Trial Outcomes & Findings for Study to Assess the Benefit of Midodrine in the Treatment of Patients With Neurogenic Orthostatic Hypotension (NCT NCT00555880)

NCT ID: NCT00555880

Last Updated: 2021-06-14

Results Overview

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

24 participants

Primary outcome timeframe

1 hour post-dose

Results posted on

2021-06-14

Participant Flow

Participant milestones

Participant milestones
Measure
Midodrine/Placebo
Participants received a single oral dose of Midodrine hydrochloride (HCl) followed by matching Placebo the next day
Placebo/Midodrine
Participants received Placebo followed by a single oral dose of Midodrine HCl the next day
Treatment Period 1 (1 Day)
STARTED
13
11
Treatment Period 1 (1 Day)
COMPLETED
13
11
Treatment Period 1 (1 Day)
NOT COMPLETED
0
0
Washout (1 Day)
STARTED
13
11
Washout (1 Day)
COMPLETED
13
11
Washout (1 Day)
NOT COMPLETED
0
0
Treatment Period 2 (1 Day)
STARTED
13
11
Treatment Period 2 (1 Day)
COMPLETED
13
11
Treatment Period 2 (1 Day)
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Assess the Benefit of Midodrine in the Treatment of Patients With Neurogenic Orthostatic Hypotension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Midodrine/Placebo
n=13 Participants
Participants received a single oral dose of Midodrine HCl followed by matching Placebo the next day
Placebo/Midodrine
n=11 Participants
Participants received matching Placebo followed by a single oral dose of Midodrine HCl the next day
Total
n=24 Participants
Total of all reporting groups
Age, Continuous
59.4 years
STANDARD_DEVIATION 17.84 • n=5 Participants
60.8 years
STANDARD_DEVIATION 12.91 • n=7 Participants
60.0 years
STANDARD_DEVIATION 15.46 • n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
5 Participants
n=7 Participants
12 Participants
n=5 Participants
Sex: Female, Male
Male
6 Participants
n=5 Participants
6 Participants
n=7 Participants
12 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
13 Participants
n=5 Participants
11 Participants
n=7 Participants
24 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
2 Participants
n=5 Participants
1 Participants
n=7 Participants
3 Participants
n=5 Participants
Race (NIH/OMB)
White
11 Participants
n=5 Participants
10 Participants
n=7 Participants
21 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 1 hour post-dose

Population: The Full Analysis Set (FAS), defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Onset of Near-syncopal Symptoms During Tilt Table Testing
551.3 seconds
Standard Deviation 115.63
461.0 seconds
Standard Deviation 190.74

PRIMARY outcome

Timeframe: 1 hour post-dose

Population: The FAS, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near- syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out. In this outcome measure, the data analyzed are the same as for Outcome Measure 1 but the summary data are presented as least squares mean (standard error).

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Onset of Near-syncopal Symptoms During Tilt Table Testing Analysis #2
552.4 seconds
Standard Error 32.93
460.0 seconds
Standard Error 32.93

PRIMARY outcome

Timeframe: 1 hour post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out. The Koch procedure is a 3-step process to analyze results while utilizing the available information on magnitude of differences.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Onset of Near-syncopal Symptoms During Tilt Table Testing-Re-analysis With The Koch Procedure
551.3 seconds
Standard Deviation 115.63
461.0 seconds
Standard Deviation 190.74

SECONDARY outcome

Timeframe: 1 hour post-dose

Population: The Per-protocol set, defined as participants in the Full Analysis Set who completed the study and were protocol compliant.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=21 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=21 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Onset of Near-syncopal Symptoms in The Per-protocol Population
544.3 seconds
Standard Deviation 122.34
447.9 seconds
Standard Deviation 199.31

SECONDARY outcome

Timeframe: 1 hour post-dose

Population: The Per-protocol set, defined as participants in the Full Analysis Set who completed the study and were protocol compliant.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near- syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out. In this outcome measure, the data analyzed are the same as for Outcome Measure 4 but the summary data are presented as least squares mean (standard error).

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=21 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=21 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Onset of Near-syncopal Symptoms in The Per-protocol Population Analysis #2
545.1 seconds
Standard Error 36.90
447.2 seconds
Standard Error 36.90

SECONDARY outcome

Timeframe: 1 hour post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The head-up tilt table test in this study was conducted 1 hour after administration of the study medication. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=13 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=11 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Time to Near-syncopal Symptoms at Treatment Visit 1
539.5 seconds
Standard Error 45.91
448.4 seconds
Standard Error 49.91

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

Duration of effect was defined as the difference in time to onset of near-syncopal symptoms between the first and second tilt table test, conducted at 1 hour and 3 hours post-dose, respectively, at Treatment Visit 2 (time to onset at 3 hours minus time at 1 hour). The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. The endpoint was a confirmed report of a near-syncopal symptom(s) (of sufficient severity that caused the patient to ask that the tilt table be returned to the horizontal position). Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=11 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=13 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Duration of The Effect of Treatment at 3 Hours Post-dose
45.1 seconds
Standard Deviation 94.03
48.0 seconds
Standard Deviation 114.96

SECONDARY outcome

Timeframe: Approximately 1 hour post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The OHSA measures the severity of six symptoms/symptom complexes associated with orthostatic hypotension. Subjects rated symptoms experienced during the tilt table test on an eleven-point scale from "none" to "worst possible". The OHSA total score is the sum of six subscales, ranging from 0 (no symptoms) to 60 (worst possible symptoms). The OHSA was completed after the tilt table test was over, but was answered with reference to symptoms experienced during testing. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Total Score of the Orthostatic Hypotension Symptom Assessment (OHSA)
13.7 scores on a scale
Standard Error 2.95
19.4 scores on a scale
Standard Error 2.95

SECONDARY outcome

Timeframe: Approximately 1 hour post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The OHSA measures the severity of six symptoms/symptom complexes associated with OH: dizziness, lightheadedness, and feeling faint; problems with vision; weakness; fatigue; trouble concentrating; and head/neck discomfort. Subjects rated symptoms experienced during the tilt table test on an eleven-point scale from "none" to "worst possible". Scores for each subscale range from 0 (no symptoms) to 10 (worst possible symptoms). The OHSA was completed after the tilt table test was over, but was answered with reference to symptoms experienced during testing. The tilt table test is a 10-minute assessment performed using a tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured with straps to prevent injury. After an equilibration period with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, and feeling faint.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Scores for 6 Items of The OHSA
Dizziness, Lightheadedness, and Feeling Faint
3.4 scores on a scale
Standard Deviation 3.44
4.7 scores on a scale
Standard Deviation 3.57
Scores for 6 Items of The OHSA
Problems with Vision
1.5 scores on a scale
Standard Deviation 2.32
2.7 scores on a scale
Standard Deviation 3.43
Scores for 6 Items of The OHSA
Weakness
3.0 scores on a scale
Standard Deviation 3.13
4.1 scores on a scale
Standard Deviation 3.17
Scores for 6 Items of The OHSA
Fatigue
2.7 scores on a scale
Standard Deviation 3.22
3.5 scores on a scale
Standard Deviation 3.48
Scores for 6 Items of The OHSA
Trouble Concentrating
1.7 scores on a scale
Standard Deviation 2.04
2.6 scores on a scale
Standard Deviation 3.13
Scores for 6 Items of The OHSA
Head/Neck Discomfort
1.9 scores on a scale
Standard Deviation 2.52
2.1 scores on a scale
Standard Deviation 3.21

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The CGI-I instrument assesses the overall impression of the subject's orthostatic hypotension during the tilt table test by using a 7-point scale, with 1 being Very much improved; 2, Much improved; 3, Slightly improved; 4, No change; 5, Slightly worse; 6, Much worse; and 7, Very much worse. The clinician completed the CGI-I after each of the tilt table tests. A patient was assessed as "Improved" if the score was 1, 2, or 3. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Number of Participants With Improvement of Clinician Clinician's Global Impression- Improvement (CGI-I) Scores After Tilt Table Test
Treatment Visit 1- 1 hour post-dose, n=13, 11
4 participants
4 participants
Number of Participants With Improvement of Clinician Clinician's Global Impression- Improvement (CGI-I) Scores After Tilt Table Test
Treatment Visit 2- 1 hour post-dose, n=11, 13
6 participants
4 participants
Number of Participants With Improvement of Clinician Clinician's Global Impression- Improvement (CGI-I) Scores After Tilt Table Test
Treatment Visit 2- 3 hour post-dose, n= 11, 12
3 participants
5 participants

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

The CGI-I instrument assesses the overall impression of the subject's orthostatic hypotension during the tilt table test by using a 7-point scale, with 1 being Very much improved; 2, Much improved; 3, Slightly improved; 4, No change; 5, Slightly worse; 6, Much worse; and 7, Very much worse. The patient completed the CGI-I after each of the tilt table tests. A patient was assessed as "Improved" if the score was 1, 2, or 3. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Number of Participants With Improvement of Patient CGI-I Scores After Tilt Table Test
Treatment Visit 1-1 hour post-dose, n= 13, 11
3 participants
2 participants
Number of Participants With Improvement of Patient CGI-I Scores After Tilt Table Test
Treatment Visit 2-1 hour post-dose, n= 11, 13
5 participants
3 participants
Number of Participants With Improvement of Patient CGI-I Scores After Tilt Table Test
Treatment Visit 2-3 hour post-dose, n= 11, 12
3 participants
3 participants

SECONDARY outcome

Timeframe: 1 hour post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

Blood pressure was recorded just before tilt table testing and immediately after. Timed readings were stopped once a subject experienced near-syncopal symptoms, except for subjects for whom the table was returned to horizontal before 1 minute; for these subjects, a reading was made at 1 minute and was included in analyses. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Final Blood Pressure During Tilt Table Testing
Systolic Pressure
106.5 mmHg
Standard Deviation 37.27
90.2 mmHg
Standard Deviation 24.17
Final Blood Pressure During Tilt Table Testing
Diastolic Pressure
70.6 mmHg
Standard Deviation 16.44
60.6 mmHg
Standard Deviation 15.04

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

Blood pressure was recorded just before tilt table testing, at each minute during tilt table testing, and immediately after. Timed readings were stopped once a subject experienced near-syncopal symptoms, except for subjects for whom the table was returned to horizontal before 1 minute; for these subjects, a reading was made at 1 minute and was included in analyses. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Systolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 1 hour post-dose, n= 11, 13
142.2 mmHg
Standard Deviation 44.20
97.3 mmHg
Standard Deviation 24.55
Systolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 3 hours post-dose, n= 11, 12
110.1 mmHg
Standard Deviation 19.06
94.9 mmHg
Standard Deviation 31.36
Systolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 1 hour post-dose, n= 10, 8
121.7 mmHg
Standard Deviation 37.57
102.9 mmHg
Standard Deviation 21.03
Systolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 3 hours post-dose, n= 7, 6
108.3 mmHg
Standard Deviation 30.48
107.2 mmHg
Standard Deviation 19.32

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

Blood pressure was recorded just before tilt table testing, at each minute during tilt table testing, and immediately after. Timed readings were stopped once a subject experienced near-syncopal symptoms, except for subjects for whom the table was returned to horizontal before 1 minute; for these subjects, a reading was made at 1 minute and was included in analyses. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Diastolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 1 hour post-dose, n= 11, 12
81.5 mmHg
Standard Deviation 19.09
60.8 mmHg
Standard Deviation 13.99
Diastolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 1 hour post-dose, n= 10, 8
79.6 mmHg
Standard Deviation 16.18
64.9 mmHg
Standard Deviation 11.22
Diastolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 3 hours post-dose, n= 11, 12
66.1 mmHg
Standard Deviation 9.66
60.3 mmHg
Standard Deviation 18.53
Diastolic Blood Pressure at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 3 hours post-dose, n= 7, 6
69.3 mmHg
Standard Deviation 13.55
60.7 mmHg
Standard Deviation 14.69

SECONDARY outcome

Timeframe: 1 and 3 hours post-dose

Population: The Full Analysis Set, defined as subjects who were randomized, received at least one dose of study drug, and had at least one measurement of time to syncope during tilt table testing.

Heart rate was recorded just before tilt table testing, at each minute during tilt table testing, and immediately after. Timed readings were stopped once a subject experienced near-syncopal symptoms, except for subjects for whom the table was returned to horizontal before 1 minute; for these subjects, a reading was made at 1 minute and was included in analyses. The tilt table test is a 10-minute assessment performed using a manual or automated tilt table in a specialized laboratory. Subjects were moved onto the horizontal table and secured to the table with straps to prevent injury. After an equilibration period of at least 10 minutes with the subject at rest, the test began and the head of the tilt table was elevated to a 70-degree angle over a period of up to 30 seconds. Symptoms of near syncope were defined as dizziness, lightheadedness, feeling faint, or feeling like the subject might black out.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
n=24 Participants
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Heart Rate at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 1 hour post-dose, n= 11, 13
77.6 beats per minute
Standard Deviation 13.93
80.5 beats per minute
Standard Deviation 14.07
Heart Rate at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 1 hour post-dose, n= 10, 8
77.7 beats per minute
Standard Deviation 16.08
79.5 beats per minute
Standard Deviation 16.48
Heart Rate at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 1 minute 3 hours post-dose, n= 11, 12
79.7 beats per minute
Standard Deviation 11.30
80.9 beats per minute
Standard Deviation 13.19
Heart Rate at 1 Minute and 10 Minutes Into The Tilt Table Test Conducted 1 and 3 Hours Post-dose at Treatment Visit 2
At 10 minutes 3 hours post-dose, n= 8, 6
80.9 beats per minute
Standard Deviation 11.66
81.3 beats per minute
Standard Deviation 20.16

SECONDARY outcome

Timeframe: Baseline to discharge

Population: The Safety population, defined as all subjects who received at least one dose of investigational product.

For hematology, blood samples (5.0mL) were taken at screening, study admission (if greater than 14 days since screening) and discharge/early termination. The following parameters were assessed: hemoglobin, hematocrit, red blood cells (RBC), mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), mean corpuscular hemoglobin concentration (MCHC), white blood cell count - total and differential (WBC), and platelet count. A shift in reference to normal was either lower or higher at discharge.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
Hemoglobin
1 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
Hematocrit
0 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
RBC
0 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
MCV
0 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
MCH
0 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
MCHC
2 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
WBC
1 participants
Number of Participants With Shifts in Reference to Normal Range For Hematology Analytes at Discharge
Platelet count
0 participants

SECONDARY outcome

Timeframe: Baseline to discharge

Population: The Safety population, defined as all subjects who received at least one dose of investigational product.

For biochemistry, blood samples (10.0mL) were taken at screening, admission (if greater than 14 days since screening) and discharge/early termination. The following parameters were assessed: sodium, potassium, calcium, blood urea nitrogen (BUN)/Urea, creatinine, albumin, total protein and albumin/globulin (A/G) ratio, globulin, aspartate transaminase (AST), alanine transaminase (ALT), alkaline phosphatase (ALP), gamma glutamyl transferase (GGT), total bilirubin, glucose, chloride, and creatine kinase. A shift in reference to normal was either lower or higher at discharge.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
AST
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Sodium
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Potassium
2 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Calcium
1 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
BUN/Urea
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Creatinine
1 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Albumin
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Total protein and A/G ratio
2 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Globulin
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
ALT
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
ALP
1 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
GGT
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Total bilirubin
1 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Glucose
6 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Chloride
0 participants
Number of Participants With Shifts in Reference to Normal Range For Clinical Chemistry at Discharge
Creatine kinase
1 participants

SECONDARY outcome

Timeframe: Baseline to discharge

Population: The Safety population, defined as all subjects who received at least one dose of investigational product.

For urinalysis, samples were taken at screening, study admission (if greater than 14 days since screening) and discharge/early termination): glucose, blood, protein, pH, specific gravity, leukocyte esterase, and microscopic examination. A shift in reference to normal was higher at discharge.

Outcome measures

Outcome measures
Measure
Midodrine HCl
n=24 Participants
Midodrine HCl: one dose, 10-30mg, given orally
Placebo
Placebo: A daily dose of Placebo tablets matching Midodrine HCl in appearance and number
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Glucose
1 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Blood
2 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Protein
0 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
pH
0 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Specific gravity
0 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Leukocyte esterase
2 participants
Number of Participants With Shifts in Reference to Normal Range For Urinalysis at Discharge
Microscopic examination
0 participants

Adverse Events

Midodrine/Placebo

Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths

Placebo/Midodrine

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

Serious adverse events

Serious adverse events
Measure
Midodrine/Placebo
n=13 participants at risk
Participants received a single oral dose of Midodrine HCl (10-30mg) followed by placebo the next day.
Placebo/Midodrine
n=11 participants at risk
Participants received Placebo followed by a single oral dose of Midodrine HCl (10-30mg) the next day.
Nervous system disorders
Multiple sclerosis aggravated
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.

Other adverse events

Other adverse events
Measure
Midodrine/Placebo
n=13 participants at risk
Participants received a single oral dose of Midodrine HCl (10-30mg) followed by placebo the next day.
Placebo/Midodrine
n=11 participants at risk
Participants received Placebo followed by a single oral dose of Midodrine HCl (10-30mg) the next day.
Gastrointestinal disorders
Nausea
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Cardiac disorders
Atrioventricular block first degree
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Gastrointestinal disorders
Vomiting NOS
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Metabolism and nutrition disorders
Hypokalaemia
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Nervous system disorders
Dizziness
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Nervous system disorders
Headache NOS
7.7%
1/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
0.00%
0/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.
Vascular disorders
Hypertension aggravated
0.00%
0/13
Adverse events are reported by randomized arm and not by actual treatment intervention received.
18.2%
2/11
Adverse events are reported by randomized arm and not by actual treatment intervention received.

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
  • Publication restrictions are in place

Restriction type: OTHER