Clinical trial NCT02734277: Type 1 Diabetes Extension Study
Official title: Type 1 Diabetes Extension Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Type 1 Diabetes Mellitus
T1DM
T1D
Keywords: Insulin
Glucose Intolerance
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Sponsor class: NIH
Collaborators: name: Immune Tolerance Network (ITN)
First posted: 2016-04-12
Enrollment: 111
Sex: ALL
Minimum age (years): 8
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Former participants in Immune Tolerance Network (ITN) new-onset Type 1 Diabetes Mellitus (T1DM) studies with immunomodulatory agents as the intervention.

Brief summary: This is a multi-center, prospective, non-interventional study that focuses on the long- term effects following participation in selected ITN new-onset Type1 Diabetes Mellitus studies with immunomodulatory agents (T1DM, T1D).

This observational study will:

* follow participants to determine how long they continue to produce insulin, and
* will also assess how changes in the immune system over time relate to the ability to produce insulin.

This information could help design better therapies for type 1 diabetes in the future.

Detailed description: Depending upon a participant's level of insulin production, participation may be as short as one return visit or a maximum of five years. Evaluation visits will include:

* Overall health assessments
* Blood and urine collections
* Mixed meal tolerance test (MMTTs) for certain participants, per protocol.

Eligibility criteria: Inclusion Criteria:

* Prior participant in an Immune Tolerance Network (ITN) executive committee approved T1DM study.
* Ability to sign informed consent/assent (as applicable for children).

Exclusion Criteria:

* Any medical condition that in the opinion of the principal investigator would interfere with safe completion of the trial; or
* Inability to comply with the study visit schedule and required assessments.

Primary outcomes:
measure: Change in Beta Cell Function by MMTT-Stimulated Mean C-peptide Area Under the Curve (AUC)
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
Secondary outcomes:
measure: Change in Insulin Use in Units per Kilogram Body Weight Per Day
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
measure: Change in HbA1C
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
measure: Count of Participant-Reported Major Hypoglycemic Events
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
measure: Time to Undetectable C-Peptide
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
measure: Frequency of Grade 3 or Higher Adverse Events (AEs) of Interest
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
measure: Severity of Grade 3 or Higher Adverse Events (AEs) of Interest
timeFrame: Baseline (Visit 0) to Month 60 (Year 5)
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF School of Medicine
state: California
city: Stanford
country: United States
facility: Stanford University
state: California
city: Aurora
country: United States
facility: University of Colorado School of Medicine: Barbara Davis Center for Diabetes
state: Colorado
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University Riley Hospital for Children
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Health Care Division of Pediatric Endocrinology
state: Iowa
city: Boston
country: United States
facility: Joslin Diabetes Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Sioux Falls
country: United States
facility: Sanford Research
state: South Dakota
city: Seattle
country: United States
facility: Benaroya Research Institute
state: Washington
Study officials:
affiliation: Riley Hospital for Children at Indiana University Health
name: Linda A. DiMeglio, MD, MPH,MA
role: STUDY_CHAIR
References:
citation: Herold KC, Gitelman SE, Ehlers MR, Gottlieb PA, Greenbaum CJ, Hagopian W, Boyle KD, Keyes-Elstein L, Aggarwal S, Phippard D, Sayre PH, McNamara J, Bluestone JA; AbATE Study Team. Teplizumab (anti-CD3 mAb) treatment preserves C-peptide responses in patients with new-onset type 1 diabetes in a randomized controlled trial: metabolic and immunologic features at baseline identify a subgroup of responders. Diabetes. 2013 Nov;62(11):3766-74. doi: 10.2337/db13-0345. Epub 2013 Jul 8.
citation: Rigby MR, Harris KM, Pinckney A, DiMeglio LA, Rendell MS, Felner EI, Dostou JM, Gitelman SE, Griffin KJ, Tsalikian E, Gottlieb PA, Greenbaum CJ, Sherry NA, Moore WV, Monzavi R, Willi SM, Raskin P, Keyes-Elstein L, Long SA, Kanaparthi S, Lim N, Phippard D, Soppe CL, Fitzgibbon ML, McNamara J, Nepom GT, Ehlers MR. Alefacept provides sustained clinical and immunological effects in new-onset type 1 diabetes patients. J Clin Invest. 2015 Aug 3;125(8):3285-96. doi: 10.1172/JCI81722. Epub 2015 Jul 20.
citation: Rigby MR, DiMeglio LA, Rendell MS, Felner EI, Dostou JM, Gitelman SE, Patel CM, Griffin KJ, Tsalikian E, Gottlieb PA, Greenbaum CJ, Sherry NA, Moore WV, Monzavi R, Willi SM, Raskin P, Moran A, Russell WE, Pinckney A, Keyes-Elstein L, Howell M, Aggarwal S, Lim N, Phippard D, Nepom GT, McNamara J, Ehlers MR; T1DAL Study Team. Targeting of memory T cells with alefacept in new-onset type 1 diabetes (T1DAL study): 12 month results of a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Diabetes Endocrinol. 2013 Dec;1(4):284-94. doi: 10.1016/S2213-8587(13)70111-6. Epub 2013 Sep 23.