Heart FailurePhase 2NCT02914665
A Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure
A Phase 2 study testing elamipretide in people with Heart Failure, run by Stealth BioTherapeutics Inc. at 46 sites in 10 countries.
Trial summary
This is a phase 2 randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of short term treatment with elamipretide in patients hospitalized with congestion due to heart failure Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Heart Failure
- What is being tested?
- elamipretide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 308 participants
- Where
- 46 sitesin 10 countries
- Study dates
- 20 Oct 2016to 27 Nov 2017
- 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
- A history of chronic heart failure for at least 1 month
- Treated with ≥40 mg/day of furosemide or bumetanide ≥1 mg/day or torasemide ≥10 mg/day for at least 1 month
- In-hospital observation/admission and treatment for ≤72 hours and primary cause for admission is heart failure with persistent congestion in the opinion of the Investigator (i.e. at least +2 pitting oedema and/or an estimated 8 kg gain in weight over baseline over the past 4 weeks) requiring intravenous loop diuretic therapy
- Sufficiently severe oedema to justify treatment by an intravenous infusion of furosemide of 10 mg/hour for at least 48 hours
- Systolic blood pressure >90 mmHg and considered to be haemodynamically stable, in the opinion of the Investigator
- History of left ventricular ejection fraction (LVEF) ≤40% confirmed in the last 18 months
- NT-proBNP >1500 pg/ml or BNP >500 pg/ml
- An eGFR of >30 mL/min/1.73 m2 using the eGFR (mL/min/1.73 m2) = 175 x (Scr)-1.154 x (Age)-0.203 x (0.742 if female) x (1.212 if African American)
Who cannot join
- Acute coronary syndrome, stroke, or transient ischemic attack (TIA), coronary or peripheral revascularization procedures, valve procedures, OR any major surgical procedure within the previous 6 weeks
- Invasive cardiac investigation and/or treatment (i.e. coronary angiography, percutaneous coronary intervention [PCI] or surgery) or other surgical procedure planned in the next 4 weeks
- Use of intravenous radiographic contrast agent within 72 hours prior to screening or planned use during the study
- Severe, in the investigators opinion, uncorrected valve disease or congenital heart disease as the cause for cardiac decompensation
- Acute mechanical cause of decompensated heart failure such as papillary muscle rupture
- Obstructive or infiltrative cardiomyopathy (e.g. amyloid, sarcoid, etc), suspected acute myocarditis, or heart failure related to an untreated metabolic condition (e.g. haemochromatosis)
Show all 22Show fewer
- Second or third degree heart block unless the subject has a ventricular pacemaker
- Atrial fibrillation/flutter with sustained ventricular response of >130 bpm
- Placement of a ventricular resynchronization device within the previous 6 weeks
- Treatment or planned treatment with intravenous inotropic agents other than digoxin at any time on this admission
- Receipt of intravenous vasodilator therapy ≤ 6 hours prior to randomization
- The presence of any mechanical assist device or listed for or a history of a heart transplant
- Severe respiratory disease or anticipated need for mechanical respiratory support (i.e. mechanical ventilation)
- Anuric in the previous 24 hours
- Haemoglobin <9 g/dL at screening or planned blood transfusions in the next 30 days
- Serum potassium >5.5 mEq/L
- Marked proteinuria suggestive of nephrotic syndrome
- Estimated GFR (eGFR) as per MDRD equation <30 ml/min
- Serum albumin of < 2.8 g/dL
- Liver enzymes (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST]) elevation >5 times the upper limit of normal (ULN)
- Total bilirubin >2.0 times ULN in the absence of Gilbert's Syndrome
- Current or planned ultrafiltration, paracentesis, haemofiltration or dialysis
Trial locations and contacts
46 sites in 10 countries.
46 sites in 10 countries
Spain9 sites
Complexo Hospitalario Universitario de A Coruña
A Coruña, 15006 A
Hospital Universitario de Bellvitge
Barcelona, 08907
Hospital General Universitario Gregorio Marañón
Madrid, 28007
Hospital Clínico San Carlos
Madrid, 28040
Hospital Universitario Puerta de Hierro Majadahonda
Madrid, 28222 Majadahonda
Hospital Alvaro Cunqueiro
Pontevedra, 36312 Vigo
Hospital Clínico Universitario de Valencia
Valencia, 46010
Consorci Hospital General Universitari de Valencia
Valencia, 46014
Hospital Universitari i Politècnic La Fe
Valencia, 46026
Netherlands7 sites
Hospital Jeroen Bosch
's-Hertogenbosch, 5223 GV 's
Deventer Hospital
Deventer, 7416 S
Hospital Gelderse Vallei
Ede, 6716 RP
University Medical Center Groningen
Groningen, 9713 GZ
Hospital Antonius/D&A research Genetic
Sneek, 8601 ZR
Hospital Elisabeth -Tweesteden
Tilburg, 5042 AD
Hospitals Gelre
Zutphen, 7207 AE
Bulgaria6 sites
Department of Internal Diseases, "Multiprofile Hospital for Active Treatment Sveta Ekaterina - Dimitrovgrad" EOOD
Dimitrovgrad, 6400
Clinic of Cardiology, "Second Multiprofile Hospital for Active Treatement - Sofia" EAD
Sofia, 1202
Clinic of Cardiology, Multiprofile Hospital for Active Treatment National Heart Hospital" EAD
Sofia, 1309
Clinic of Cardiology, "City Clinic University Multiprofile Hospital for Active Treatment" EOOD
Sofia, 1407
Clinic of Internal Diseases, "Multiprofile Hospital for Active Treatment, "Sveta Anna Sofia" AD
Sofia, 1750
Department of Cardiology, "Multiprofile Regional Hospital for Active Treatement Dr. Stefan Cherkezov" AD
Veliko Tarnovo, 5000
Serbia6 sites
Clinical Centre of Serbia, Emergency Centre
Belgrade, 11000
Clinical Centre of Serbia, Institute for Cardiovascular Diseases
Belgrade, 11000
Clinical Hospital Centre Bezanijska kosa
Belgrade, 11070
Clinical Hospital Centre Zemun
Belgrade, 11080
Institute for Cardiovascular Diseases of Vojvodina
Kamenitz, 21204
Clinical Centre Nis
Niš, 18000
Hungary4 sites
Hungarian Institute Of Cardiology, Department of Adult Cardiology
Budapest, 1096
Semmelweis University Heart and Vascular Center
Budapest, 1122
County St. George University Teaching Hospital, Department of Internal Medicine Cardiology
Székesfehérvár, 8000
Zala County Hospital Cardiology Department
Zalaegerszeg, 8900
Latvia4 sites
Daugavpils Regional Hospital
Daugavpils, LV5417
Riga East Clinical University Hospital, Clinic Gailezers
Liepāja, LV1038
Liepaja Regional Hospital
Liepāja, LV3402
Pauls Stradins Clinical University Hospital, Latvian Centre of Cardiology
Riga, LV1002
Poland3 sites
Bieganski Provincial Specialist Hospital, Department of Cardiology, Clinic of Cardiology UM
Lodz, 91-347
Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic Heart Failure and Transplantation
Warsaw, 04-628
Cardinal Stefan Wyszynski, Institute of Cardiology, Clinic of Coronary Heart Disease and Structural Heart Disease
Warsaw, 04-628
United Kingdom3 sites
Bradford Royal Infirmary
Bradford, BD6 6RJ
Glenfield Hospital
Leicester, LE3 9QP
Torbay Hospital
Torquay, TQ2 7AA
Belgium2 sites
Hospital Onze Lieve Vrouw campus Aalst
Aalst, 9300
Hospital ZNA Middelheim
Antwerp, 2020
France2 sites
Hôpital Henri Mondor
Créteil, 94000
CHU de Rangueil
Toulouse, 31059
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure
This is a phase 2 randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of short term treatment with elamipretide in patients hospitalized with congestion due to heart failure
- Conditions
- Heart Failure
- Being tested
- elamipretide, Placebo
- Keywords
- Heart failure, diuresis, dropsy, congestion, elamipretide, MTP-131, Bendavia™
- Medical subject headings
- Heart Failure, Edema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elamipretideDrug
Also called MTP-131, Bendavia
20 mg elamipretide administered intravenously once daily for 7 consecutive days
PlaceboDrug
Placebo administered intravenously once daily for 7 consecutive days
| Study group | Role | What they receive |
|---|---|---|
| 20 mg elamipretide | Experimental | 20 mg elamipretide once daily for 7 consecutive days Receives: Drug: elamipretide |
| Placebo | Placebo comparator | Placebo once daily for 7 consecutive days Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in NT-proBNP between Baseline and Day 8/Early Discharge
Measured: Baseline to Day 8
Secondary outcomes (10)
Number of patients staying in the same functional renal function class measured with MDRD formula compared to the number of patients with decreasing or increasing renal function measured with MDRD formula
Measured: Baseline to Day 3 and Day 8
Number of patients showing a decrease in body weight compared to baseline as well as number of patients showing either no decrease or an increase in body weight compared to baseline
Measured: Baseline to Day 3 and Day 8
Calculation of decrease or increase in body weight normalised to the average dose (in mg) of furosemide administered
Measured: Baseline to Day 3 and Day 8
The patient and physician global assessment is a 7-point scale which either the patient or the physician will assess from 0 to 10 and will be mathematically averaged on a daily basis in order to compare the different averages throughout the study.
Measured: Baseline to Day 3 and Day 8
The average daily dose of diuretic (furosemide - adjusted for thiazide dose if administered) between baseline and Day 3 and Day 8/Early Discharge
Measured: Baseline to Day 3 and Day 8
All adverse events, serious adverse events and SUSARs will be summarised per treatment group, namely elamipretide versus placebo, and compared at the end of the study.
Measured: Baseline to Day 8
Half-life (T1/2)
Measured: Baseline to Day 8
Area under the concentration - time curve from 0-24 h (AUC 0-24)
Measured: Baseline to Day 8
Area under the concentration - time curve from 0- infinity (AUC 0-∞)
Measured: Baseline to Day 8
Peak Plasma Concentration (Cmax)
Measured: Baseline to Day 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2016 (estimated)
- Study start20 Oct 2016
- Primary completion27 Nov 2017
- Study completion27 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heart FailureStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 308 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stealth BioTherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02914665
- Sponsor's protocol ID
- SPIHF-204
- Other IDs
- 2016-000126-19, 2016
- Status last verified
- Jul 2020
- First submitted
- 14 Sep 2016
- Last updated
- 28 Jul 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure
- ClinicalTrials.gov ID
- NCT02914665
- Acronym / sponsor ID
- SPIHF-204
- Conditions
- Heart Failure
- Keywords
- Heart failure, diuresis, dropsy, congestion, elamipretide, MTP-131, Bendavia™
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 308 participants (actual)
- Lead sponsor
- Stealth BioTherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Oct 2016 (actual)
- Primary completion
- 27 Nov 2017 (actual)
- Study completion
- 27 Nov 2017 (actual)
- First posted
- 26 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2 randomized, double-blind, placebo-controlled study to evaluate the cardiac and renal effects of short term treatment with elamipretide in patients hospitalized with congestion due to heart failure
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 |
|---|---|---|
| 20 mg elamipretide | Experimental | Drug: elamipretide20 mg elamipretide once daily for 7 consecutive days |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo once daily for 7 consecutive days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- A history of chronic heart failure for at least 1 month
- Treated with ≥40 mg/day of furosemide or bumetanide ≥1 mg/day or torasemide ≥10 mg/day for at least 1 month
- In-hospital observation/admission and treatment for ≤72 hours and primary cause for admission is heart failure with persistent congestion in the opinion of the Investigator (i.e. at least +2 pitting oedema and/or an estimated 8 kg gain in weight over baseline over the past 4 weeks) requiring intravenous loop diuretic therapy
- Sufficiently severe oedema to justify treatment by an intravenous infusion of furosemide of 10 mg/hour for at least 48 hours
- Systolic blood pressure >90 mmHg and considered to be haemodynamically stable, in the opinion of the Investigator
Key exclusion criteria (22 listed)
- Acute coronary syndrome, stroke, or transient ischemic attack (TIA), coronary or peripheral revascularization procedures, valve procedures, OR any major surgical procedure within the previous 6 weeks
- Invasive cardiac investigation and/or treatment (i.e. coronary angiography, percutaneous coronary intervention [PCI] or surgery) or other surgical procedure planned in the next 4 weeks
- Use of intravenous radiographic contrast agent within 72 hours prior to screening or planned use during the study
- Severe, in the investigators opinion, uncorrected valve disease or congenital heart disease as the cause for cardiac decompensation
- Acute mechanical cause of decompensated heart failure such as papillary muscle rupture
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 |
|---|---|---|
| elamipretideAlso: MTP-131, Bendavia20 mg elamipretide administered intravenously once daily for 7 consecutive days | Drug | 20 mg elamipretide |
| PlaceboPlacebo administered intravenously once daily for 7 consecutive days | Drug | Placebo |
Primary outcome
Change in NT-proBNP between Baseline and Day 8/Early Discharge
Time frame: Baseline to Day 8
10 secondary outcomes
Number of patients staying in the same functional renal function class measured with MDRD formula compared to the number of patients with decreasing or increasing renal function measured with MDRD formula
Time frame: Baseline to Day 3 and Day 8
Number of patients showing a decrease in body weight compared to baseline as well as number of patients showing either no decrease or an increase in body weight compared to baseline
Time frame: Baseline to Day 3 and Day 8
Calculation of decrease or increase in body weight normalised to the average dose (in mg) of furosemide administered
Time frame: Baseline to Day 3 and Day 8
The patient and physician global assessment is a 7-point scale which either the patient or the physician will assess from 0 to 10 and will be mathematically averaged on a daily basis in order to compare the different averages throughout the study.
Time frame: Baseline to Day 3 and Day 8
The average daily dose of diuretic (furosemide - adjusted for thiazide dose if administered) between baseline and Day 3 and Day 8/Early Discharge
Time frame: Baseline to Day 3 and Day 8
All adverse events, serious adverse events and SUSARs will be summarised per treatment group, namely elamipretide versus placebo, and compared at the end of the study.
Time frame: Baseline to Day 8
Half-life (T1/2)
Time frame: Baseline to Day 8
Area under the concentration - time curve from 0-24 h (AUC 0-24)
Time frame: Baseline to Day 8
Area under the concentration - time curve from 0- infinity (AUC 0-∞)
Time frame: Baseline to Day 8
Peak Plasma Concentration (Cmax)
Time frame: Baseline to Day 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2020.
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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.
The record lists no contact. Use the official record to reach the sponsor.
46 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing elamipretide in people with Heart Failure, run by Stealth BioTherapeutics Inc. at 46 sites in 10 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 8 inclusion and 22 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 28 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in Spain, Netherlands, Bulgaria, Serbia, Hungary and 5 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 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 NT-proBNP between Baseline and Day 8/Early Discharge (time frame: Baseline to Day 8). 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 28 Jul 2020.