CS1 Added to Standard of Care Therapy for Pulmonary Arterial Hypertension

NCT07761572 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CS1-0052026-526523-22-00
• Study Type: interventional
• Target: 126
• Locations: 0 countries
• Sites: N/A

Brief Summary

This Phase 2 study (EPIMODE) will evaluate the efficacy and safety of CS1 compared with placebo when added to standard-of-care therapy in participants with pulmonary arterial hypertension. Participants completing Treatment Period 1 (Week 36) will be re-randomized to continue or switch study treatment for Treatment Period 2 through Week 52.

Conditions

Pulmonary Arterial Hypertension

Keywords

CS1; Pulmonary Arterial Hypertension; PAH; Sodium Valproate; Valproic Acid; Pulmonary Vascular Resistance; 6-Minute Walk Distance

Outcome Measures

Primary Outcomes (1)

• Change from baseline in pulmonary vascular resistance (PVR)

  Pulmonary vascular resistance calculated from right heart catheterization measurements using the Thermodilution or Fick method for determination of cardiac output.

  Baseline to Week 36

Secondary Outcomes (21)

• Change from baseline in 6-minute walk distance (6MWD)

  Baseline to Week 36

• Change from baseline in Registry to Evaluate Early and Long-term Pulmonary Arterial Hypertension Disease Management (REVEAL) Lite 2 score

  Baseline to Week 36

• Change from baseline in REVEAL Risk Score (RRS) 2.0

  Baseline to Week 36

• Change from baseline in tricuspid annular plane systolic excursion (TAPSE)

  Baseline to Week 36

• Change from baseline in right ventricular (RV) strain

  Baseline to Week 36

Study Arms (3)

CS1 320 mg

EXPERIMENTAL

2 capsules of CS1 160 mg and 2 capsules of placebo

Drug: CS1; Drug: Placebo

CS1 640 mg

EXPERIMENTAL

4 capsules of CS1 160 mg

Drug: CS1

Placebo

PLACEBO COMPARATOR

4 capsules of placebo

Drug: Placebo

Interventions

CS1 DRUG

160 mg delayed release capsules taken orally once daily in the evening

CS1 320 mg; CS1 640 mg

Placebo DRUG

Matching placebo capsules taken orally once daily in the evening

CS1 320 mg; Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 to 80 years
• WHO Group 1 pulmonary arterial hypertension
• WHO Functional Class II or III
• REVEAL Lite 2 score ≥6
• Receiving stable standard-of-care PAH therapy for at least 90 days prior to screening
• Hemodynamic confirmation of PAH by right heart catheterization
• minute walk distance ≥150 m and \<550 m

You may not qualify if:

• Pulmonary hypertension WHO Group 2, 3, 4, or 5
• Significant left-sided heart disease
• Recent major cardiovascular or cerebrovascular event
• Prior heart or heart-lung transplantation or active lung transplant listing
• Significant renal or hepatic dysfunction
• Current use of prohibited medications that cannot be discontinued
• Pregnant or breastfeeding

Sponsors & Collaborators

• Cereno Scientific AB: lead

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2026
• First Posted: August 12, 2026
• Study Start: September 15, 2026
• Primary Completion (Estimated): February 2, 2029
• Study Completion (Estimated): April 30, 2029
• Last Updated: August 12, 2026

Record last verified: 2026-08