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

Cereno Scientific AB1 site in 1 country25 target enrollmentMay 5, 2022

ConditionsPulmonary Arterial Hypertension

InterventionsCS1 Administration

DrugsCS1 Administration

Overview

Phase: Phase 2
Intervention: CS1 Administration
Conditions: Pulmonary Arterial Hypertension
Sponsor: Cereno Scientific AB
Enrollment: 25
Locations: 1
Primary Endpoint: Patient reported adverse events
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

May 5, 2022

End Date

August 1, 2024

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Cereno Scientific AB

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Subjects will be eligible for inclusion in the study if they meet all of the following criteria:
•Subject must be willing and able to sign a written informed consent prior to any study-related procedures and able to understand and follow instructions; return to the study unit for specified study visits; and able to participate in the study for the entire period.
•Subject is male or female, aged 18 to 80 years.
•Subject must have a body mass index (BMI) 18 to 40 kg/m2 at Screening. If BMI is \>35 kg/m2, subject chest circumference should be \<65 inches (165 cm).
•Subject with PAH belonging to 1 of the following subgroups of NICE Clinical Classification of PAH category:
•Idiopathic PAH.
•Heritable PAH.
•Drug or toxin-induced (anorexigen or methamphetamine use).
•PAH associated with connective tissue disease.
•Subject with PAH who are symptomatic and have reduced exercise capacity due primarily to their PAH diagnosis, having been assessed by qualified individual, ie, physician, physician assistant, or nurse practitioner, to be in NYHA/WHO functional class II or III and having an RRS 2.0 of 6 to

Exclusion Criteria

•Subjects will be excluded from the study if they meet any of the following criteria:
•Subject has pulmonary hypertension category 2 to
•Subject has adult congenital heart disease (ACHD).
•Subject has concomitant medical or psychiatric disorder, condition, history, or any other condition that in the opinion of the Investigator would either put the subject at risk or impair the subject's ability to participate in or complete the requirements of the study or confound the objectives of the study.
•Subject has a concomitant medical disorder that is expected to limit the subject's life-expectancy to ≤1 year.
•Subject has RRS 2.0 score of ≤5 or ≥
•Subject has heart fai
•lure with preserved ejection fraction defined as those with \>50% ejection fraction (with signs and symptoms of heart failure) or left atrial volume (LAV) \>34 mL/ m
•Subject is not able to have CardioMEMS PA Sensor implanted due to:
•An active, ongoing infection defined as being febrile, an elevated white blood cell count, on intravenous antibiotics, and/or positive cultures (blood, sputum, or urine).