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Imatinib Treatment in Recent Onset Type 1 Diabetes Mellitus

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ClinicalTrials.gov Identifier: NCT01781975
Recruitment Status : Completed
First Posted : February 1, 2013
Results First Posted : August 8, 2018
Last Update Posted : September 28, 2018
Sponsor:
Collaborator:
Juvenile Diabetes Research Foundation
Information provided by (Responsible Party):
Stephen E. Gitelman, University of California, San Francisco

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Conditions Diabetes Mellitus, Type I
Diabetes Mellitus, Insulin-Dependent, 1
Type 1 Diabetes Mellitus
Insulin-Dependent Diabetes Mellitus 1
IDDM
Interventions Drug: Imatinib Mesylate
Drug: Placebo (For imatinib mesylate)
Enrollment 67

Recruitment Details Recruitment Period: March 2013 to May 2016 at 9 clinical sites (8 US, 1 Australia)
Pre-assignment Details  
Arm/Group Title Imatinib Mesylate Placebo
Hide Arm/Group Description

400 mg imatinib given once daily basis.

Imatinib Mesylate

Placebo given once daily basis.

Placebo (For imatinib mesylate)

Period Title: Overall Study
Started 45 22
Treatment Period 44 22
Follow-Up Period 43 21
Completed 43 21
Not Completed 2 1
Reason Not Completed
Death             1             0
Withdrawal by Subject             1             1
Arm/Group Title Imatinib Mesylate Placebo Total
Hide Arm/Group Description

400 mg imatinib given once daily basis.

Imatinib Mesylate

Placebo given once daily basis.

Placebo (For imatinib mesylate)

Total of all reporting groups
Overall Number of Baseline Participants 45 22 67
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
45
 100.0%
22
 100.0%
67
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants 22 participants 67 participants
28.2  (7.2) 26.2  (6.6) 27  (7.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
Female
18
  40.0%
12
  54.5%
30
  44.8%
Male
27
  60.0%
10
  45.5%
37
  55.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
Hispanic or Latino
2
   4.4%
3
  13.6%
5
   7.5%
Not Hispanic or Latino
43
  95.6%
19
  86.4%
62
  92.5%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
   2.2%
0
   0.0%
1
   1.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
44
  97.8%
21
  95.5%
65
  97.0%
More than one race
0
   0.0%
1
   4.5%
1
   1.5%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
United States
36
  80.0%
18
  81.8%
54
  80.6%
Australia
9
  20.0%
4
  18.2%
13
  19.4%
Number of Autoantibodies Positive  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 22 participants 67 participants
1
8
  17.8%
4
  18.2%
12
  17.9%
2
8
  17.8%
4
  18.2%
12
  17.9%
3
12
  26.7%
1
   4.5%
13
  19.4%
4
8
  17.8%
6
  27.3%
14
  20.9%
5
9
  20.0%
7
  31.8%
16
  23.9%
1.Primary Outcome
Title Area Under the Stimulated C-peptide Curve (AUC) Mean Over the First 2 Hours of a 4 Hour Mixed Meal Tolerance Test at the 1 Year Visit
Hide Description The primary outcome of each participant is the area under the stimulated c-peptide curve (AUC) mean based on data collected at time 0 to 2 hours of a 4-hour mixed meal tolerance test (MMTT) conducted at the primary endpoint visit. The timed measurements are done at: 0, 15, 30, 60, 90, and 120 minutes. The term “AUC mean” comes from the mean value theorem in calculus. It is the value on the scale of the y-axis that is equal to the AUC divided by the range on the x-axis (in this case 120 minutes).
Time Frame Visit 9 (Week 52) at 0, 15, 30, 60, 90, 120 minutes post-dose
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Imatinib Mesylate Placebo
Hide Arm/Group Description:

400 mg imatinib given once daily basis.

Imatinib Mesylate

Placebo given once daily basis.

Placebo (For imatinib mesylate)

Overall Number of Participants Analyzed 45 22
Mean (Standard Deviation)
Unit of Measure: nmol/L
0.834  (0.446) 0.775  (0.278)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Imatinib Mesylate, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.048
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter ANCOVA
Estimated Value 0.0616
Confidence Interval (2-Sided) 90%
0.00176 to 0.121
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.0364
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Area Under the Stimulated C-peptide Curve (AUC) Mean Over 4 Hours at 24 Months
Hide Description Area under the MMTT-stimulated peak, 2 hour C-peptide, and 4 hour C-peptide AUC mean at week 104.
Time Frame Visit 13 (Week 104)
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Change in HbA1c Levels Over Time
Hide Description Changea in HbA1c levels from Week 52 to Week 104
Time Frame Visit 9 (Week 52) and Visit 13 (Week 104)
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Change in Insulin Dose (Units/kg) Over Time
Hide Description Assess insulin use in units per kilogram body weight per day at weeks 52 and 104.
Time Frame Visit 9 (Week 52) and Visit 13 (Week 104)
Outcome Measure Data Not Reported
5.Secondary Outcome
Title Number of Severe Hypoglycemic Events
Hide Description Major hypoglycemic events occurring from randomization at weeks 0, 52 and 104.
Time Frame Visit 0 (Week 0), Visit 9 (Week 52), and Visit 13 (Week 52)
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Number and Severity of Adverse Events
Hide Description Number and severity of adverse events that were reported throughout the study.
Time Frame Adverse Events will be assessed at Visit 0 (week 0), Visit 1 (Week 2), Visit 2 (Week 4), and every month thereafter.
Outcome Measure Data Not Reported
Time Frame 1 year
Adverse Event Reporting Description Adverse events that were not considered Serious Adverse Events (SAE) were monitored/assessed without regard to the specific Adverse Event Term so the categories they belong to are provided below.
 
Arm/Group Title Imatinib Mesylate Placebo
Hide Arm/Group Description

400 mg imatinib given once daily basis.

Imatinib Mesylate

Placebo given once daily basis.

Placebo (For imatinib mesylate)

All-Cause Mortality
Imatinib Mesylate Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/45 (2.22%)      0/22 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Imatinib Mesylate Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/45 (8.89%)      3/22 (13.64%)    
Endocrine disorders     
Diabetes ketoacidosis  1  0/45 (0.00%)  0 1/22 (4.55%)  1
Metabolism and nutrition disorders     
Hypoglycemia  1  1/45 (2.22%)  1 1/22 (4.55%)  1
Pregnancy, puerperium and perinatal conditions     
Fluid in Lungs  1  0/45 (0.00%)  0 1/22 (4.55%)  1
Psychiatric disorders     
Suicide Attempt  1  1/45 (2.22%)  3 0/22 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Wheezing  1  1/45 (2.22%)  1 0/22 (0.00%)  0
Skin and subcutaneous tissue disorders     
Folliculitis  1  1/45 (2.22%)  1 0/22 (0.00%)  0
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Imatinib Mesylate Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   34/45 (75.56%)      13/22 (59.09%)    
Blood and lymphatic system disorders     
Blood and lymphatic system disorders  1  3/45 (6.67%)  4 0/22 (0.00%)  0
Cardiac disorders     
Cardiac disorders  1  2/45 (4.44%)  3 0/22 (0.00%)  0
Ear and labyrinth disorders     
Ear and labyrinth disorders  1  1/45 (2.22%)  1 0/22 (0.00%)  0
Endocrine disorders     
Endocrine disorders  1  4/45 (8.89%)  45 1/22 (4.55%)  2
Eye disorders     
Eye disorders  1  2/45 (4.44%)  2 0/22 (0.00%)  0
Gastrointestinal disorders     
Gastrointestinal disorders  1  7/45 (15.56%)  14 0/22 (0.00%)  0
General disorders     
General disorders & administration site condt’ns  1  2/45 (4.44%)  2 0/22 (0.00%)  0
Hepatobiliary disorders     
Hepatobiliary disorders  1  3/45 (6.67%)  4 2/22 (9.09%)  2
Immune system disorders     
Immune system disorders  1  0/45 (0.00%)  0 1/22 (4.55%)  1
Infections and infestations     
Infections and infestations  1  10/45 (22.22%)  18 4/22 (18.18%)  7
Injury, poisoning and procedural complications     
Injury, poisoning and procedural complications  1  2/45 (4.44%)  4 0/22 (0.00%)  0
Investigations     
Investigations  1  10/45 (22.22%)  16 1/22 (4.55%)  3
Metabolism and nutrition disorders     
Metabolism and nutrition disorders  1  5/45 (11.11%)  10 2/22 (9.09%)  2
Musculoskeletal and connective tissue disorders     
Musculoskeletal and connective tissue disorders  1  2/45 (4.44%)  3 1/22 (4.55%)  1
Nervous system disorders     
Nervous system disorders  1  2/45 (4.44%)  2 0/22 (0.00%)  0
Pregnancy, puerperium and perinatal conditions     
Pregnancy, puerperium and perinatal conditions  1  0/45 (0.00%)  0 1/22 (4.55%)  1
Psychiatric disorders     
Psychiatric disorders  1  4/45 (8.89%)  8 0/22 (0.00%)  0
Renal and urinary disorders     
Renal and urinary disorders  1  1/45 (2.22%)  2 0/22 (0.00%)  0
Reproductive system and breast disorders     
Reproductive system and breast disorders  1  1/45 (2.22%)  1 0/22 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Respiratory, thoracic and mediastinal disorders  1  4/45 (8.89%)  4 1/22 (4.55%)  1
Skin and subcutaneous tissue disorders     
Skin and subcutaneous tissue disorders  1  4/45 (8.89%)  5 2/22 (9.09%)  3
Surgical and medical procedures     
Surgical and medical procedures  1  0/45 (0.00%)  0 1/22 (4.55%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
FDA did not approve for hold on enrollment of pediatric population to be lifted; only adult population was recruited.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Nichole Reed
Organization: University of South Florida
Phone: 813-396-9219
Responsible Party: Stephen E. Gitelman, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT01781975     History of Changes
Other Study ID Numbers: 17-2013-6
First Submitted: January 17, 2013
First Posted: February 1, 2013
Results First Submitted: May 25, 2018
Results First Posted: August 8, 2018
Last Update Posted: September 28, 2018