Trial Outcomes & Findings for Effect of Methyldopa on MHC Class II Antigen Presentation in Type 1 Diabetes (NCT NCT01883804)

NCT ID: NCT01883804

Last Updated: 2022-01-18

Results Overview

Cryopreserved primary peripheral blood mononuclear cells were used as antigen presenting cells to stimulate engineered T-cells (T-cell receptor transductant) responding to a specific peptide presented by HLA-DQ8. Secreted IL-2 from the engineered T-cell was measured by a highly sensitive ELISA. This was done for both an α-gliadin/DQ8 responding T-cell and a separate insulin/DQ8 responding T-cell.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

30 participants

Primary outcome timeframe

6 Weeks (Baseline and week 6)

Results posted on

2022-01-18

Participant Flow

Participants were recruited through the adult clinic of the Barbara Davis Center, University of Colorado School of Medicine. Recruitment began in July of 2013 and concluded in November of 2015.

A total of 30 participants joined the study. Five participants withdrew prior to the first visit or any study procedures. The remaining 25 participants started the study and 20 completed all study visits and procedures.

Participant milestones

Participant milestones
Measure
Methyldopa Group
All participants selected to continue with Methyldopa administration. Methyldopa: 6 weeks of Methyldopa administration; where the dose will be increased according to safety of efficacy.
Overall Study
STARTED
25
Overall Study
COMPLETED
20
Overall Study
NOT COMPLETED
5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effect of Methyldopa on MHC Class II Antigen Presentation in Type 1 Diabetes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Study Group
n=20 Participants
All participants selected to continue with Methyldopa administration. Methyldopa: 6 weeks of Methyldopa administration; where the dose will be increased according to safety of efficacy.
Age, Continuous
Mean Age
24.75 years
n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
Sex: Female, Male
Male
13 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
19 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
2 Participants
n=5 Participants
Race (NIH/OMB)
White
18 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
20 Participants
n=5 Participants
Duration of Type 1 Diabetes
133 Days
n=5 Participants
Hemoglobin A1c
7.4 Percentage
n=5 Participants
C-Peptide AUC following a 2 Hour Mixed Meal Tolerance Test
0.64 nmol/L/Min
n=5 Participants
Positive Insulin Autoantibodies (Index)
1 Participants
n=5 Participants
Positive GAD Autoantibodies
15 Participants
n=5 Participants
Positive IA-2 Autoantibodies
14 Participants
n=5 Participants
Positive ZnT8 Autoantibodies
11 Participants
n=5 Participants
Systolic Blood Pressure
113.2 mmHg
STANDARD_DEVIATION 10.03 • n=5 Participants
Diastolic Blood Pressure
69.65 mmHg
STANDARD_DEVIATION 8.16 • n=5 Participants
Elevated AST Values
0 Participants
n=5 Participants
Elevated ALT Values
0 Participants
n=5 Participants
Abnormal Hemoglobin Levels
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 6 Weeks (Baseline and week 6)

Cryopreserved primary peripheral blood mononuclear cells were used as antigen presenting cells to stimulate engineered T-cells (T-cell receptor transductant) responding to a specific peptide presented by HLA-DQ8. Secreted IL-2 from the engineered T-cell was measured by a highly sensitive ELISA. This was done for both an α-gliadin/DQ8 responding T-cell and a separate insulin/DQ8 responding T-cell.

Outcome measures

Outcome measures
Measure
Study Group
n=20 Participants
open label treatment; dose escalation of methyldopa
The Change From Baseline of DQ8 Antigen Presentation by Peripheral Blood Mononuclear Cells After 6 Weeks of Methyldopa Treatment.
Baseline, α-gliadin/DQ8
642 pg/mL
Standard Error 104
The Change From Baseline of DQ8 Antigen Presentation by Peripheral Blood Mononuclear Cells After 6 Weeks of Methyldopa Treatment.
6 weeks of treatment, α-gliadin/DQ8
430 pg/mL
Standard Error 70
The Change From Baseline of DQ8 Antigen Presentation by Peripheral Blood Mononuclear Cells After 6 Weeks of Methyldopa Treatment.
Baseline, insulin/DQ8
27.4 pg/mL
Standard Error 5.2
The Change From Baseline of DQ8 Antigen Presentation by Peripheral Blood Mononuclear Cells After 6 Weeks of Methyldopa Treatment.
6 weeks of treatment, insulin/DQ8
17.9 pg/mL
Standard Error 3.3

SECONDARY outcome

Timeframe: 12 weeks (Baseline and week 12)

Investigators aim to observe changes in residual endogenous insulin production as measured by C-peptide 2 hour area under the curve following a Mixed Meal Tolerance Test (MMTT). C-peptide is a measure of endogenous insulin secretion as both are secreted in a 1:1 molar ratio. Individuals ingested a liquid meal (Boost) with a fixed amount of protein, fat and carbohydrate in the fasting state followed by the timed measurements of serum C-peptide at 0, 15, 30, 60, 90 and 120 minutes to compute the AUC.

Outcome measures

Outcome measures
Measure
Study Group
n=20 Participants
open label treatment; dose escalation of methyldopa
The Change in C-Peptide AUC Following a MMTT From Baseline to Study Completion.
Baseline
0.6 nmol/L/min
Standard Deviation 0.5
The Change in C-Peptide AUC Following a MMTT From Baseline to Study Completion.
Study Completion
0.7 nmol/L/min
Standard Deviation 0.6

SECONDARY outcome

Timeframe: 12 weeks (Baseline and week 12)

Investigators aim to observe changes in hemoglobin A1c values, a measure of average blood glucose over the preceding 3 months.

Outcome measures

Outcome measures
Measure
Study Group
n=20 Participants
open label treatment; dose escalation of methyldopa
The Change in Hemoglobin A1c From Baseline to Study Completion.
Baseline
7.4 percentage
Standard Deviation 1.6
The Change in Hemoglobin A1c From Baseline to Study Completion.
Study Completion
6.5 percentage
Standard Deviation 0.8

SECONDARY outcome

Timeframe: 12 weeks (Baseline and week 12)

Exogenous insulin use per kg of body weight.

Outcome measures

Outcome measures
Measure
Study Group
n=20 Participants
open label treatment; dose escalation of methyldopa
The Change in Insulin Use From Baseline to Study Completion.
Baseline
0.37 units/Kg body weight
Standard Deviation 0.20
The Change in Insulin Use From Baseline to Study Completion.
Study Completion
0.36 units/Kg body weight
Standard Deviation 0.19

Adverse Events

Study Group

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Study Group
n=25 participants at risk
All participants selected to continue with Methyldopa administration. Methyldopa: 6 weeks of Methyldopa administration; where the dose will be increased according to safety of efficacy.
General disorders
Fatigue
20.0%
5/25 • Number of events 5 • Over the course of the 12 week study.
Adverse event collection occurred at each visit through standard questions asked by the research coordinator and discussions with the investigator.

Additional Information

Dr. Aaron Michels

Barbara Davis Center for Diabetes, University of Colorado School of Medicine

Phone: 303-724-1923

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place