Trial Outcomes & Findings for Long-term Efficacy Study of Sodium Channel Blocker in LQT3 Patients (NCT NCT01648205)

NCT ID: NCT01648205

Last Updated: 2022-04-07

Results Overview

Change in QTc at 2 months on ranolazine vs. at 1 month on placebo. This was prespecified outcome.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

25 participants

Primary outcome timeframe

1 month to 2 months

Results posted on

2022-04-07

Participant Flow

Participant milestones

Participant milestones
Measure
Placebo
There were 25 subjects enrolled receiving placebo for 1 month with ECG to be obtained at baseline and at 1 month.
Placebo
STARTED
25
Placebo
COMPLETED
21
Placebo
NOT COMPLETED
4
Ranolazine
STARTED
23
Ranolazine
COMPLETED
18
Ranolazine
NOT COMPLETED
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo
There were 25 subjects enrolled receiving placebo for 1 month with ECG to be obtained at baseline and at 1 month.
Placebo
Pregnancy
1
Placebo
Withdrawal by Subject
1
Placebo
Protocol Violation
2
Ranolazine
Death
1
Ranolazine
Withdrawal by Subject
1
Ranolazine
Protocol Violation
3

Baseline Characteristics

Long-term Efficacy Study of Sodium Channel Blocker in LQT3 Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
All Participants
n=25 Participants
There were 25 subjects that started the study receiving placebo for 1 month.
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
22 Participants
n=99 Participants
Age, Categorical
>=65 years
3 Participants
n=99 Participants
Age, Continuous
49 years
STANDARD_DEVIATION 14 • n=99 Participants
Sex: Female, Male
Female
13 Participants
n=99 Participants
Sex: Female, Male
Male
12 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
25 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=99 Participants
Race (NIH/OMB)
White
25 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Region of Enrollment
United States
25 participants
n=99 Participants
QTc duration
493 miliseconds
STANDARD_DEVIATION 55 • n=99 Participants

PRIMARY outcome

Timeframe: 1 month to 2 months

Change in QTc at 2 months on ranolazine vs. at 1 month on placebo. This was prespecified outcome.

Outcome measures

Outcome measures
Measure
Placebo
n=25 Participants
Placebo was administered for 1 month.
Ranolazine at 2 Months
n=23 Participants
Ranolazine was administered after 1 month
Change in QTc Duration at 2 Months
503 miliseconds
Standard Deviation 46
497 miliseconds
Standard Deviation 47

SECONDARY outcome

Timeframe: 1 month to 6 months

Change in QTc at 6 months on ranolazine vs. at 1 month on placebo. This was prespecified outcome.

Outcome measures

Outcome measures
Measure
Placebo
n=23 Participants
Placebo was administered for 1 month.
Ranolazine at 2 Months
n=21 Participants
Ranolazine was administered after 1 month
Change in QTc at 6 Months
504 miliseconds
Standard Deviation 53
501 miliseconds
Standard Deviation 49

Adverse Events

Ranolazine

Serious events: 2 serious events
Other events: 3 other events
Deaths: 1 deaths

Placebo

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Ranolazine
n=25 participants at risk
During 5 months on ranolazine following adverse events were reported: Ventricular fibrillation successfully treated with shock from implantable cardioverter-defibrillator (n=1) Ventricular fibrillation unsuccessfully treated with shock from implantable cardioverter-defibrillator with subsequent death (n=1) Bundle branch block (n=1) Dizziness/unsteadiness (n=3) Headache (n=1) Constipation (n=1) Hematuria (n=1) Hemorrhoids (n=1)
Placebo
n=25 participants at risk
During 1 month on placebo following adverse events were reported: Pregnancy (n=1) Gastritis (n=1) Generalized weakness (n=1)
Cardiac disorders
Ventricular Fibrillation
8.0%
2/25 • Number of events 2 • Adverse events data were collected for 1 month on placebo and for up to 5 months on ranolazine.
0.00%
0/25 • Adverse events data were collected for 1 month on placebo and for up to 5 months on ranolazine.

Other adverse events

Other adverse events
Measure
Ranolazine
n=25 participants at risk
During 5 months on ranolazine following adverse events were reported: Ventricular fibrillation successfully treated with shock from implantable cardioverter-defibrillator (n=1) Ventricular fibrillation unsuccessfully treated with shock from implantable cardioverter-defibrillator with subsequent death (n=1) Bundle branch block (n=1) Dizziness/unsteadiness (n=3) Headache (n=1) Constipation (n=1) Hematuria (n=1) Hemorrhoids (n=1)
Placebo
n=25 participants at risk
During 1 month on placebo following adverse events were reported: Pregnancy (n=1) Gastritis (n=1) Generalized weakness (n=1)
Nervous system disorders
Dizziness
12.0%
3/25 • Number of events 3 • Adverse events data were collected for 1 month on placebo and for up to 5 months on ranolazine.
0.00%
0/25 • Adverse events data were collected for 1 month on placebo and for up to 5 months on ranolazine.

Additional Information

Wojciech Zareba, MD, PhD

University of Rochester

Phone: 5852755391

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place