NCT02927366

Safety, Pharmacokinetics and Efficacy Study of QCC374 in PAH Patients

A Randomized, Parallel-group, Placebo-controlled Subject and Investigator Blinded Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of QCC374 in the Treatment of Pulmonary Arterial Hypertension

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a non-confirmatory, randomized, placebo controlled, subject and investigator blinded study of QCC374 in PAH subjects. The study was planned to have 2 Parts: Part 1, an initial safety cohort with a 0.03 mg bid starting dose, and Part 2, a larger cohort with a 0.06 mg bid starting dose. However, due to early study termination following Part 1, Part 2 was not completed. Both study parts were comprised of four phases: a screening period for up to 28 days, a titration period of 2 weeks, a stable dose period of 14 weeks and safety follow-up period for 28 days. At the end of the treatment period of 16 weeks, eligible patients were given the option to participate in a separate long-term extension study (CQCC374X2201E1 (NCT02939599)), where all patients were treated with an individual optimal dose of QCC374.

Detailed description

The decision for early termination was based on changes in Novartis strategy, and was not based on any safety concerns regarding QCC374. Only Part 1 of the study was completed.

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	QCC374	0.03 mg (2 capsules of 0.015 mg) BID 0.06 mg (1 capsule of 0.06 mg) BID 0.12 mg (2 capsules of 0.06 mg) BID
DRUG	Placebo Matching	Placebo matching to QCC374: 0.03 mg BID, 0.06 mg BID and 0.12 mg BID

Timeline

• Start date: 2017-09-19
• Primary completion: 2018-06-07
• Completion: 2018-06-07
• First posted: 2016-10-07
• Last updated: 2021-01-05
• Results posted: 2019-08-07

Locations

5 sites across 4 countries: United States, Germany, South Korea, United Kingdom

Regulatory

• FDA-regulated drug study