Study to Evaluate the Effect of Eleclazine on QT, Safety, and Tolerability in Participants With Long QT2 Syndrome

Gilead Sciences

Status and phase: Completed; Phase 1

Conditions: LQT2 Syndrome

Treatments: Drug: Eleclazine; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02365506

GS-US-394-1658

Details and patient eligibility

About

The primary objective of the study is to evaluate the effect of oral eleclazine (formerly GS-6615) on corrected QT (QTc) interval in participants with long QT2 syndrome.

Enrollment: 13 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants with an established diagnosis of LQT2 (by genotype testing)
• Mean (of triplicate) QTc interval ≥ 480 msec for at least four out of seven time points, determined by standard 12-lead electrocardiogram (ECG), at screening

Key Exclusion Criteria:

• Known mutations associated with long QT syndrome type 1 or long QT syndrome type 3
• Known or suspected history of seizures or epilepsy
• History of heart failure defined as New York Heart Association (NYHA) Class IV and/or known left ventricular ejection fraction (EF) ≤ 45%
• Body mass index (BMI) ≥ 36 kg/m^2 at screening
• Severe renal impairment at screening (defined as an estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m^2, using the 4 variable modification of diet in renal disease (MDRD) equation), as determined by the study center
• Abnormal liver function tests at screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x upper limit of normal (ULN), or total bilirubin > 1.5 x ULN
• An aborted cardiac arrest (ACA), implantable cardioverter-defibrillator (ICD) implantation, syncopal episode, or appropriate ICD therapy within 3 months prior to screening
• Any other condition or circumstance that in the opinion of the investigator would preclude compliance with the study protocol.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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13 participants in 3 patient groups, including a placebo group

Eleclazine 24 mg + Eleclazine 48 mg + Placebo

Experimental group

Description:

Participants will receive placebo to match eleclazine on Days 1 and 4, eleclazine 24 mg on Day 2 and eleclazine 48 mg on Day 3.

Treatment:

Drug: Eleclazine

Drug: Placebo

Eleclazine 48 mg + Placebo

Experimental group

Description:

Participants will receive placebo to match eleclazine on Days 1, 2 and 4, and eleclazine 48 mg on Day 3.

Treatment:

Drug: Eleclazine

Drug: Placebo

Placebo

Placebo Comparator group

Description:

Participants will receive placebo to match eleclazine on Days 1 to 4.

Treatment:

Drug: Placebo