To Evaluate the Safety, Tolerability and Efficacy of R2R01 Combined With SOC as Compared to SOC Alone in Outpatients With Worsening Heart Failure (WHF)

NCT ID: NCT06264310

Last Updated: 2024-11-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

33 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-01

Study Completion Date

2024-11-25

Brief Summary

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This Study Aims to Evaluate the Safety, Tolerability and Efficacy of R2R01 Combined With SOC as Compared to SOC Alone in Outpatients With Worsening Heart Failure (WHF)

Detailed Description

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This is a Phase 2, Double-Blind, Placebo-Controlled, Dose Range Finding Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of the Relaxin Agonist R2R01 combined with Standard of Care versus Standard of Care Alone in Outpatients with Worsening Heart Failure (WHF). Each patient will undergo a screening period of up to 21 days, an active treatment period of 7 days, and a follow up period of 23 days after the last dose (30 days after the first dose), i.e., a total of up to 51 days total study duration.

This study is a four-parallel-cohort study. All patients will be randomized to one of the following Cohorts:

1. Cohort 1 (N=8): Placebo once daily SC for 7 days.
2. Cohort 2 (N=8): 5.0 mg R2R01 once daily SC for 7 days.
3. Cohort 3 (N=8): 7.5 mg R2R01 once daily SC for 7 days.
4. Cohort 4 (N=8): 10.0 mg R2R01 once daily SC for 7 days. All patients will present with worsening heart failure with congestion requiring SOC. The patient, in the opinion of the investigator, should be able to be treated for their symptoms without being admitted to the hospital. The planned 3-day hospitalization is to adequately manage any potential sign of hypotension and should not be required to treat the patient for the intensification of the diuretic therapy or same day IV loop diuretics. During the treatment period no new treatments for WHF should be initiated. This study will be conducted across approximately 8 centers in the US.

Conditions

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Heart Failure Worsening Heart Failure (WHF)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Patients will be randomly assigned to one of four cohorts. Three groups will receive R2R01, and one group will receive placebo.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Treatment of all cohorts will be double-blind (all patients, study staff, investigators and Sponsor). Site pharmacist is unblinded.

Study Groups

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Placebo

8 study subjects will receive placebo once daily subcutaneous injection for 7 days.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching placebo SC injection

5.0 mg R2R01

8 study subjects will receive 5.0 mg R2R01 once daily subcutaneous injection for 7 days.

Group Type EXPERIMENTAL

R2R01

Intervention Type DRUG

Pharmaceutical Form: sterile 2R vials containing 10 mg of R2R01 for SC injection.

7.5 mg R2R01

8 study subjects will receive 7.5 mg R2R01 once daily subcutaneous injection for 7 days.

Group Type EXPERIMENTAL

R2R01

Intervention Type DRUG

Pharmaceutical Form: sterile 2R vials containing 10 mg of R2R01 for SC injection.

10.0 mg R2R01

8 study subjects will receive 10.0 mg R2R01 once daily subcutaneous injection for 7 days.

Group Type EXPERIMENTAL

R2R01

Intervention Type DRUG

Pharmaceutical Form: sterile 2R vials containing 10 mg of R2R01 for SC injection.

Interventions

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Placebo

Matching placebo SC injection

Intervention Type DRUG

R2R01

Pharmaceutical Form: sterile 2R vials containing 10 mg of R2R01 for SC injection.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Able and willing to give written informed consent, and able to follow instructions and comply with follow-up procedures.
2. History of symptomatic HF (heart failure).
3. Male or female ≥ 18 years of age at screening.
4. Previous hospitalization for HF within the last 12 months prior to screening.
5. Patients on optimal background therapy as per local practice for at least 30 days prior to screening and tolerating this well.
6. Patients must present with at least 2 of the following signs / symptoms of

WHF congestion:
1. Dyspnea
2. Orthopnea
3. Fatigue
4. Jugular venous distension
5. Rales
6. Edema
7. Patient requires intensification (doubling the dose, adding another diuretic targeting another tubular segment) of oral diuretics, or IV diuresis for WHF as per Investigator at screening and treated as an outpatient patient.
8. Estimated Glomerular Filtration Rate (eGFR) between 20 and 75 mL/ min/1.73 m2 (calculated using the CKD-EPI equation) at screening.
9. NT-pro-BNP levels at screening:

1. ≥ 1000 pg/mL for patients with LVEF ≤ 40%
2. ≥ 700 pg/mL for patients with LVEF \> 40%
3. ≥ 1500 pg/mL for patients with current atrial fibrillation, regardless of LVEF. For patients with BMI ≥ 30 kg/m2 the NT-pro-BNP values are lowered by 20%. The values are as follows:

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1. ≥ 800 pg/mL for patients with LVEF ≤ 40%
2. ≥ 560 pg/mL for patients with LVEF \> 40%
3. ≥ 1200 pg/mL for patients with current atrial fibrillation, regardless of LVEF. The most recent LVEF value assessed in the 12 months prior to screening should be used.
10. Systolic Blood Pressure (SBP) ≥ 105 mmHg at screening.
11. Willing and able to stay in the clinic for observation/monitoring for 3 days.
12. Willing to self-administer all SC injections.

Exclusion Criteria

1. Patients with blood pressure \> 180 mmHg or persistent heart rate \> 130 bpm at Screening.
2. History of symptomatic hypotension.
3. History of orthostatic hypotension.
4. Temperature \> 38.5°C (oral or equivalent) or sepsis or active infection requiring antimicrobial treatment.
5. Clinical evidence of acute coronary syndrome (ACS) currently or within 30 days prior to Screening.
6. Acute Heart Failure (AHF) due to significant arrhythmias, which include any of the following: sustained ventricular tachycardia, bradycardia with sustained ventricular rate \< 45 bpm or atrial fibrillation/flutter with sustained ventricular response of \> 130 bpm.
7. The daily use of IV or oral steroids (including but not limited to the use of IV or oral steroids for respiratory disorders or COPD). Note, inhaled steroids are allowed.
8. IV antimicrobial treatment for sepsis or active infection.
9. Patients with severe renal impairment defined at Screening eGFR \< 20 mL/min/1.73m2 (calculated using the CKD-EPI Equation), and/or those receiving current or planned dialysis or ultrafiltration.
10. Patients with hemoglobin \< 10 g/dL, or a history of blood transfusion within the 14 days prior to screening, or active life-threatening gastrointestinal bleeding.
11. Known hepatic impairment (as evidenced by total bilirubin \> 3 mg/dL, or increased ammonia levels, if performed) or history of cirrhosis with evidence of portal hypertension such as varices.
12. Significant, uncorrected, left ventricular outflow obstruction, such as obstructive hypertrophic cardiomyopathy or severe aortic stenosis (i.e., aortic valve area \< 1.0 cm2 or mean gradient \> 40 mmHg on echocardiogram), and/or severe mitral stenosis.
13. Severe aortic insufficiency or severe mitral regurgitation for which surgical or percutaneous intervention is indicated.
14. Documented, prior to or at the time of screening, restrictive amyloid myocardiopathy, or acute myocarditis or hypertrophic obstructive, restrictive, or constrictive cardiomyopathy.
15. Any major solid organ transplant recipient or planned/ anticipated organ transplant within 1 year.
16. Major surgery, including implantable devices (e.g., implantable cardioverter defibrillator, cardiac resynchronization therapy), or major neurologic event including cerebrovascular events, within 30 days prior to screening.
17. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past year with a current life expectancy less than 1 year due to the malignancy.
18. Pregnant or nursing (lactating) women, where pregnancy was defined as the state of a female after conception and until the termination of gestate, confirmed by a positive human chorionic gonadotropin laboratory test.
19. Use of other investigational drugs within 30 days prior to Screening.
20. History of known hypersensitivity/prior exposure to R2R01 or serelaxin.
21. Any other medical condition(s) that might put the patient at risk or influence study results in the Investigator's opinion, or that the Investigator deems unsuitable for the study including drug or alcohol abuse or psychiatric, behavioral, or cognitive disorders sufficient to interfere with the patient's ability to understand and comply with the protocol instructions or follow-up procedures.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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International HealthCare, LLC

OTHER

Sponsor Role collaborator

River 2 Renal Corp.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic

Phoenix, Arizona, United States

Site Status

California Pacific Medical Center

San Francisco, California, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

Piedmont Hospital Transplant

Atlanta, Georgia, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

University of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

VA North Texas Health Care

Dallas, Texas, United States

Site Status

Baylor Scott and White All Saints Medical Center

Fort Worth, Texas, United States

Site Status

University of Duisburg-Essen

Essen, North Rhine-Westphalia, Germany

Site Status

University of Munster

Münster, North Rhine-Westphalia, Germany

Site Status

University of Bologna

Bologna, , Italy

Site Status

University-Hospital of Padova

Padua, , Italy

Site Status

Countries

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United States Germany Italy

Other Identifiers

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R2R01-WHF-201

Identifier Type: -

Identifier Source: org_study_id