Trial Outcomes & Findings for Nitrate's Effect on Activity Tolerance in Heart Failure With Preserved Ejection Fraction (NCT NCT02053493)

NCT ID: NCT02053493

Last Updated: 2016-11-28

Results Overview

To evaluate whether isosorbide mononitrate increases daily activity as assessed by 14-day averaged arbitrary accelerometry units in comparison to placebo. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

110 participants

Primary outcome timeframe

5-6 weeks

Results posted on

2016-11-28

Participant Flow

All patients admitted to the participating HFN centers with signs and symptoms suggestive of HFpEF will be screened including their ability to wear the accelerometer belt. Patients meeting eligibility criteria will be approached regarding participation in this study.

All subjects who fulfill all the inclusion criteria and none of the exclusion criteria will undergo the baseline studies and then be randomized.

Participant milestones

Participant milestones
Measure
Isosorbide Mononitrate Crossover to Placebo
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Overall Study
STARTED
51
59
Overall Study
COMPLETED
50
58
Overall Study
NOT COMPLETED
1
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Isosorbide Mononitrate Crossover to Placebo
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Overall Study
Withdrawal by Subject
1
1

Baseline Characteristics

Nitrate's Effect on Activity Tolerance in Heart Failure With Preserved Ejection Fraction

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Isosorbide Mononitrate Crossover to Placebo
n=51 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=59 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Total
n=110 Participants
Total of all reporting groups
Age, Continuous
69.8 years
STANDARD_DEVIATION 8.7 • n=5 Participants
68.8 years
STANDARD_DEVIATION 9.7 • n=7 Participants
69.3 years
STANDARD_DEVIATION 9.3 • n=5 Participants
Sex: Female, Male
Female
25 Participants
n=5 Participants
38 Participants
n=7 Participants
63 Participants
n=5 Participants
Sex: Female, Male
Male
26 Participants
n=5 Participants
21 Participants
n=7 Participants
47 Participants
n=5 Participants
Region of Enrollment
United States
51 participants
n=5 Participants
59 participants
n=7 Participants
110 participants
n=5 Participants

PRIMARY outcome

Timeframe: 5-6 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate increases daily activity as assessed by 14-day averaged arbitrary accelerometry units in comparison to placebo. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=45 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=56 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Arbitrary Accelerometry Units (AAU) (Phase I)
9370 accelerometry units
Standard Deviation 4601
9538 accelerometry units
Standard Deviation 6286

PRIMARY outcome

Timeframe: 11-12 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate increases daily activity as assessed by 14-day averaged arbitrary accelerometry units in comparison to placebo. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=42 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=49 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Arbitrary Accelerometry Units (AAU) (Phase II)
8824 accelerometry units
Standard Deviation 3877
8900 accelerometry units
Standard Deviation 5457

SECONDARY outcome

Timeframe: Week 7

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate (ISMN) improves functional capacity by 6 minute walk distance in comparison to placebo.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=49 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=57 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Six Minute Walk Distance (Phase I)
307.8 meters
Standard Deviation 125.5
327.1 meters
Standard Deviation 126.0

SECONDARY outcome

Timeframe: Week 13

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate (ISMN) improves functional capacity by 6 minute walk distance in comparison to placebo.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=47 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=57 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Six Minute Walk Distance (Phase II)
321.3 meters
Standard Deviation 132.4
329.7 meters
Standard Deviation 132.1

SECONDARY outcome

Timeframe: Week 13

Population: Participants with usable data included in the results

Self reported participant preference for study period 1 vs. study period 2.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=48 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=57 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Patient Preference for Isosorbide Mononitrate Treatment at the End of Study.
Phase1
14 participants
14 participants
Patient Preference for Isosorbide Mononitrate Treatment at the End of Study.
Phase2
18 participants
24 participants
Patient Preference for Isosorbide Mononitrate Treatment at the End of Study.
No Preference
16 participants
19 participants

SECONDARY outcome

Timeframe: Week 7

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves quality of life in comparison to placebo. The Borg Scale consists of scale range of 0 to 10 (where 0 indicates no breathlessness at all and 10 indicates maximum breathlessness). Lower values are considered to be better than higher values.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=44 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=52 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Borg Score During 6 Minute Walk Test (Phase I)
4.1 units on a scale
Standard Deviation 2.6
4.0 units on a scale
Standard Deviation 2.2

SECONDARY outcome

Timeframe: Week 13

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves quality of life in comparison to placebo. The Borg Scale consists of scale range of 0 to 10 (where 0 indicates no breathlessness at all and 10 indicates maximum breathlessness). Lower values are considered to be better than higher values.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=40 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=49 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Borg Score During 6 Minute Walk Test (Phase II)
3.8 units on a scale
Standard Deviation 2.3
3.8 units on a scale
Standard Deviation 2.0

SECONDARY outcome

Timeframe: Week 7

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves quality of life in comparison to placebo. The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a disease-specific patient-reported outcomes measure for patients with heart failure. It consists of 23 items, is comprised of 7 clinically relevant scales (Symptom Frequency, Symptom Burden, Symptom Stability, Physical Limitation, Social Limitation, Quality of Life, and Self-Efficacy), and yields 3 summary scores (Clinical Summary, Total Symptom, and Overall Summary Scores). Scale and summary scores range between 0 and 100, with higher scores indicating better health status (eg, better functioning, fewer symptoms, better quality of life).

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=50 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=58 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Kansas City Cardiomyopathy Questionnaire Overall Summary Score (Phase I)
57.3 units on a scale
Standard Deviation 23.4
64.2 units on a scale
Standard Deviation 24.2

SECONDARY outcome

Timeframe: Week 13

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves quality of life in comparison to placebo. • The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a disease-specific patient-reported outcomes measure for patients with heart failure. It consists of 23 items, is comprised of 7 clinically relevant scales (Symptom Frequency, Symptom Burden, Symptom Stability, Physical Limitation, Social Limitation, Quality of Life, and Self-Efficacy), and yields 3 summary scores (Clinical Summary, Total Symptom, and Overall Summary Scores). Scale and summary scores range between 0 and 100, with higher scores indicating better health status (eg, better functioning, fewer symptoms, better quality of life).Higher values of the overall KCCQ score are considered to be better than lower values.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=49 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=58 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Kansas City Cardiomyopathy Questionnaire Overall Summary Score (Phase II)
59.1 units on a scale
Standard Deviation 23.6
61.6 units on a scale
Standard Deviation 23.5

SECONDARY outcome

Timeframe: Week 7

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves natriuretic peptide levels in comparison to placebo

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=49 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=57 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
N-terminal Pro-B-type Natriuretic Peptide Level (Phase I)
513.0 pg/mL
Standard Deviation 803.2
542.4 pg/mL
Standard Deviation 710.9

SECONDARY outcome

Timeframe: Week 13

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate improves natriuretic peptide levels in comparison to placebo

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=47 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=54 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
N-terminal Pro-B-type Natriuretic Peptide Level (Phase II)
466.1 pg/mL
Standard Deviation 677.4
573.3 pg/mL
Standard Deviation 756.1

SECONDARY outcome

Timeframe: 5-6 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Hours active per day during maximal dose of study drug

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=45 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=56 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Hours Active Per Day (Phase I)
9.4 Hours/day
Standard Deviation 2.4
9.1 Hours/day
Standard Deviation 2.4

SECONDARY outcome

Timeframe: 11-12 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Hours active per day during maximal dose of study drug

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=42 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=49 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Hours Active Per Day (Phase II)
9.4 Hours/day
Standard Deviation 2.4
8.8 Hours/day
Standard Deviation 2.5

SECONDARY outcome

Timeframe: 3-6 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Slope of daily averaged arbitrary accelerometry units during study drug administration. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=47 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=58 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Slope of Daily Average (Phase I)
-3.4 accelerometry units/day
Standard Deviation 23.1
-1.3 accelerometry units/day
Standard Deviation 26.9

SECONDARY outcome

Timeframe: 9-12 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Slope of daily averaged arbitrary accelerometry units during study drug administration. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement.

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=42 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=53 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Slope of Daily Average (Phase II)
2.6 accelerometry units/day
Standard Deviation 20.1
-3.9 accelerometry units/day
Standard Deviation 16.4

SECONDARY outcome

Timeframe: 3-6 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Area under the curve (AUC) of arbitrary accelerometry units during study drug administration. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement. Area under the curve is defined as ((7\*average acceleromtery units/day during 30 mg) + (7\*average acceleromtery units/day during 60 mg) + (14\*average acceleromtery units/day during 120 mg))/28

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=39 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=54 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Area Under the Curve (Phase I)
9621.4 accelerometry units
Standard Deviation 4367.8
9714.0 accelerometry units
Standard Deviation 6273.6

SECONDARY outcome

Timeframe: 9-12 weeks

Population: Participants with usable data included in the results

To evaluate whether isosorbide mononitrate in comparison to placebo improves daily activity as measured by Area under the curve (AUC) of arbitrary accelerometry units during study drug administration. An arbitrary accelerometer unit is calculated within the accelerometer device that is worn by the patient and represents level of activity based on patient movement. Higher values indicate more movement. 0 indicates no movement. Area under the curve is defined as ((7\*average acceleromtery units/day during 30 mg) + (7\*average acceleromtery units/day during 60 mg) + (14\*average acceleromtery units/day during 120 mg))/28

Outcome measures

Outcome measures
Measure
Isosorbide Mononitrate Crossover to Placebo
n=38 Participants
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=44 Participants
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Improvement in Daily Activity - Area Under the Curve (Phase II)
9146.5 accelerometry units
Standard Deviation 3695.3
9325.9 accelerometry units
Standard Deviation 5668.2

Adverse Events

Isosorbide Mononitrate Crossover to Placebo

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

Placebo Crossover to Isosorbide Mononitrate

Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Isosorbide Mononitrate Crossover to Placebo
n=51 participants at risk
Isosorbide Mononitrate with dose up-titration (30 to 120 mg/day over 4 weeks) Isosorbide Mononitrate: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg ISMN Week 4: 60 mg ISMN Weeks 5 and 6: 120 mg ISMN Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg ISMN Week 10: 60 mg ISMN Weeks 11 and 12: 120 mg ISMN
Placebo Crossover to Isosorbide Mononitrate
n=59 participants at risk
Isosorbide Mononitrate placebo with dose up-titration (30 to 120 mg/day over 4 weeks) Placebo: Dispense phase 1 study drug: Weeks 1 and 2: No study drug (baseline) Week 3: 30 mg Placebo Week 4: 60 mg Placebo Weeks 5 and 6: 120 mg Placebo Dispense phase-2 study drug: Weeks 7 and 8: No study drug (washout) Week 9: 30 mg Placebo Week 10: 60 mg Placebo Weeks 11 and 12: 120 mg Placebo
Infections and infestations
Cellulitis
2.0%
1/51 • Number of events 1 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
0.00%
0/59 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
Respiratory, thoracic and mediastinal disorders
chronic obstructive pulmonary disease
2.0%
1/51 • Number of events 1 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
0.00%
0/59 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
Infections and infestations
Herpes Zoster
0.00%
0/51 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
1.7%
1/59 • Number of events 1 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
Gastrointestinal disorders
Faecaloma
2.0%
1/51 • Number of events 1 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
0.00%
0/59 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
Renal and urinary disorders
Urinary retention
2.0%
1/51 • Number of events 1 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.
0.00%
0/59 • Informed Consent through Week 15 phone visit
Per protocol, non-serious AEs were not collected; all SAEs were collected except for anticipated, disease-related events in patients with HF with preserved EF. 3 time points summarized: Informed Consent to start of Phase 1 study drug, Phase 1 study drug start to start of Phase 2 study drug, Phase 2 study drug start to Week 15 phone call.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Adrian Hernandez

Duke Clinical Research Insitute

Phone: 919-668-7515

Results disclosure agreements

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

Restriction type: LTE60