Refer a patient to ATHENA
A Phase II, randomized, double-blind trial of tiprelestat (recombinant human elafin) added to background therapy in WHO Group 1 PAH. Randomization is 2:2:3 to 10 mg, 5 mg, or placebo, given subcutaneously once daily for 24 weeks with a 4-week withdrawal period.
Key inclusion criteria
- Age 18–75, WHO Group 1 PAH, WHO FC II–IV
- mPAP ≥ 25 mmHg; PAWP or LVEDP ≤ 15 mmHg
- PVR ≥ 400 dynes·sec/cm⁵ (5 Wood units)
- Stable PAH therapy ≥ 90 days
- 6MWD ≥ 100 m at screening
Key exclusion criteria
- WHO Group 2–5 pulmonary hypertension
- Significant left heart disease on screening echo
- eGFR < 30 mL/min/1.73 m²; Child-Pugh > 10
- TLC or FVC < 60% predicted
- PAH investigational agent within 3 months
How a referral works
1
Send the patient to the nearest center or call the hotline and the team will route it.
2
The site PI reviews eligibility and arranges screening, including a right heart catheterization.
3
You keep managing their PAH therapy; the site reports back after randomization.
Single Institutional Review Board: Advvarra · Independent Data and Safety Monitoring Board convened by NHLBI · Clinical Events Committee adjudicates endpoints · Funded by NHLBI (UG3HL180990, U24HL180994)