NCT05224531

Effect of CS1 in Subjects With Pulmonary Arterial Hypertension

A Phase 2, Prospective, Randomized, Open-label, Blinded Endpoint, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of 3 Doses of CS1 in Subjects With Pulmonary Arterial Hypertension

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Cereno Scientific AB · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, parallel group study to evaluate the safety, tolerability, PK, and exploratory efficacy of 3 doses of CS1 in the treatment of PAH using the CardioMEMS HF System to obtain repeated measurements of PAP and other hemodynamic parameters. Elegible subjects will have a RHC to implant the CardioMEMS pulmonary artery (PA) Sensor followed by a Baseline Period for the subject to become familiar with the system, its measurements, how to send the data, and establish Baseline PA pressure. Alternately, the subject may already have the CardioMEMS HF System and is willing to have the system recalibrated in conjunction with RHC. Thereafter, the subject will be randomly assigned to 1 of 3 total daily doses of CS1 1:1:1; there will be 10 subjects assigned to each dose level. Subjects will receive study drug treatment for 12 weeks. During the study, mPAP and other hemodynamic parameters from CardioMEMS PA Sensor will be measured and data captured once daily in the morning before the subject gets out of bed. The data will be transferred electronically to a repository. The analysis will look at the area under the curve (AUC) of mPAP and the doses will be compared to each other regarding change from Baseline. In addition to the CardioMEMS HF System measurements, the subjects will be followed for mortality and morbidity, important biomarkers as well as subjective, functional, and structural parameters of importance for PAH, for the duration of the study. Subjects will be enrolled for up to 22 weeks: a Screening Period of up to 2 weeks prior to the start of the Baseline Period, a Baseline Period of up to 6 weeks prior to Randomization, a Treatment Period of 12 weeks, and a Follow-up Period of 2 weeks. The primary objective of the study is to obtain safety and tolerability data.

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	CS1 Administration	CS1 delayed-release capsules will be supplied with 160 mg active pharmaceutical ingredient (sodium valproate) per capsule. Three dose levels that will be evaluated in this study; study drug will be administered as uneven, divided doses twice daily in the morning and in the evening (1/3 of the daily dose is to be administered between 7 and 9 am; 2/3 of the daily dose is to be administered between 7 and 9 pm).

Timeline

• Start date: 2022-05-05
• Primary completion: 2024-08-01
• Completion: 2024-08-01
• First posted: 2022-02-04
• Last updated: 2024-10-04

Locations

1 site across 1 country: Sweden

Regulatory

• FDA-regulated drug study