TEPH: Telaglenastat Efficacy in Pulmonary Hypertension
A Phase 2a Single Arm Open Label Safety and Efficacy Study of Telaglenastat Plus Standard of Care in Adults With Functional Class Ii-iii Precapillary Pulmonary Hypertension Across Wsph Groups 1-3
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years to 75 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Visits mentioned: 12 visits
- Procedures mentioned: Physical exams, Questionnaires, Blood draws, Heart tracing (ECG)
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Screening Visit (30-60 mins) * Consenting * Medical history, physical exam, lab tests (blood, urine) * Review of past heart/lung records * Surveys 2."
- "Baseline Visit (Visit 2, 2-3 hrs) * Physical exam, 6-minute walk test * Echo, Right Heart Catheterization (RHC) * High-Resolution CT (HRCT) \& Pulmonary Function Tests (PFTs) if applicable * Lab tests, ECG, glutamine blood sample * Surveys 3."
- "Treatment Phase (Visits 3-6, Weeks 1,2,4,8) Telaglenastat 800 mg/twice daily Short visits (30 mins) for: * Physical Exam * Surveys * Labs 4."
- "Visit 7 End of Treatment (week 12, 2-3 hours) include the following: * Physical Exam * 6 Minute Walk Test * Echo/RHC/ECG * HRCT/PFT group 3 * Labs * Surveys * Optional Extension (Visits 8-12, Weeks 13-24) Same procedures as initial 12 weeks Final visit includes full reassessment"
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests whether telaglenastat can lower blood pressure in the lungs and help the heart and lungs work better. It is for adults ages 18–75 with pulmonary hypertension (PH), or high blood pressure in the lung arteries. Telaglenastat is not FDA-approved for PH and will not be available to participants after the study.
The research study is being conducted to evaluate the effectiveness of a drug called Telaglenastat in adults diagnosed with Pulmonary Hypertension (PH). PH is a progressive condition that affects the arteries in the lungs, specifically the pulmonary arteries, which carry blood from the right side of the heart to the lungs. Telaglenastat is not currently approved by the Food and Drug Administration for the treatment of PH. However, the study investigators believe that Telaglenastat may help lower blood pressure in the lungs and improve both heart and lung function. It is important to note that the drug will not be available to participants once the study concludes.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be 18–75 years old, give written consent, and be able to complete study visits, exercise tests, and a heart catheter test that measures pressure.
- Have PH with symptoms during activity, rated as class II or III by the study team.
- For Group 1 or Group 3 PH, have heart catheter test results that meet the study’s lung pressure and blood flow limits, with stable treatment for at least 30 days.
- For Group 2 PH with heart failure, have a heart pumping strength of at least 50%, certain heart scan and pressure test results, and stable heart failure treatment for at least 30 days.
- Have a body mass index (BMI), a measure of weight compared with height, of 18–40. If BMI is over 35, chest size must be under 65 inches. Walk at least 100 but less than 550 meters in 6 minutes.
- For Group 1 PH, be on PH treatment. Doses of drugs that widen blood vessels or sotatercept must be stable for at least 3 months before study group assignment.
- For Group 3 PH with lung scarring, have a lung CT scan from the past year confirming the disease. Certain patients need extra lung tests. If taking lung disease or PH drugs, doses must be stable for at least 30 days.
- If able to become pregnant, use effective birth control during the study and for 30 days after the last dose.
Who can't join
- Have Group 1 PH without prior PH drug treatment, or Group 3 PH with lung scarring and recent use of certain PH drugs not allowed by the study.
- Have certain other main causes of PH, significant leaking heart valves, a heart defect present at birth, heart pumping strength below 50%, or untreated moderate to severe sleep apnea.
- Have uncontrolled high blood pressure, repeated very low blood pressure, low blood pressure with clear symptoms, or a repeatedly fast resting pulse over 120 beats per minute.
- Have low platelets, which help blood clot; a bleeding disorder; serious long-term blood clot disease; or a blood clot in the lungs within 6 months.
- Have severe kidney or liver problems, long-term pancreas disease, porphyria, mitochondrial disease, or urea cycle disease. The last three are disorders that affect how the body processes substances or makes energy.
- Be pregnant or breastfeeding, have active COVID-19, or be allergic to the study drug or its ingredients. Past COVID-19 is allowed.
- Have had lung reduction surgery, expect a lung transplant within 6 months, or have recent major surgery, injury, or a hospital stay over 23 hours. Planned surgery or participation in certain other studies or exercise-based heart or lung rehab programs may also prevent joining.
- Have another health or mental health condition, or recent substance or alcohol abuse, that the study doctor believes could make participation unsafe or affect study results. A condition expected to limit life to 1 year or less also prevents joining.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Male or female age 18- 75 years old. 2. Able to provide written informed consent. 3. Able to comply with study procedures, able to undergo cardiac catheterization and exercise testing. 4. For Group 1and 3 PH, prior right heart catheterization (RHC) should show documented diagnosis of precapillary PH at mean pulmonary arterial pressure (mPAP \> 20 mm Hg, PCWP\<15 mm Hg, and PVR\>3.5 WU) at screening.. Minimum pulmonary vascular resistance (PVR) of \>3.5 Wood units by RHC at screening AT screening and on at least 30 days of stable medical therapy. 5. For Group 2 PH-HFpEF, documented transthoracic echocardiogram or cardiac MRI with LV ejection fraction ≥50%, along with meeting at least one of the following criteria by echo: Echocardiographic/MRI evidence of (1) diastolic dysfunction, (2) left atrial enlargement (LA diameter \>3.6 cm). Prior right heart catheterization data indicating PCWP\>15 mm Hg, mPAP \> 20 mm Hg and PVR\>3.5 WU. Stable heart failure therapy for at least 30 days. 6. Symptomatic PH classified as WHO functional class II or III. 7. Body mass index (BMI) 18 to 40 kg/m2 at Screening. If BMI is \>35 kg/m2, subject chest circumference should be \<65 inches (165cm). 8.6-minute walk distance (6MWD) ≥100 meters (m) and \<550 m at Screening. 9.For Group 1 PH, patients on SOC medical treatment for PH with vasodilators or sotatercept are required to have been receiving a stable dose for at least 3 months before undergoing randomization. 10.For Group 3 PH-ILD, documented diagnosis of presence of interstitial lung disease (diffuse parenchymal lung disease) by high resolution lung CT scan within 1year of randomization. Patients with Group 3 PH with connective tissue disease should have confirmed baseline FVC \<70% within 1 year of randomization. For subjects with a history of lobectomy or pneumonectomy, and for whom there are no population-based normalization methods, assessment based on residual lung volume will be permitted to assess eligibility. Patients receiving drug treatment (i.e., pirfenidone or nintedanib) for their underlying lung disease or pulmonary hypertension (i.e., inhaled Treprostinil, tadalafil or sildenafil) should receive a stable dose for at least 30 days before undergoing randomization. 11.Women of childbearing potential must be willing and able to practice medically acceptable effective contraception during the study and continuing contraception for 30 days after their last dose of study drug. Women who are surgically sterile or those who are post-menopausal for at least 2 years are not considered to be of childbearing Exclusion Criteria: 1. Group 3 PH-ILD, patients receiving approved therapies other than inhaled Treprostinil, tadalafil or sildenafil for PAH less than 30 days before randomization are not eligible for enrollment. 2. Group 1 PH, patients naïve to medical treatment for PH are not eligible for enrollment. 3. History of lung reduction surgery or likely to undergo lung transplantation within the next 6 months. 4. Enrolled in, or planned participation in, device or other interventional clinical studies or cardio-pulmonary rehabilitation programs, based upon exercise within 90 days of Screening or during study participation. 5. Patients with other secondary causes of PH including, but not limited to, left or right heart failure, valvular heart disease, chronic obstructive lung disease, atrial septal defect with left to right shunt, and sleep apnea will be excluded if it was the primary cause of PAH. 6. Diagnosed with significant (≥2+ regurgitation) mitral regurgitation or aortic regurgitation. 7. Adult congenital heart disease (ACHD) 8. Left ventricular ejection fraction (LVEF) \<50%. 9. Uncontrolled hypertension (SBP\>160 mm Hg, DBP\>90). 10. Sustained systolic blood pressure (SBP) \<95 mmHg and/or diastolic blood pressure (DBP) \<50 mmHg (confirmed by duplicate seated readings) on at least 3 consecutive occasions (self-monitored or office) prior to or at Screening, or overt symptomatic hypotension. 11. Sustained resting heart rate (HR) \>120 beats per minute (confirmed by duplicate assessments of office vital signs) or consecutive electrocardiogram (ECG) assessments on at least 3 consecutive occasions prior to or at Screening. 12. Concomitant medical or psychiatric disorder, condition, history, or any other condition that, in the opinion of the Investigator, would either put the participant at risk or impair their ability to participate in or complete the requirements of the study or confound the objectives of the study. 13. Concomitant medical disorder that is expected to limit the subject's life-expectancy to ≤1 year. 14. Untreated, moderate to severe obstructive sleep apnea 15. Evidence of thrombocytopenia (platelets \<150,000/mm3, significant chronic thromboembolic disorder, or recent pulmonary embolism within 6 months prior to Screening. 16. History of a bleeding disorder. 17. Known porphyria, mitochondrial, or urea cycle disease. 18. History of chronic pancreatic disease 19. Pregnant or lactating female. 20. Active coronavirus disease 19 (COVID-19); however, those with previous COVID-19 are permitted. 21. Participated in another investigational drug study within 30 days prior to Screening or is participating in a non-medication study which, in the opinion of the Investigator, would interfere with the study compliance or outcome assessments 22. Glomerular Filtration Rate (GFR) of \<30 mL/min/1.73m2. 23. Significant liver dysfunction as measured by any one of the following at Screening (including subjects with acute or chronic hepatitis as well as subjects with own or family history of serious hepatitis, especially drug related): * Alanine aminotransferase (ALT) \>2.0 × upper limit of normal (ULN) * Aspartate aminotransferase (AST) \>2.0 × ULN * Serum bilirubin ≥1.6 mg/dL or \>2.0 × ULN 24. Known history of substance abuse including alcohol abuse within the 1 year prior to Screening that in the opinion of the Investigator would impair the subject's ability to participate in or complete the requirements of the study 25. Any major surgical procedure or trauma within 30 days prior to Screening or planned surgical procedure during the study period. 26. Any inpatient hospitalization (defined as \>23 hours) within 30 days prior to screening. 27. Known hypersensitivity to study drug or any of the excipients of the drug formulation. 28. The discretion of the primary investigator
Study sites (1)
PA
- UPMC Presybeterian · Pittsburgh, PA 15213Yassmin Al Aaraj, MPH · Study contact · 4126479227 · yaa29@pitt.eduMore trials in Pittsburgh