Trial Outcomes & Findings for Phase II Safety and Efficacy Study of Oral ORMD-0801 in Patients With Type 2 Diabetes Mellitus (NCT NCT02496000)

Last Updated: 2019-11-13

Results Overview

The Effect of ORMD-0801 (Doses 1 \& 2, Pooled) on Mean Night Time Glucose Levels (measured in mg/dL) Based on 2 Nights of Continuous Glucose Monitor (CGM) Data by Comparison of the Mean Change Between Baseline and Wk 4 of ORMD-0801 Treatment and Placebo Groups. The primary analysis will be based on the results from the two last days, unless technical difficulties preclude calculation of the weighted mean glucose levels. In this case, the last two days (selected between days 5, 6, and 7) with at least 80% of the expected number of measurements will be used. If days 5, 6 and 7 do not have 2 days with at least 80% of the expected number of measurements for a specific subject, then the value will be missing for that subject.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

188 participants

Primary outcome timeframe

Baseline-Study day -7 (± 1 day) through Study day 1 (± 1 day), and Week 4 -Study day 22 (± 1 day) through Study day 29 (± 1 day)

Results posted on

2019-11-13

Participant Flow

Study design: screening, followed by a 14 day wash-out followed by a 14-day single-blind run-in prior to the 28-day treatment period. Efficacy based on the Intend-to-treat population 80% trimming of the data. The safety (adverse events) data is based on the safety population.

Participant milestones

Participant milestones
Measure	Placebo Comparator three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin
Overall Study STARTED	64	61	63
Overall Study COMPLETED	62	56	61
Overall Study NOT COMPLETED	2	5	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Comparator three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin
Overall Study Withdrawal by Subject	1	2	0
Overall Study Lost to Follow-up	1	0	1
Overall Study Protocol Violation	0	0	1
Overall Study Non-compliance or lack of cooperation	0	2	0
Overall Study undetermined	0	1	0

Baseline Characteristics

Phase II Safety and Efficacy Study of Oral ORMD-0801 in Patients With Type 2 Diabetes Mellitus

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo Comparator n=64 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=61 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=63 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	Total n=188 Participants Total of all reporting groups
Age, Continuous	58.61 years STANDARD_DEVIATION 9.203 • n=5 Participants	57.89 years STANDARD_DEVIATION 8.021 • n=7 Participants	57.25 years STANDARD_DEVIATION 8.786 • n=5 Participants	57.92 years STANDARD_DEVIATION 8.69 • n=4 Participants
Sex: Female, Male Female	35 Participants n=5 Participants	22 Participants n=7 Participants	29 Participants n=5 Participants	86 Participants n=4 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	39 Participants n=7 Participants	34 Participants n=5 Participants	102 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	31 Participants n=5 Participants	32 Participants n=7 Participants	36 Participants n=5 Participants	99 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	33 Participants n=5 Participants	29 Participants n=7 Participants	27 Participants n=5 Participants	89 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	6 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	2 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants
Race (NIH/OMB) Black or African American	7 Participants n=5 Participants	8 Participants n=7 Participants	4 Participants n=5 Participants	19 Participants n=4 Participants
Race (NIH/OMB) White	53 Participants n=5 Participants	50 Participants n=7 Participants	55 Participants n=5 Participants	158 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline-Study day -7 (± 1 day) through Study day 1 (± 1 day), and Week 4 -Study day 22 (± 1 day) through Study day 29 (± 1 day)

Population: Intend-to-Treat Population, 80% trimming. The mean values will be analyzed using a one-way analysis of variance (ANOVA) model. The residuals from the ANOVA will be analyzed to verify that they are normally distributed. If not normally distributed, then a Kruskal-Wallis test (one-way analysis of variance on the ranks) will be performed.

Outcome measures

Outcome measures
Measure	Placebo Comparator n=46 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=44 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=47 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	ORMD-0801 Doses 1 and 2 Combined n=91 Participants The combined measurements of dose 1 and dose 2, in units of mg/dL
Measure of the Mean Night Time Glucose Levels Based on Two Nights of Glucose Measurements.	13.70 mg/dL Standard Deviation 26.140	-3.67 mg/dL Standard Deviation 18.983	6.64 mg/dL Standard Deviation 26.361	1.66 mg/dL Standard Deviation 23.541

SECONDARY outcome

Timeframe: Study day -7 (± 1 day) through Study day 1 (± 1 day), and Study day 22 (±1 day) - Study day 29 (± 1 day)

The effect of ORMD-0801 (Dose 1 and Dose 2 individually) on mean 24-hour glucose based on 2 nights of CGM data by comparison of the mean percent change between baseline and Wk 4 of ORMD-0801 treatment and the placebo groups.

Outcome measures

Outcome measures
Measure	Placebo Comparator n=56 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=54 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=59 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	ORMD-0801 Doses 1 and 2 Combined The combined measurements of dose 1 and dose 2, in units of mg/dL
The Effect of ORMD-0801 on Mean 24-hour Glucose	5.34 percent change Standard Deviation 13.881	3.33 percent change Standard Deviation 21.499	1.93 percent change Standard Deviation 19.795	—

SECONDARY outcome

Timeframe: Baseline (Run-in days 13-14) and Study day 1 (± 1 day) through Study day 29 (± 1 day)

Population: Intend to Treat (ITT)

The percent change in the Continuous Glucose Monitoring Mean Fasting Glucose between treatment and mean of the last two days of the baseline(run-in period).

Outcome measures

Outcome measures
Measure	Placebo Comparator n=58 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=54 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=58 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	ORMD-0801 Doses 1 and 2 Combined The combined measurements of dose 1 and dose 2, in units of mg/dL
Measure Percent Change in Continuous Glucose Monitoring Mean Fasting Glucose Between Treatment and Run-In	12.76 percent change Standard Deviation 23.245	4.54 percent change Standard Deviation 24.153	3.28 percent change Standard Deviation 27.331	—

SECONDARY outcome

Timeframe: Study day 1 (±1 day) through Study day43 (± 1 day)

Population: Intend to Treat (ITT) population

The measurement of the change in Morning Fasting C-peptide between baseline to end of the study, measured in Nmol/L

Outcome measures

Outcome measures
Measure	Placebo Comparator n=54 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=54 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=58 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	ORMD-0801 Doses 1 and 2 Combined The combined measurements of dose 1 and dose 2, in units of mg/dL
Measure the Change From Baseline to End of the Study of Morning Fasting C-Peptide (Nmol/L)	0.08 Nmol/L Standard Deviation 0.252	0.03 Nmol/L Standard Deviation 0.476	0.7 Nmol/L Standard Deviation 0.300	—

SECONDARY outcome

Timeframe: Study day 1 (± 1 day) through Study day 29 (± 1 day)

Population: Intend To Treat (ITT) population

The effect of ORMD-0801 (Dose 1 and Dose 2 individually) on the percent change from baseline to Wk 4 in HbA1c

Outcome measures

Outcome measures
Measure	Placebo Comparator n=58 Participants three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=53 Participants three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=59 Participants three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin	ORMD-0801 Doses 1 and 2 Combined The combined measurements of dose 1 and dose 2, in units of mg/dL
The Effect of ORMD-0801 on the Percent Change in HbA1c	0.2 percent change Standard Deviation 0.497	0.00 percent change Standard Deviation 0.544	-.03 percent change Standard Deviation 0.554	—

Adverse Events

Placebo Comparator

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

ORMD-0801 Dose 1

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

ORMD-0801 Dose 2 = 1.5 * Dose 1

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo Comparator n=64 participants at risk three identical capsules containing placebo Placebo Comparator: Placebo	ORMD-0801 Dose 1 n=61 participants at risk three identical capsules, as follows: capsule #1: one half of Dose 1 capsule #2: one half of Dose 1 capsule #3: placebo ORMD-0801: Oral Insulin	ORMD-0801 Dose 2 = 1.5 * Dose 1 n=63 participants at risk three identical capsules, as follows: capsule #1, 2, and 3: one half of Dose 1 ORMD-0801: Oral Insulin
Nervous system disorders Serious Non-Treatment Emergent Adverse Event	0.00% 0/64 • eight (8) months	1.6% 1/61 • Number of events 1 • eight (8) months	0.00% 0/63 • eight (8) months

Other adverse events

Adverse event data not reported

Additional Information

Joel M. Neutel, M. D.

Orange County Research Center

Phone: 714-541-5591

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60