, Boucherat O, Potus F, Meloche J, Pflieger A, Breuils-Bonnet S, Nadeau V, Paradis R, Tremblay E, Girerd B, Hautefort A, Montani D, Fadel E, Dorfmuller P, Humbert M, Perros F, Paulin R, Provencher S, Bonnet S. Role for Runt-related Transcription Factor 2 in Proliferative and Calcified Vascular Lesions in Pulmonary Arterial Hypertension. Am J Respir Crit Care Med. 2016 Nov 15;194(10):1273-1285. doi: 10.1164/rccm.201512-2380OC.

    PubMed 27149112
  • Lehmann N, Erbel R, Mahabadi AA, Rauwolf M, Mohlenkamp S, Moebus S, Kalsch H, Budde T, Schmermund A, Stang A, Fuhrer-Sakel D, Weimar C, Roggenbuck U, Dragano N, Jockel KH; Heinz Nixdorf Recall Study Investigators. Value of Progression of Coronary Artery Calcification for Risk Prediction of Coronary and Cardiovascular Events: Result of the HNR Study (Heinz Nixdorf Recall). Circulation. 2018 Feb 13;137(7):665-679. doi: 10.1161/CIRCULATIONAHA.116.027034. Epub 2017 Nov 15.

    PubMed 29142010
  • Nicholls SJ, Puri R, Wolski K, Ballantyne CM, Barter PJ, Brewer HB, Kastelein JJ, Hu B, Uno K, Kataoka Y, Herrman JP, Merkely B, Borgman M, Nissen SE. Effect of the BET Protein Inhibitor, RVX-208, on Progression of Coronary Atherosclerosis: Results of the Phase 2b, Randomized, Double-Blind, Multicenter, ASSURE Trial. Am J Cardiovasc Drugs. 2016 Feb;16(1):55-65. doi: 10.1007/s40256-015-0146-z.

    PubMed 26385396
  • Vonk-Noordegraaf A, Haddad F, Chin KM, Forfia PR, Kawut SM, Lumens J, Naeije R, Newman J, Oudiz RJ, Provencher S, Torbicki A, Voelkel NF, Hassoun PM. Right heart adaptation to pulmonary arterial hypertension: physiology and pathobiology. J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D22-33. doi: 10.1016/j.jacc.2013.10.027.

    PubMed 24355638
  • Bogaard HJ, Abe K, Vonk Noordegraaf A, Voelkel NF. The right ventricle under pressure: cellular and molecular mechanisms of right-heart failure in pulmonary hypertension. Chest. 2009 Mar;135(3):794-804. doi: 10.1378/chest.08-0492.

    PubMed 19265089
  • Lajoie AC, Lauziere G, Lega JC, Lacasse Y, Martin S, Simard S, Bonnet S, Provencher S. Combination therapy versus monotherapy for pulmonary arterial hypertension: a meta-analysis. Lancet Respir Med. 2016 Apr;4(4):291-305. doi: 10.1016/S2213-2600(16)00027-8. Epub 2016 Feb 27.

    PubMed 26935844
  • Humbert M, Lau EM, Montani D, Jais X, Sitbon O, Simonneau G. Advances in therapeutic interventions for patients with pulmonary arterial hypertension. Circulation. 2014 Dec 9;130(24):2189-208. doi: 10.1161/CIRCULATIONAHA.114.006974. No abstract available.

    PubMed 25602947
  • Humbert M, Sitbon O, Yaici A, Montani D, O'Callaghan DS, Jais X, Parent F, Savale L, Natali D, Gunther S, Chaouat A, Chabot F, Cordier JF, Habib G, Gressin V, Jing ZC, Souza R, Simonneau G; French Pulmonary Arterial Hypertension Network. Survival in incident and prevalent cohorts of patients with pulmonary arterial hypertension. Eur Respir J. 2010 Sep;36(3):549-55. doi: 10.1183/09031936.00057010. Epub 2010 Jun 18.

    PubMed 20562126
  • Humbert M, Sitbon O, Chaouat A, Bertocchi M, Habib G, Gressin V, Yaici A, Weitzenblum E, Cordier JF, Chabot F, Dromer C, Pison C, Reynaud-Gaubert M, Haloun A, Laurent M, Hachulla E, Cottin V, Degano B, Jais X, Montani D, Souza R, Simonneau G. Survival in patients with idiopathic, familial, and anorexigen-associated pulmonary arterial hypertension in the modern management era. Circulation. 2010 Jul 13;122(2):156-63. doi: 10.1161/CIRCULATIONAHA.109.911818. Epub 2010 Jun 28.

    PubMed 20585011
  • Benza RL, Gomberg-Maitland M, Miller DP, Frost A, Frantz RP, Foreman AJ, Badesch DB, McGoon MD. The REVEAL Registry risk score calculator in patients newly diagnosed with pulmonary arterial hypertension. Chest. 2012 Feb;141(2):354-362. doi: 10.1378/chest.11-0676. Epub 2011 Jun 16.

    PubMed 21680644
  • Benza RL, Miller DP, Gomberg-Maitland M, Frantz RP, Foreman AJ, Coffey CS, Frost A, Barst RJ, Badesch DB, Elliott CG, Liou TG, McGoon MD. Predicting survival in pulmonary arterial hypertension: insights from the Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management (REVEAL). Circulation. 2010 Jul 13;122(2):164-72. doi: 10.1161/CIRCULATIONAHA.109.898122. Epub 2010 Jun 28.

    PubMed 20585012

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Apabetalone in people with Pulmonary Arterial Hypertension, run by Steeve Provencher at 2 sites 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 10 inclusion and 34 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 25 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 Pulmonary Vascular Resistance (PVR), dyn·s·cm-5 (time frame: Baseline,and 16 weeks later). 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 NCT03655704, which also lists 82 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 25 Apr 2022.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in