Imatinib Treatment in Recent Onset Type 1 Diabetes Mellitus
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01781975|
Recruitment Status : Completed
First Posted : February 1, 2013
Results First Posted : August 8, 2018
Last Update Posted : February 11, 2020
Type 1 diabetes mellitus (T1DM) results from the autoimmune destruction of insulin-producing ß cells. Although exogenous insulin is widely available, it is not possible for affected individuals to consistently achieve euglycemia with current technology, and thus they are at risk for devastating long-term complications. This phase II study is designed to evaluate the safety and efficacy of imatinib mesylate as a novel therapy for new-onset T1DM. Imatinib is a first-in-class tyrosine kinase inhibitor.
This study will explore the potential role of short-term therapy with imatinib to induce tolerance and possibly lead to a durable long-term remission of T1DM.
|Condition or disease||Intervention/treatment||Phase|
|Diabetes Mellitus, Type I Diabetes Mellitus, Insulin-Dependent, 1 Type 1 Diabetes Mellitus Insulin-Dependent Diabetes Mellitus 1 IDDM||Drug: Imatinib Mesylate Drug: Placebo (For imatinib mesylate)||Phase 2|
Eligible participants will be randomized to receive either imatinib mesylate or placebo daily.
All participants randomized into this study will be seen at a study site for a follow-up evaluation, 2 weeks and 4 weeks after randomization, and every month month thereafter for the first year. Participants will come in for a visit ever 6 months for the second year.
At the study visits, participants will undergo assessments of their insulin production, immunologic status, and overall health. Subjects will be followed until the conclusion of the study. The trial is expected to last approximately 2-4 years or until the required amount of information is gathered.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||67 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Safety and Efficacy of Imatinib for Preserving Beta-Cell Function in New-Onset Type 1 Diabetes Mellitus|
|Actual Study Start Date :||January 2014|
|Actual Primary Completion Date :||May 2017|
|Actual Study Completion Date :||May 2018|
Experimental: Imatinib Mesylate
400 mg imatinib given once daily basis.
Drug: Imatinib Mesylate
Placebo Comparator: Placebo
Placebo given once daily basis.
Drug: Placebo (For imatinib mesylate)
- 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 [ Time Frame: Visit 9 (Week 52) at 0, 15, 30, 60, 90, 120 minutes post-dose ]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).
- Area Under the Stimulated C-peptide Curve (AUC) Mean Over 4 Hours at 24 Months [ Time Frame: Visit 13 (Week 104) ]Area under the MMTT-stimulated peak, 4 hour C-peptide AUC mean at week 104. The units are reported as nano-moles/Liter because this is AUC mean (the AUC is divided by the time internal so that the units return to the c-peptide units of measure).
- Change in HbA1c Levels Over Time [ Time Frame: Visit 9 (Week 52) and Visit 13 (Week 104) ]Change in HbA1c levels from Week 52 to Week 104
- Change in Insulin Dose (Units/kg) Over Time [ Time Frame: Visit 9 (Week 52) and Visit 13 (Week 104) ]Assess insulin use in units per kilogram body weight per day at weeks 52 and 104.
- Number of Severe Hypoglycemic Events [ Time Frame: Visit 0 (Week 0), Visit 9 (Week 52), and Visit 13 (Week 104) ]Major hypoglycemic events occurring from randomization at weeks 0, 52 and 104.
- Number of Adverse Events [ Time Frame: Adverse Events will be assessed at Visit 0 (week 0), Visit 1 (Week 2), Visit 2 (Week 4), and every month thereafter. ]Number of adverse events that were reported throughout the study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01781975
|United States, California|
|University of California-San Francisco|
|San Francisco, California, United States, 94143|
|United States, Colorado|
|Barbara Davis Center|
|Aurora, Colorado, United States, 80045|
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|United States, Indiana|
|Indianapolis, Indiana, United States, 46202|
|United States, Iowa|
|University of Iowa|
|Iowa City, Iowa, United States, 52242|
|United States, Massachusetts|
|Joslin Diabetes Center|
|Boston, Massachusetts, United States, 02215|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, Texas|
|University of Texas Southwestern|
|Dallas, Texas, United States, 75390|
|Walter and Eliza Hall Institute of Medical Research|
|Melbourne, Victoria, Australia, 3050|
|Principal Investigator:||Stephen E Gitelman, MD||University of California, San Francisco|