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New treatment option for Pulmonary Arterial Hypertension (PAH)
Official title A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)
ClinicalTrials.gov ID: NCT07175038
What this study is testing
What is ROC-101?
ROC-101 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for pulmonary arterial hypertension (pah).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). Each eligible participant will receive standard of care (SOC) plus ROC-101 for a 24-week treatment period, followed by a long-term extension period of the study through the end of the program or marketing approval/authorization.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 24 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Must be age 18 or older at the time of signing the informed consent form (ICF). The participant must understand and voluntarily sign an ICF prior to...
- Documented findings on a right heart catheterization (RHC) consistent with a diagnosis World Health Organization (WHO) Group 1 PAH or WHO GROUP 3 PAH
- Symptomatic Pulmonary Hypertension (PH) classified as WHO Functional Class II or III symptoms
- PAH participants: Pulmonary Vascular Resistance (PVR) of ≥ 5 Wood units, Pulmonary Capillary Wedge Pressure (PCWP) ≤ 15 mmHg and Mean Pulmonary...
- Participants on stable background therapy for PAH or ILD-PH.
You likely can't join if
- Diagnosis of PH WHO Groups 2, 4, or 5
- Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH, PAH associated with portal hypertension...
- Positive blood test for hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) antibody (HCVAb) (unless participants have had treatment...
- Participants with known hypersensitivity to ROC-101 or any components of its formulations
- History of malignancy within the last 5 years, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤...
- History of clinically significant (as determined by the Investigator) non-PAH related cardiac, endocrine, hematologic, hepatic, immune, metabolic...
See the full eligibility criteria
Who can join
- Must be age 18 or older at the time of signing the informed consent form (ICF). The participant must understand and voluntarily sign an ICF prior to any study-related procedures
- Documented findings on a right heart catheterization (RHC) consistent with a diagnosis World Health Organization (WHO) Group 1 PAH or WHO GROUP 3 PAH
- Symptomatic Pulmonary Hypertension (PH) classified as WHO Functional Class II or III symptoms
- PAH participants: Pulmonary Vascular Resistance (PVR) of ≥ 5 Wood units, Pulmonary Capillary Wedge Pressure (PCWP) ≤ 15 mmHg and Mean Pulmonary Arterial Pressure (mPAP) \> 20 mm Hg and ILD-PH participants: PVR of ≥ 3...
- Participants on stable background therapy for PAH or ILD-PH.
- Females of childbearing potential (as defined in protocol) must agree to use highly effective contraception (as defined in the protocol)
- Male participants must follow protocol-specified contraception guidance.
- Participants must be able to communicate well with Investigators, understand the study procedures in the ICF and are agreeable to complete the study in accordance with the protocol.
- Must be able to swallow tablets.
- Pulmonary function tests (PFT): PAH participants at Screening as follows:
- Forced Vital Capacity (FVC) \> 70% predicted; or if between 60% to 70% predicted, or if not possible to be determined, confirmatory High-Resolution Computed Tomography (HRCT) indicating no more than mild (\<10%...
- The ratio of FEV1 (first second)/FVC \> 0.70 of predicted. ILD-PH participants at Screening as follows:
- PFTs consistent with their ILD diagnosis and showing FEV1/ FVC ratio \> 65% and HRCT \> 10% fibrosis, based on the proportion of lung parenchyma affected by fibrotic changes. and,
- Minimum FVC of 50% and diffusing capacity for carbon monoxide (DLCO) (corrected for Hb g/dl) \>25%
- In PAH participants, i.e., Cohorts 1 and 2 only, ventilation-perfusion (VQ) scan (or, if unavailable, a negative CT pulmonary angiogram [CTPA] or pulmonary angiography result), any time prior to Screening or conducted...
- Acceptable Electrocardiogram (ECG) findings as assessed by the Investigator or qualified designee at the Screening Visit and at the Baseline Visit (Day 1), including each criterion as listed below:
- Normal sinus rhythm (HR) between 40 and 100 beats per minute, inclusive);
- Corrected QT Interval (QTcF) interval ≤ 450 msec (males) and ≤ 460 msec (females);
- QRS interval ≤ 120 msec; if \> 120 msec, result will be confirmed by a manual over read
- Body weight at the Screening visit and at Baseline (Day 1) is greater than 50.0 kg and the body mass index (BMI) is in the range of 19.00 to 36.00 kg/m2, inclusive
- 6MWD ≥ 100 and ≤ 550 meters repeated twice, once during Screening Period and once at the Baseline Visit (Day 1) and both values within 15% of each other, allowing for a third repeat if \> 15% difference, calculated from...
What rules you out
- Diagnosis of PH WHO Groups 2, 4, or 5
- Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH, PAH associated with portal hypertension, schistosomiasis-associated PAH and pulmonary veno-occlusive disease and/or...
- Positive blood test for hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) antibody (HCVAb) (unless participants have had treatment for HCV and have a negative HCV ribonucleic acid [RNA] polymerase chain...
- Participants with known hypersensitivity to ROC-101 or any components of its formulations
- History of malignancy within the last 5 years, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
- History of clinically significant (as determined by the Investigator) non-PAH related cardiac, endocrine, hematologic, hepatic, immune, metabolic, urologic, pulmonary, neurologic, neuromuscular, dermatologic...
- Participation in another clinical trial involving intervention with another investigational drug, approved therapy for investigational use, or investigational device within 4 weeks prior to Baseline Visit (unless it is...
- Major surgery within 8 weeks prior to Baseline Visit (Day 1) or major surgery scheduled or planned in the main study. Participants must have completely recovered from any previous surgery prior to the Screening Visit
- Prior heart or heart-lung transplants, or a participant listed for heart and/or lung transplantation or prior pneumonectomy
- Pregnant or breastfeeding females
- Males who do not agree to protocol contraception guidelines
- Uncontrolled systemic hypertension as evidenced by sitting SBP \> 160 mm Hg or sitting diastolic BP \> 100 mm Hg during Screening Visit and Baseline Visit (Day 1) after a period of rest
- Systolic BP \< 90 mm Hg during Screening Visit or at Baseline Visit (Day 1)
- History of known pericardial constriction or a clinically significant (more than trace or trivial [i.e., ≥10 mm]) pericardial effusion seen in diastole or in both systole and diastole on echocardiogram (ECHO)...
- RHC contraindicated during the study per Investigator
- Cerebrovascular accident within 3 months (120 days) of start of Screening
- History of restrictive or constrictive or congestive cardiomyopathy
- Left ventricular ejection fraction (LVEF) \< 50% on historical echocardiogram (ECHO) performed within 6 months prior to start of Screening period (and confirmed during the Screening ECHO) or grade 2 or higher diastolic...
- Any current symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain in the past 6 months (180 days) prior to start of...
- History of acutely decompensated left heart failure or right heart failure within 90 days prior to Baseline, as per Investigator assessment
- Significant (≥ 2+ [or \> mild] regurgitation) mitral regurgitation or aortic regurgitation valvular disease, or more than mild mitral stenosis or aortic stenosis valvular disease as seen on Screening ECHO
- Started or stopped receiving any general supportive therapy for PH (e.g., oxygen, anticoagulants, digoxin) within 30 days prior to start of Screening
- Use of supplemental oxygen \> 10 liters/minute and SaO2 \< 90% while receiving typical oxygen supplementation
- Received intravenous (IV) inotropes (e.g., dobutamine, dopamine, norepinephrine, vasopressin) within 30 days prior to start of Screening
- History of atrial septostomy within 180 days prior to start of Screening
- History of portal hypertension or chronic liver disease, defined as mild to severe hepatic impairment (Child-Pugh Classes A to C)
- Untreated, severe (defined as apnea hypoxia index of \> 30) obstructive sleep apnea
- Active daily smoker of cannabis or tobacco
- Current alcohol abuse or current illicit drug use
- WHO Group 3 due to severe chronic obstructive pulmonary disease (COPD) or chronic pulmonary fibrosis and emphysema (CPFE) or PFT with FVC \< 50% or FEV1/FVC \< 65% or DLCO \< 25% (corrected for Hb g/dl)
- Presence of lab abnormalities at Screening
- History of greater than severe renal disease, including any episode of acute renal failure, with or without a prior history of renal disease in which acute dialysis (e.g., intermittent hemodialysis or continuous...
- Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to Baseline or planned initiation during the study (participants who are stable in the maintenance phase of a program and who...
- Participants who plan to continue, or start during the study, medications which are sensitive cytochrome (CYP) 2D6 substrates with a narrow therapeutic index, such as nortriptyline, venlafaxine, and amitriptyline or...
- History or presence of impaired cardiac function
- Participants who plan to donate blood after signing consent for the study and for 28 days after their last dose of study drug Key Inclusion Criteria for Extension Period:
- Participants must complete the main study period (defined as completion of assessments through the Week 24 visit)
- Women of child-bearing potential (WOCBP) must have negative pregnancy test
- All participants must comply with contraceptive guidance until 28 days after last dose of study drug for WOCBP and 90 days after the last dose of study drug for males Key Exclusion Criteria for Extension Period:
- Participant withdrew from main study period due to an AE related to study drug
- Female participant who is pregnant, breastfeeding, or intends to conceive during the long-term extension period
- Males who do not agree to follow protocol contraception guidelines
- Any condition that in the opinion of the investigator may pose a risk to the participant, interferes with the participant's participation or confounds assessments of the participant
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Rancho Cordova, California, United States
- Aurora, Colorado, United States
- Washington D.C., District of Columbia, United States
- Jacksonville, Florida, United States
- Kansas City, Kansas, United States
- Ann Arbor, Michigan, United States
- Columbus, Ohio, United States
- Bend, Oregon, United States
- Philadelphia, Pennsylvania, United States
- Camperdown, New South Wales, Australia
- Auchenflower, Queensland, Australia
- Calgary, Alberta, Canada
- London, Ontario, Canada
- Le Kremlin-Bicêtre, Val-de-Marne, France
- Hanover, Lower Saxony, Germany
- Pavia, Lombardy, Italy
- Riga, Latvia
- Madrid, Madrid, Spain
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 24 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Rancho Cordova, California, United States; Aurora, Colorado, United States; Washington D.C., District of Columbia, United States; Jacksonville, Florida, United States; Kansas City, Kansas, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.