Trial Outcomes & Findings for Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus (NCT NCT00419562)
NCT ID: NCT00419562
Last Updated: 2020-05-07
Results Overview
Primary outcome is reported as the rate of type 1 diabetes per year among the primary stratum; type 1 diabetes was diagnosed based on metabolic testing and assessment of symptoms. This is calculated by dividing the number of participants who develop diabetes by the total number of years of follow-up.
COMPLETED
PHASE3
560 participants
Metabolic and immunological tests were conducted every 6 months; participants were followed for a median of 2.7 years
2020-05-07
Participant Flow
Participant milestones
| Measure |
7.5 mg Oral Insulin Capsules
given before breakfast on a daily basis.
|
Placebo Capsule
designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Overall Study
STARTED
|
283
|
277
|
|
Overall Study
COMPLETED
|
250
|
246
|
|
Overall Study
NOT COMPLETED
|
33
|
31
|
Reasons for withdrawal
| Measure |
7.5 mg Oral Insulin Capsules
given before breakfast on a daily basis.
|
Placebo Capsule
designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
12
|
13
|
|
Overall Study
Withdrawal by Subject
|
20
|
18
|
|
Overall Study
Participant Institutionalized
|
1
|
0
|
Baseline Characteristics
Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus
Baseline characteristics by cohort
| Measure |
Oral Insulin
n=283 Participants
7.5 mg oral insulin capsules given before breakfast on a daily basis.
|
Placebo
n=277 Participants
Placebo capsule designed to mimic appearance of treatment capsule
|
Total
n=560 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.2 years
n=99 Participants
|
8.2 years
n=107 Participants
|
8.2 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
113 Participants
n=99 Participants
|
107 Participants
n=107 Participants
|
220 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
170 Participants
n=99 Participants
|
170 Participants
n=107 Participants
|
340 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
27 Participants
n=99 Participants
|
25 Participants
n=107 Participants
|
52 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
256 Participants
n=99 Participants
|
252 Participants
n=107 Participants
|
508 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Self-Reported Race · White
|
252 Participants
n=99 Participants
|
249 Participants
n=107 Participants
|
501 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Self-Reported Race · African American
|
8 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Self-Reported Race · Asian/Pacific Islander
|
4 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Self-Reported Race · Not Reported
|
19 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
32 Participants
n=206 Participants
|
|
Region of Enrollment
Canada
|
17 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
32 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
226 Participants
n=99 Participants
|
227 Participants
n=107 Participants
|
453 Participants
n=206 Participants
|
|
Region of Enrollment
Finland
|
4 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Region of Enrollment
Italy
|
7 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
|
Region of Enrollment
United Kingdom
|
6 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Region of Enrollment
Australia
|
7 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
Region of Enrollment
Sweden
|
5 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Region of Enrollment
Germany
|
9 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Region of Enrollment
New Zealand
|
3 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Stratum
1
|
203 Participants
n=99 Participants
|
186 Participants
n=107 Participants
|
389 Participants
n=206 Participants
|
|
Stratum
2
|
28 Participants
n=99 Participants
|
27 Participants
n=107 Participants
|
55 Participants
n=206 Participants
|
|
Stratum
3
|
51 Participants
n=99 Participants
|
63 Participants
n=107 Participants
|
114 Participants
n=206 Participants
|
|
Stratum
4
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Metabolic and immunological tests were conducted every 6 months; participants were followed for a median of 2.7 yearsPrimary outcome is reported as the rate of type 1 diabetes per year among the primary stratum; type 1 diabetes was diagnosed based on metabolic testing and assessment of symptoms. This is calculated by dividing the number of participants who develop diabetes by the total number of years of follow-up.
Outcome measures
| Measure |
Oral Insulin
n=203 Participants
7.5 mg oral insulin capsules given before breakfast on a daily basis.
|
Placebo
n=186 Participants
Placebo capsule designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Rate of Type 1 Diabetes Per Year Among Individuals in the Primary Stratum When Treated With Oral Inulin Versus Placebo
|
0.088 Proportion with diabetes/year
Interval 0.067 to 0.112
|
0.102 Proportion with diabetes/year
Interval 0.078 to 0.129
|
SECONDARY outcome
Timeframe: Metabolic and immunological tests were conducted every 6 months; participants were followed for a median of 2.7 yearsPopulation: Stratum 2: mIAA Confirmed, (ICA Confirmed) OR (ICA Not Confirmed AND ICA512+ AND GAD65ab+), low functioning beta cells
Secondary outcome is reported as the rate of type 1 diabetes per year among secondary stratum 2; type 1 diabetes was diagnosed based on metabolic testing and assessment of symptoms. This is calculated by dividing the number of participants who develop diabetes by the total number of years of follow-up.
Outcome measures
| Measure |
Oral Insulin
n=28 Participants
7.5 mg oral insulin capsules given before breakfast on a daily basis.
|
Placebo
n=27 Participants
Placebo capsule designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Rate of Type 1 Diabetes Per Year in Secondary Stratum (Stratum 2) When Treated With Oral Insulin Versus Placebo
|
0.181 Proportion with diabetes/year
Interval 0.096 to 0.291
|
0.341 Proportion with diabetes/year
Interval 0.206 to 0.511
|
SECONDARY outcome
Timeframe: Metabolic and immunological tests were conducted every 6 months; participants were followed for a median of 2.7 yearsPopulation: Combine stratum 3 (mIAA Confirmed, ICA Not Confirmed, ICA512+ OR GAD65ab+, high functioning beta cells) and stratum 4 (mIAA Confirmed, ICA Not Confirmed, ICA512+ OR GAD65ab+, low functioning beta cells)
Secondary outcome is reported as the rate of type 1 diabetes per year among secondary stratum 3+4; type 1 diabetes was diagnosed based on metabolic testing and assessment of symptoms. This is calculated by dividing the number of participants who develop diabetes by the total number of years of follow-up.
Outcome measures
| Measure |
Oral Insulin
n=52 Participants
7.5 mg oral insulin capsules given before breakfast on a daily basis.
|
Placebo
n=64 Participants
Placebo capsule designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Rate of Type 1 Diabetes in Secondary Stratum (Stratum 3+4) When Treated With Oral Insulin Versus Placebo
|
0.051 Proportion with diabetes/year
Interval 0.026 to 0.086
|
0.047 Proportion with diabetes/year
Interval 0.022 to 0.08
|
Adverse Events
Oral Insulin
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Oral Insulin
n=283 participants at risk
7.5 mg oral insulin capsules given before breakfast on a daily basis.
|
Placebo
n=277 participants at risk
Placebo capsule designed to mimic appearance of treatment capsule
|
|---|---|---|
|
Infections and infestations
Infection
|
23.7%
67/283 • Number of events 134
|
22.4%
62/277 • Number of events 120
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory
|
10.6%
30/283 • Number of events 51
|
10.8%
30/277 • Number of events 37
|
|
Endocrine disorders
Endocrine
|
6.4%
18/283 • Number of events 18
|
4.3%
12/277 • Number of events 12
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal/Soft Tissue
|
13.4%
38/283 • Number of events 45
|
6.5%
18/277 • Number of events 20
|
|
Immune system disorders
Allergy/Immunology
|
6.0%
17/283 • Number of events 18
|
4.0%
11/277 • Number of events 11
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin
|
8.8%
25/283 • Number of events 29
|
6.5%
18/277 • Number of events 20
|
|
Gastrointestinal disorders
Gastrointestinal
|
9.9%
28/283 • Number of events 30
|
9.0%
25/277 • Number of events 34
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place