Trial Outcomes & Findings for Unacylated Ghrelin to Improve Functioning in PAD (NCT NCT04377126)
NCT ID: NCT04377126
Last Updated: 2026-06-01
Results Overview
Change in six minute walk distance at 4-month follow-up will be compared between those randomized to subcutaneously administered unacylated ghrelin vs. those randomized to placebo.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
Baseline to 4 months
Results posted on
2026-06-01
Participant Flow
Participant milestones
| Measure |
Unacylated Ghrelin
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
Placebo
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
13
|
|
Overall Study
COMPLETED
|
12
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Unacylated Ghrelin to Improve Functioning in PAD
Baseline characteristics by cohort
| Measure |
Unacylated Ghrelin
n=12 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
Placebo
n=13 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
68.26 years
STANDARD_DEVIATION 10.05 • n=24 Participants
|
72.70 years
STANDARD_DEVIATION 10.95 • n=24 Participants
|
70.57 years
STANDARD_DEVIATION 10.55 • n=48 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=24 Participants
|
6 Participants
n=24 Participants
|
7 Participants
n=48 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=24 Participants
|
7 Participants
n=24 Participants
|
18 Participants
n=48 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=24 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=48 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=24 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=48 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=24 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=48 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=24 Participants
|
6 Participants
n=24 Participants
|
10 Participants
n=48 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=24 Participants
|
6 Participants
n=24 Participants
|
14 Participants
n=48 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=24 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=48 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=24 Participants
|
1 Participants
n=24 Participants
|
1 Participants
n=48 Participants
|
|
Ankle brachial index
|
0.78 Ratio
STANDARD_DEVIATION 0.54 • n=24 Participants
|
0.72 Ratio
STANDARD_DEVIATION 0.24 • n=24 Participants
|
0.75 Ratio
STANDARD_DEVIATION 0.40 • n=48 Participants
|
|
Body mass index
|
28.43 kg/m^2
STANDARD_DEVIATION 5.98 • n=24 Participants
|
29.69 kg/m^2
STANDARD_DEVIATION 7.23 • n=24 Participants
|
29.09 kg/m^2
STANDARD_DEVIATION 6.55 • n=48 Participants
|
|
Six-minute walking distance
|
338.89 meters
STANDARD_DEVIATION 125.86 • n=24 Participants
|
344.96 meters
STANDARD_DEVIATION 158.19 • n=24 Participants
|
342.05 meters
STANDARD_DEVIATION 140.65 • n=48 Participants
|
PRIMARY outcome
Timeframe: Baseline to 4 monthsPopulation: One participant in the Unacylated ghrelin group was missing FV4 six-minute walk test and was excluded from this analysis.
Change in six minute walk distance at 4-month follow-up will be compared between those randomized to subcutaneously administered unacylated ghrelin vs. those randomized to placebo.
Outcome measures
| Measure |
Placebo
n=13 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Unacylated Ghrelin
n=11 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
|---|---|---|
|
4-month Change in Six-minute Walk Distance
|
23.06 meters
Standard Error 13.05
|
-17.13 meters
Standard Error 17.24
|
SECONDARY outcome
Timeframe: Baseline to 4 monthsPopulation: Seven participants in the Unacylated ghrelin group and four participants in the Placebo group were missing treadmill testing due to COVID-19.
Change in maximal treadmill walking time at 4 month follow-up will be compared between those randomized to subcutaneously administered unacylated ghrelin vs. those randomized to placebo.
Outcome measures
| Measure |
Placebo
n=9 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Unacylated Ghrelin
n=5 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
|---|---|---|
|
4-month Change in Maximal Treadmill Walking Time
|
-0.67 minutes
Standard Error 0.67
|
-2.70 minutes
Standard Error 0.98
|
SECONDARY outcome
Timeframe: Baseline to 4 monthsPopulation: Eleven participants in the Unacylated ghrelin group and eight participants in the Placebo group were missing MRI perfusion test due to COVID-19.
Perfusion was measured using cuff occlusion hyperemia while the participant was supine. The cuff occlusion hyperemia MRI was performed by inflating a thigh cuff to 250 mm Hg in the leg with lowest ABI for five minutes while the participant was supine. After five minutes, the cuff was rapidly deflated. Seven control-tagged image pairs were acquired over 60 seconds using PASL pulse sequence with single-shot echo-planar imaging readouts. Images were interpreted using a Siemens Healthcare workstation.
Outcome measures
| Measure |
Placebo
n=5 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Unacylated Ghrelin
n=1 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
|---|---|---|
|
4-month Change in Calf Muscle Perfusion
|
-2.81 ml/min/100g
Standard Error 3.57
|
-5.86 ml/min/100g
Standard Error 8.79
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline to 4 monthsPopulation: One participant in the Unacylated ghrelin group was missing FV4 WIQ and was excluded from this analysis.
Change in Walking Impairment Questionnaire (WIQ) distance score at 4 month follow-up will be compared between those randomized to subcutaneously administered unacylated ghrelin vs. those randomized to placebo. The WIQ distance score ranges from 0-100 with a higher score indicating a better outcome.
Outcome measures
| Measure |
Placebo
n=13 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Unacylated Ghrelin
n=11 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
|---|---|---|
|
4-month Change in Walking Impairment Questionnaire (WIQ) Distance Score
|
5.13 score on a scale
Standard Error 5.54
|
-7.06 score on a scale
Standard Error 7.28
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline to 4 monthsPopulation: One participant in the Unacylated ghrelin group was missing FV4 PROMIS mobility questionnaire and was excluded from this analysis.
The PROMIS mobility score use a T-score metric, with a mean of 50 and a standard deviation of 10 compared with the general population. Higher scores are better. The minimal clinically important differences is 2.0 points for the PROMIS Mobility score. The PROMIS measures are based on 'item response theory' and therefore have no defined minimum or maximum value. The lowest and highest scores observed for PROMIS have been 20 and 80, respectively.
Outcome measures
| Measure |
Placebo
n=13 Participants
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
Unacylated Ghrelin
n=11 Participants
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
|---|---|---|
|
4-month Change in the Patient-Reported Outcomes Measurement Information System (PROMIS) Mobility Questionnaire
|
-1.67 score on a scale
Standard Error 1.05
|
1.05 score on a scale
Standard Error 1.36
|
Adverse Events
Unacylated Ghrelin
Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Unacylated Ghrelin
n=12 participants at risk
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
Placebo
n=13 participants at risk
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
|---|---|---|
|
General disorders
Acute coronary syndrome
|
8.3%
1/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
0.00%
0/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Leukocytosis
|
0.00%
0/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
7.7%
1/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Coronary revascularization
|
8.3%
1/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
0.00%
0/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Colon Cancer surgery
|
8.3%
1/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
0.00%
0/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Gastroenteritis
|
0.00%
0/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
7.7%
1/13 • Number of events 2 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Stroke
|
0.00%
0/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
7.7%
1/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Hernia repair
|
0.00%
0/12 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
7.7%
1/13 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
Other adverse events
| Measure |
Unacylated Ghrelin
n=12 participants at risk
Participants randomized to unacylated ghrelin will self-administer 20 ug/kg unacylated ghrelin daily. Study drug is dispensed in syringes labeled with the participant's name, date of birth, expiration date, and instructions for administration. Syringes will NOT be labeled with the group assignment, ensuring that both the research team collecting data and study participants are blinded to group assignment (i.e. double blinded status). Study drug is stored and handled according to the University of Chicago Research Pharmacy Standard Operating Procedure (SOP).
|
Placebo
n=13 participants at risk
Participants randomized to placebo will self-administer an identical-appearing solution of bacteriostatic saline daily.
|
|---|---|---|
|
General disorders
Swelling
|
8.3%
1/12 • Number of events 2 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
15.4%
2/13 • Number of events 3 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Dizziness or lightheadedness
|
16.7%
2/12 • Number of events 3 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
15.4%
2/13 • Number of events 2 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Headaches
|
16.7%
2/12 • Number of events 3 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
38.5%
5/13 • Number of events 8 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
|
General disorders
Other symptoms
|
33.3%
4/12 • Number of events 7 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
46.2%
6/13 • Number of events 8 • Adverse events data were collected monthly from baseline to 4-month follow-up.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place