Trial Outcomes & Findings for Use of Functional MRI to Assess Functional Hypothalamic Activation in Response to Diazoxide (NCT NCT03566511)
NCT ID: NCT03566511
Last Updated: 2025-05-31
Results Overview
Arterial Spin Labeling data was acquired and converted by the Gruss Magnetic Resonance Research Center (MRRC) and reported as Hypothalamic Cerebral Blood Flow (CBF) which is a more meaningful clinical endpoint measurement. Data was collected at three time points during each of the two study visits (pre dosing, 2 hours post dosing, and 5 hours post dosing). CBF values at the respective timepoints are summarized in mL/100g/min units and reported by study arm. An increase in CBF is correlated with an increase in brain activity.
TERMINATED
PHASE2
37 participants
2 hours and 5 hours post dosing
2025-05-31
Participant Flow
37 patients signed consent and were screened for eligibility. Following screening, 22 patients were deemed eligible. Of the 22, 11 did not enroll in the study after passing the screening for various reasons including being lost to follow-up or unable to commit. In summary, 11 patients were enrolled and randomized into the study; however, randomization order cannot be verified from documentation for 3 study patients. These 3 patients did not complete the overall study and were never unblinded.
Participant milestones
| Measure |
Diazoxide First Then Matched Placebo
Proglycem, oral suspension (4-7 mg/kg). Healthy participants will receive Diazoxide between MRI scans.
Diazoxide: Healthy participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Healthy participants will receive placebo between baseline MRI scan and second MRI scan.
|
Matched Placebo First Then Diazoxide
Proglycem, oral suspension (4-7 mg/kg). T2D participants will receive Diazoxide between MRI scans.
Diazoxide: T2D participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
T2D participants will receive placebo between baseline MRI scan and second MRI scan.
|
|---|---|---|
|
1st Intervention (1 Day)
STARTED
|
4
|
4
|
|
1st Intervention (1 Day)
COMPLETED
|
3
|
4
|
|
1st Intervention (1 Day)
NOT COMPLETED
|
1
|
0
|
|
Washout (~1 Month)
STARTED
|
3
|
4
|
|
Washout (~1 Month)
COMPLETED
|
3
|
4
|
|
Washout (~1 Month)
NOT COMPLETED
|
0
|
0
|
|
2nd Intervention (1 Day)
STARTED
|
3
|
4
|
|
2nd Intervention (1 Day)
COMPLETED
|
3
|
4
|
|
2nd Intervention (1 Day)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
Diazoxide First Then Matched Placebo
Proglycem, oral suspension (4-7 mg/kg). Healthy participants will receive Diazoxide between MRI scans.
Diazoxide: Healthy participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Healthy participants will receive placebo between baseline MRI scan and second MRI scan.
|
Matched Placebo First Then Diazoxide
Proglycem, oral suspension (4-7 mg/kg). T2D participants will receive Diazoxide between MRI scans.
Diazoxide: T2D participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
T2D participants will receive placebo between baseline MRI scan and second MRI scan.
|
|---|---|---|
|
1st Intervention (1 Day)
Study terminated and participant never received Diazoxide (DZX)
|
1
|
0
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
All Study Participants
n=11 Participants
Proglycem, oral suspension (4-7 mg/kg). Healthy participants will receive Diazoxide between MRI scans.
Diazoxide: Participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Participants will receive placebo between baseline MRI scan and second MRI scan.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=11 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=11 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=11 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=11 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=11 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=11 Participants
|
PRIMARY outcome
Timeframe: 2 hours and 5 hours post dosingPopulation: 7 patients were able to complete both studies within the crossover design as described in the 'Participant Flow' module and have their data analyzed. An additional 4 patients (3 healthy, and 1 T2D) were not able to complete their measurements and, therefore, no data are available for these patients. Standard error (SE) data for the DZX studies were derived from visual estimates of the bar graphs. SE data for the Placebo studies were extrapolated from DZX studies.
Arterial Spin Labeling data was acquired and converted by the Gruss Magnetic Resonance Research Center (MRRC) and reported as Hypothalamic Cerebral Blood Flow (CBF) which is a more meaningful clinical endpoint measurement. Data was collected at three time points during each of the two study visits (pre dosing, 2 hours post dosing, and 5 hours post dosing). CBF values at the respective timepoints are summarized in mL/100g/min units and reported by study arm. An increase in CBF is correlated with an increase in brain activity.
Outcome measures
| Measure |
Diazoxide
n=7 Participants
Proglycem, oral suspension (4-7 mg/kg). Healthy participants will receive Diazoxide between MRI scans.
Diazoxide: Participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Participants will receive placebo between baseline MRI scan and second MRI scan.
|
Matched Placebo
n=7 Participants
Proglycem, oral suspension (4-7 mg/kg). T2D participants will receive Diazoxide between MRI scans.
Diazoxide: Participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Participants will receive placebo between baseline MRI scan and second MRI scan.
|
|---|---|---|
|
Change in Arterial Spin Labeling (ASL) Signal Measured Using 3T MRI From Baseline to 5 Hours Post Dosing
Baseline
|
29.78 mL/100g/min
Standard Error 1.3
|
28.36 mL/100g/min
Standard Error 1.3
|
|
Change in Arterial Spin Labeling (ASL) Signal Measured Using 3T MRI From Baseline to 5 Hours Post Dosing
2 hours
|
26.52 mL/100g/min
Standard Error 1.7
|
26.25 mL/100g/min
Standard Error 2.0
|
|
Change in Arterial Spin Labeling (ASL) Signal Measured Using 3T MRI From Baseline to 5 Hours Post Dosing
5 hours
|
28.88 mL/100g/min
Standard Error 1.4
|
27.75 mL/100g/min
Standard Error 1.4
|
Adverse Events
Diazoxide
Matched Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Diazoxide
n=7 participants at risk
Proglycem, oral suspension (4-7 mg/kg). Healthy participants will receive Diazoxide between MRI scans.
Diazoxide: Participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Participants will receive placebo between baseline MRI scan and second MRI scan.
|
Matched Placebo
n=7 participants at risk
Proglycem, oral suspension (4-7 mg/kg). T2D participants will receive Diazoxide between MRI scans.
Diazoxide: Participants will receive Diazoxide at a dose of 4-7 mg/kg (based upon body weight) between baseline MRI scan and second MRI scan.
Matched placebo:
Participants will receive placebo between baseline MRI scan and second MRI scan.
|
|---|---|---|
|
General disorders
Uncomfortable
|
0.00%
0/7 • Two, 8-hour visits, up to one month apart
|
14.3%
1/7 • Two, 8-hour visits, up to one month apart
|
Additional Information
Dr. Meredith Hawkins
Albert Einstein College of Medicine
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place