💬In plain words
What the trial is, and whether it could suit you
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.
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Key Inclusion Criteria:
1. 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
2. Documented findings on a right heart catheterization (RHC) consistent with a diagnosis World Health Organization (WHO) Group 1 PAH or WHO GROUP 3 PAH
3. Symptomatic Pulmonary Hypertension (PH) classified as WHO Functional Class II or III symptoms
4. PAH participants: Pulmonary Vascular Resistance (PVR) of ≥ 5 Wood units (≥ 400 dynes/sec/cm-5) and, Pulmonary Capillary Wedge Pressure (PCWP) ≤ 15 mmHg and Mean Pulmonary Arterial Pressure (mPAP) \> 20 mm Hg and ILD-PH participants: PVR of ≥ 3 Wood units (≥ 240 dynes/sec/cm-5) with a maximum enrollment of 5 participants with PVR between 3 and 4 Wood units inclusive (240-320 dynes/sec/cm-5 inclusive) and the remaining 5 participants must have PVR \>4 Wood units (\> 320 dynes/sec/cm-5) and, PCWP ≤ 15 mmHg and mPAP \> 20 mm Hg
5. Participants on stable background therapy for PAH or ILD-PH.
6. Females of childbearing potential (as defined in protocol) must agree to use highly effective contraception (as defined in the protocol)
7. Male participants must follow protocol-specified contraception guidance.
8. 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.
9. Must be able to swallow tablets.
10. Pulmonary function tests (PFT):
PAH participants at Screening as follows:
1. 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% fibrosis) ILD and emphysema (less than 10%); and
2. The ratio of FEV1 (first second)/FVC \> 0.70 of predicted.
ILD-PH participants at Screening as follows:
1. PFTs consistent with their ILD diagnosis and showing FEV1/ FVC ratio \> 65% and HRCT having more than 10% fibrosis and less than 10% emphysema, based on the proportion of lung parenchyma affected by fibrotic and emphysema changes.
and,
2. Minimum FVC of 50% and diffusing capacity for carbon monoxide (DLCO) (corrected for Hb g/dl) \>25%
11. In PAH participants, i.e., Cohorts 1 and 2 only, ventilation-perfusion (VQ) scan (or, if unavailable, a negative Computed tomography pulmonary angiogram \[CTPA\] or pulmonary angiography result), any time prior to Screening or conducted during the Screening Period, with a normal or low probability result that is not clinically significant
12. 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 overread
13. 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
14. 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 the higher/highest value
Key Exclusion Criteria:
1. Diagnosis of PH WHO Groups 2, 4, or 5
2. 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 pulmonary capillary hemangiomatosis
3. 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 reaction \[PCR\]) or HIV antibody
4. Participants with known hypersensitivity to ROC-101 or any components of its formulations
5. 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
6. History of clinically significant (as determined by the Investigator) non-PAH related cardiac, endocrine, hematologic, hepatic, immune, metabolic, urologic, pulmonary, neurologic, neuromuscular, dermatologic, psychiatric, renal, and/or other diseases that may limit participation in the study
7. 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 in the follow-up period of an interventional study), or if the half-life of the previous product is known, within 5× the half-life prior to Baseline Visit (Day 1), whichever is longer
8. 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
9. Prior heart or heart-lung transplants, or a participant listed for heart and/or lung transplantation or prior pneumonectomy
10. Pregnant or breastfeeding females
11. Males who do not agree to protocol contraception guidelines
12. 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
13. Systolic BP \< 90 mm Hg during Screening Visit or at Baseline Visit (Day 1) or, if during screening, orthostatic systolic BP decreases by more than 20 mm Hg and the participant is symptomatic.
14. History of known pericardial constriction or a clinic