NCT04316143

Pharmacokinetics, Safety and Efficacy of BIA 5-1058 in PAH (Zamicastat)

An Open-label, Multicentre Study to Evaluate Pharmacokinetics, Safety and Efficacy of Zamicastat as Adjunctive Therapy in Pulmonary Arterial Hypertension (PAH)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Bial - Portela C S.A. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This Study evaluates the pharmacokinetic (PK) profile of different zamicastat doses in Pulmonary arterial hypertension (PAH) patients to find the most promising therapeutic dosage range for the treatment of PAH disease

Detailed description

This is an open-label, multi-centre study in patients with PAH who are currently on stable treatment with at least one PAH medication. It is planned to evaluate the PK profile (24 hour profile and trough levels) and the safety, tolerability and efficacy of four different zamicastat doses. Each patient will start treatment with the lowest dose (50 mg zamicastat once daily) and the dose will be up-titrated to the individual highest tolerated dose (HTD) i.e. up to 200 mg zamicastat once daily. A data safety monitoring board (DSMB) will periodically review the safety data and will issue a recommendation if the doses can be used as planned. This study will consist of: * A screening period, 5 to 12 days: visit V1 * Up to four dose finding periods, 14 days each: * Dose A (50 mg zamicastat once daily): visits A1, A2 and A3 * Dose B (100 mg zamicastat once daily): visits B2 and B3 * Dose C (150 mg zamicastat once daily): visits C2 and C3 * Dose D (200 mg zamicastat once daily): visits D2 and D3 * Maintenance period, 42 days: maintenance period visit (MPV)1, MPV2 and MPV3 * Follow-up (FU) period, 14 to 28 days: visits FU (down-titration) and FU

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	Oral zamicastat	Tablets for oral administration under fed conditions containing 100 mg of zamicastat

Timeline

• Start date: 2019-06-03
• Primary completion: 2021-10-20
• Completion: 2021-10-20
• First posted: 2020-03-20
• Last updated: 2024-10-15
• Results posted: 2024-10-15

Locations

13 sites across 7 countries: Austria, Germany, Italy, Portugal, Spain, Ukraine, United Kingdom