Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects (RUBATO)

Actelion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Congenital Heart Disease

Treatments: Drug: Placebo; Drug: Macitentan 10 mg

Study type: Interventional

Funder types: Industry

Identifiers

NCT03153137

AC-055H301

2016-003320-23 (EudraCT Number)

Details and patient eligibility

About

The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.

Enrollment: 142 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Written informed consent/assent from the subject and/or a legal representative prior to initiation of any study-mandated procedures
• Fontan-palliated subjects with either intra-atrial lateral tunnel total cavopulmonary connection (LT-TCPC), or extra cardiac tunnel TCPC (EC-TCPC) surgery > 1 year before Screening. Either LT- or EC-TCPC can be primary or secondary to atrio-pulmonary connection
• New York Heart Association (NYHA) functional class (FC) II or III (assessed by the investigator using the Specific Activity Scale
• Women of childbearing potential must have a negative serum pregnancy test use reliable contraception

Exclusion criteria

• Pattern of Fontan circulation severity
• Deterioration of the Fontan-palliated condition.
• Limitations to Cardiopulmonary exercise testing (CPET)
• Peak VO2 < 15 mL/kg/min.
• Any known factor or disease that may interfere with treatment compliance or full participation in the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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142 participants in 2 patient groups, including a placebo group

Macitentan

Experimental group

Description:

Macitentan 10 mg per day; film-coated tablet; oral use

Treatment:

Drug: Macitentan 10 mg

Placebo

Placebo Comparator group

Description:

film-coated tablet; oral use

Treatment:

Drug: Placebo