Summary

for people ages 18-75 (full criteria)
at UC Davis UCLA
study started
estimated completion

Description

Summary

This study is an open-label extension study to determine the long-term safety and tolerability of APD811 in patients with WHO Group 1 PAH who have completed the Phase 2 study, APD811-003. Patients must have completed the APD811-003 study and must meet eligibility criteria for APD811-007.

Official Title

An Open-Label Extension Study of Ralinepag in Patients With Pulmonary Arterial Hypertension

Details

This study is an open-label extension study to determine the long-term safety and tolerability of APD811 in patients with WHO Group 1 PAH who have completed the Phase 2 Study APD811-003. Patients must have completed Study APD811-003 and met eligibility criteria for Study APD811-007. Additionally, placebo-treated patients who discontinue study drug treatment due to clinical worsening in Study APD811-003 will be permitted to enroll in Study APD811-007, upon approval of the medical monitor, provided that all end of study procedures including right heart catheterization are performed per protocol. The Week 25 Visit in Study APD811-003 will serve as the Baseline Visit for Study APD811-007.

All patients enrolled in Study APD811-007 will receive open-label treatment with APD811. The starting dose and titration schedule will be individually determined and in accordance with the starting dose and titration schedule optimized from Study APD811-003. Adjustments in the dose and titration schedule may be made according to patient tolerability.

After an individual patient completes Study APD811-003 and that patient's database is locked, patient unblinding will occur. Patients on active treatment (APD811) will remain on current dose and have onsite clinical assessments performed every 3 months until the patient is discontinued from the study.

Patients in the placebo treatment group will undergo a dose titration period until a stable, maximum tolerated dose (MTD) is reached (up to 9 weeks), followed by a treatment period after the MTD is determined during which monthly onsite clinic assessments will be performed for the first 3 months and then every 3 months until the patient is discontinued from the study or the study is terminated (see Table 2).

Discontinuation of the study may also occur at Sponsor's decision to terminate the study. Dose reductions may be made at any time for safety reasons.

Incremental dose increases will also be allowed during the Treatment Period at the discretion of the investigator (as clinically indicated) and according to the stepwise titration scheme. Uptitration should not occur within 6 weeks of an efficacy assessment.

Patients will be assessed for clinical worsening during each clinic visit. If clinical worsening is confirmed, the Investigator may opt to either continue treatment with APD811 at the current dose, increase the dose of APD811, interrupt treatment, or discontinue the patient at his/her discretion.

In addition, all patients will be contacted yearly following discontinuation in Study APD811-007 to assess mortality status. The mortality status follow-up contact of patients who withdraw consent will depend on local regulations or specific agreement with the subject and investigator.

After the last patient enrolled in Study APD811-007 has completed approximately 6 months of the study, a cumulative all-patient data analysis will be performed for all patients who entered the study. Patients will continue to have visits to the clinic every 3 months indefinitely to collect data until marketing approval of APD811 is granted or until the Sponsor discontinues the study. At the time of marketing approval or the Sponsor's decision to discontinue the study, all on-going patients will complete an End of Study Visit. A 28-day Follow-up Visit will be conducted to ensure appropriate subject safety.

Keywords

Pulmonary Arterial Hypertension Familial Primary Pulmonary Hypertension Hypertension Ralinepag

Eligibility

You can join if…

Open to people ages 18-75

  • Evidence of a personally signed and dated informed consent document
  • Is willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures and is deemed an appropriate candidate for participation in a long-term extension study and administration of APD811
  • Fulfilled all eligibility criteria for APD811-003 and completed the study as planned
  • Patients who discontinued for clinical worsening in APD811-003 and were assigned to placebo and completed all end of study procedures including right heart catheterization (RHC) may participate after their data from the APD811-003 study is cleaned and locked

You CAN'T join if...

  • Patients who enrolled in APD811-003 and were withdrawn from study drug treatment due to any AE, SAE, or clinical worsening if assigned to APD811, or patients who did not complete the APD811 003 study for other reasons.
  • Female patients who wish to become pregnant
  • Systolic BP <90 mmHg at Baseline/Day 1
  • Other severe acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study

Locations

  • David Geffen School of Medicine at UCLA
    Los Angeles California 90095 United States
  • University of California Davis Medical Center
    Sacramento California 95817 United States
  • Harbor-UCLA Medical Center
    Torrance California 90502 United States
  • Cedars-Sinai Medical Center
    Beverly Hills California 90211 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
United Therapeutics
ID
NCT02279745
Phase
Phase 2
Study Type
Interventional
Last Updated