Trial Outcomes & Findings for Phase IIa: Safety, PK, & Tolerability of Sodium Nitrite in Patients With Peripheral Arterial Disease-SONIC (NCT NCT01401517)

NCT ID: NCT01401517

Last Updated: 2020-04-21

Results Overview

The primary objective of this clinical study is to evaluate the safety and tolerability of multiple doses of twice daily 40mg and 80mg sodium nitrite compared with placebo over a 10 week treatment period. Subjects will be asked to report any adverse events during the trial period, and blood pressure, methemoglobin levels and other blood chemistries will be assessed during the trial period for changes from baseline.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

55 participants

Primary outcome timeframe

11 weeks

Results posted on

2020-04-21

Participant Flow

Participant milestones

Participant milestones
Measure
Placebo
sodium nitrite: 0, 40 or 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
40 mg Sodium Nitrite
40 mg dose, BID sodium nitrite: 0, 40 or 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
80 mg dose, BID sodium nitrite: 0, 40 or 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Overall Study
STARTED
18
19
18
Overall Study
COMPLETED
15
17
15
Overall Study
NOT COMPLETED
3
2
3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Phase IIa: Safety, PK, & Tolerability of Sodium Nitrite in Patients With Peripheral Arterial Disease-SONIC

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo
n=18 Participants
Placebo twice each day for 11 weeks
40 mg Sodium Nitrite
n=19 Participants
40 mg dose, BID 40 mg sodium nitrite: 40 twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=18 Participants
80 mg dose, BID sodium nitrite: 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Total
n=55 Participants
Total of all reporting groups
Age, Continuous
64.9 years
STANDARD_DEVIATION 8.98 • n=5 Participants
65.3 years
STANDARD_DEVIATION 8.86 • n=7 Participants
67.9 years
STANDARD_DEVIATION 9.99 • n=5 Participants
66.0 years
STANDARD_DEVIATION 9.21 • n=4 Participants
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
Age, Categorical
Between 18 and 65 years
6 Participants
n=5 Participants
11 Participants
n=7 Participants
4 Participants
n=5 Participants
21 Participants
n=4 Participants
Age, Categorical
>=65 years
12 Participants
n=5 Participants
8 Participants
n=7 Participants
14 Participants
n=5 Participants
34 Participants
n=4 Participants
Sex: Female, Male
Female
5 Participants
n=5 Participants
4 Participants
n=7 Participants
5 Participants
n=5 Participants
14 Participants
n=4 Participants
Sex: Female, Male
Male
13 Participants
n=5 Participants
15 Participants
n=7 Participants
13 Participants
n=5 Participants
41 Participants
n=4 Participants
Region of Enrollment
United States
18 participants
n=5 Participants
19 participants
n=7 Participants
18 participants
n=5 Participants
55 participants
n=4 Participants

PRIMARY outcome

Timeframe: 11 weeks

The primary objective of this clinical study is to evaluate the safety and tolerability of multiple doses of twice daily 40mg and 80mg sodium nitrite compared with placebo over a 10 week treatment period. Subjects will be asked to report any adverse events during the trial period, and blood pressure, methemoglobin levels and other blood chemistries will be assessed during the trial period for changes from baseline.

Outcome measures

Outcome measures
Measure
40 mg Sodium Nitrite
n=19 Participants
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=18 Participants
80 mg dose, BID sodium nitrite:80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Placebo
n=18 Participants
0 mg twice each day for 11 weeks.
Reporting of Adverse Events During 11 Week Treatment Period.
at least 1 AE
12 participants
14 participants
9 participants
Reporting of Adverse Events During 11 Week Treatment Period.
SAEs
0 participants
0 participants
2 participants

SECONDARY outcome

Timeframe: 10 weeks

Population: Change in flow mediated dilation from baseline to final visit (10 weeks post-treatment)

Demonstrate the pharmacodynamic effect of sodium nitrite on changes in FMD by imaging before investigational product administration and 10 weeks after administration of investigational product but before dose escalation. FMD was measured at baseline and again after 10 weeks of treatment. The value at ten weeks was subtracted from the baseline value. A negative value represents a worsening of FMD while a positive value represents an improvement in FMD.

Outcome measures

Outcome measures
Measure
40 mg Sodium Nitrite
n=15 Participants
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=16 Participants
80 mg dose, BID sodium nitrite:80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Placebo
n=10 Participants
0 mg twice each day for 11 weeks.
Assessment of Changes in Brachial Artery Flow-Mediated Dilation (FMD)at 10 Weeks From Baseline
Intent to Treat Population
-0.93 percentage from baseline in FMD
Standard Deviation 4.58
0.06 percentage from baseline in FMD
Standard Deviation 3.15
-2.41 percentage from baseline in FMD
Standard Deviation 6.20
Assessment of Changes in Brachial Artery Flow-Mediated Dilation (FMD)at 10 Weeks From Baseline
Per Protocol Population
-0.69 percentage from baseline in FMD
Standard Deviation 4.65
0.50 percentage from baseline in FMD
Standard Deviation 3.28
-2.69 percentage from baseline in FMD
Standard Deviation 6.50

SECONDARY outcome

Timeframe: 10 weeks

Population: The change in distance walked in 6 minutes at Day 70 compared to baseline.

Demonstrate the pharmacodynamic effect of sodium nitrite on changes in functional measures of walking distance. The distance a subject can walk in 6 minutes will be measured prior to the first administration of the investigational product and 10 weeks after taking the investigational product but before the dose escalation. Distance walked in 6 minutes was measured at baseline and again after 10 weeks of treatment. The distance walked at ten weeks was subtracted from the baseline distance walked. A negative value represents a worsening of walking distance while a positive value represents an improvement in walking distance.

Outcome measures

Outcome measures
Measure
40 mg Sodium Nitrite
n=18 Participants
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=17 Participants
80 mg dose, BID sodium nitrite:80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Placebo
n=17 Participants
0 mg twice each day for 11 weeks.
Assessment of Changes in Walking Distance.
47.39 Feet
Standard Deviation 180.46
-28.82 Feet
Standard Deviation 259.59
7.35 Feet
Standard Deviation 124.67

SECONDARY outcome

Timeframe: 10 weeks

Population: Change in scores on components of quality of life questionnaires at day 70 compared to baseline. The minimum (-100; represents worsening disease) and maximum differences (100, representing complete improvement) from baseline to 10 weeks for the total score of each subsection of the questionnaires are provided.

Demonstrate the pharmacodynamic effect of sodium nitrite on changes in measures of claudication symptoms using Quality of Life questionnaires (WIQ \& RAND SF-36). The WIQ is a disease-specific instrument that measures community-based walking. The questionnaire consists of four subscales (pain severity, distance, speed, and stairs). The SF-36 is a set of 36 questions that the subject provides short answers on concerning general health issues that the subject chooses. Questionnaires were administered at baseline and again after 10 weeks of treatment. Scores from the individual sections were summed and divided by the maximal score for the section to obtain a percent score, ranging from 0 (inability to perform task) to 100 (no difficulty). The scores for each section at ten weeks was subtracted from the baseline scores for each section of the questionnaires. A negative represents declining function, a positive value represents improvement over the study period.

Outcome measures

Outcome measures
Measure
40 mg Sodium Nitrite
n=18 Participants
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=17 Participants
80 mg dose, BID sodium nitrite:80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Placebo
n=17 Participants
0 mg twice each day for 11 weeks.
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
WIQ: Walking Distance
2.13 units on a scale
Interval -70.3 to 63.4
5.05 units on a scale
Interval -66.5 to 61.8
-1.07 units on a scale
Interval -14.2 to 45.6
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
WIQ: Walking Speed
6.52 units on a scale
Interval -17.4 to 51.5
7.07 units on a scale
Interval -57.6 to 28.3
0.00 units on a scale
Interval -23.9 to 38.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
WIQ: Stair Climbing
12.5 units on a scale
Interval -25.0 to 58.3
0.0 units on a scale
Interval -37.5 to 45.8
0.0 units on a scale
Interval -41.7 to 45.8
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Physical Functioning
5.0 units on a scale
Interval -25.0 to 35.0
0.0 units on a scale
Interval -35.0 to 30.0
0.0 units on a scale
Interval -20.0 to 15.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Limitations due to Physcial Health
0.0 units on a scale
Interval -75.0 to 100.0
0.0 units on a scale
Interval -100.0 to 50.0
25.0 units on a scale
Interval -75.0 to 50.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Limitations due to Emotional Health
0.00 units on a scale
Interval -100.0 to 100.0
0.0 units on a scale
Interval -100.0 to 66.7
0.0 units on a scale
Interval -100.0 to 100.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Energy/Fatigue
0.0 units on a scale
Interval -25.0 to 65.0
5.0 units on a scale
Interval -30.0 to 30.0
-2.50 units on a scale
Interval -25.0 to 20.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Emotional Well Being
2.0 units on a scale
Interval -24.0 to 48.0
4.0 units on a scale
Interval -12.0 to 32.0
-4.0 units on a scale
Interval -44.0 to 20.0
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Social Functioning
0.0 units on a scale
Interval -25.0 to 62.5
0.0 units on a scale
Interval -37.5 to 50.0
0.0 units on a scale
Interval -25.0 to 12.5
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: Pain
12.5 units on a scale
Interval -12.5 to 55.0
0.0 units on a scale
Interval -37.5 to 22.5
0.0 units on a scale
Interval -55.0 to 22.5
Assessment of Improvement of Quality of Life Using the WIQ (Walking Impairment Questionnaire) and SF-36 Questionnaire.
SF-36: General Health
7.5 units on a scale
Interval -15.0 to 25.0
0.0 units on a scale
Interval -20.0 to 30.0
0.0 units on a scale
Interval -25.0 to 20.0

Adverse Events

Placebo

Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths

40 mg Sodium Nitrite

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

80 mg Sodium Nitrite

Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Placebo
n=18 participants at risk
sodium nitrite: 0 mg twice each day for 11 weeks .
40 mg Sodium Nitrite
n=19 participants at risk
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=18 participants at risk
80 mg dose, BID sodium nitrite: 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
Vascular disorders
Worsening PAD
5.6%
1/18 • Number of events 1
0.00%
0/19
0.00%
0/18
Gastrointestinal disorders
GI Bleed
5.6%
1/18 • Number of events 1
0.00%
0/19
0.00%
0/18

Other adverse events

Other adverse events
Measure
Placebo
n=18 participants at risk
sodium nitrite: 0 mg twice each day for 11 weeks .
40 mg Sodium Nitrite
n=19 participants at risk
40 mg dose, BID sodium nitrite: 40 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
80 mg Sodium Nitrite
n=18 participants at risk
80 mg dose, BID sodium nitrite: 80 mg twice each day for 10 weeks followed by a 1 week escalation of 2 times the dose.
General disorders
Headaches and dizziness
0.00%
0/18
21.1%
4/19 • Number of events 4
44.4%
8/18 • Number of events 8
Gastrointestinal disorders
GI Disorders
22.2%
4/18 • Number of events 4
10.5%
2/19 • Number of events 2
16.7%
3/18 • Number of events 3
Infections and infestations
Infections
5.6%
1/18 • Number of events 1
10.5%
2/19 • Number of events 2
5.6%
1/18 • Number of events 1
Musculoskeletal and connective tissue disorders
Muscoskeletal and Connective Tissues
11.1%
2/18 • Number of events 2
15.8%
3/19 • Number of events 3
11.1%
2/18 • Number of events 2
Investigations
Investigations
16.7%
3/18 • Number of events 3
5.3%
1/19 • Number of events 1
16.7%
3/18 • Number of events 3

Additional Information

Tony Giordano

TheraVasc Inc.

Phone: 318-349-3851

Results disclosure agreements

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

Restriction type: LTE60