An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients. (RUBATO OL)

Actelion Pharmaceuticals

Status and phase: Terminated; Phase 3

Conditions: Congenital Heart Disease With Fontan Circulation

Treatments: Drug: macitentan 10 mg

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03775421

2018-002821-45 (EudraCT Number)

AC-055H302

Details and patient eligibility

About

The aim of this open-label (OL) trial is to study the long-term use of macitentan for up to 2 years in Fontan-palliated adult and adolescent patients beyond the 52 weeks of treatment in the parent RUBATO double-blind (DB) study (AC-055H301, NCT03153137). This OL trial studies the long-term effect of macitentan in Fontan-palliated patients as it is not known if the effect of macitentan is sustained beyond 52 weeks (end of the parent RUBATO DB study). In addition, the trial also studies the long-term safety of macitentan as this is also unknown. Furthermore, the opportunity will be given to patients who were on placebo in the parent RUBATO DB study to receive macitentan 10 mg and benefit from a potentially active treatment.

Enrollment: 112 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.

• Subjects who have completed Week 52 of the parent AC-055H301/RUBATO DB study (NCT03153137)

• Women of childbearing potential must:

  1. have a negative serum pregnancy test prior to first intake of OL study drug, and,
  2. agree to perform monthly pregnancy tests up to the end of the safety follow up (S-FU) period, and,
  3. use reliable methods of contraception from enrollment up to at least 30 days after study treatment discontinuation.

Exclusion criteria

• Clinical worsening leading to medical interventions including reoperation of Fontan circulation (Fontan take-down) during the enrollment period
• Systolic blood pressure < 90 mmHg (< 85 mmHg for subjects < 18 years old and < 150 cm of height) at rest
• Criteria related to macitentan use
• Any known factor or disease that may interfere with treatment compliance or full participation in the study

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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112 participants in 1 patient group

Open-label treatment period

Experimental group

Description:

oral administration of 10 mg macitentan once daily

Treatment:

Drug: macitentan 10 mg