| Trial ID: | L5684 |
| Source ID: | NCT02293837
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| Associated Drug: |
Tocilizumab (Tcz)
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| Title: |
Tocilizumab (TCZ) in New-onset Type 1 Diabetes
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| Acronym: |
EXTEND
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| Status: |
COMPLETED
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| Study Results: |
YES
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| Results: |
https://ClinicalTrials.gov/show/NCT02293837/results
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| Conditions: |
Type 1 Diabetes Mellitus|New-onset Type 1 Diabetes Mellitus|T1DM|T1D
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| Interventions: |
DRUG: Tocilizumab (TCZ)|DRUG: Placebo|OTHER: Standard of Care
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| Outcome Measures: |
Primary: Change From Baseline in 2-Hour C-peptide Mean Area Under the Curve (mAUC) in Pediatric Participants, C-peptide is a substance released by the pancreas into the bloodstream in equal amounts to insulin and reflects how much insulin pancreatic beta cells are making. The standardized MMTT evaluates whether beta cells are producing endogenous insulin. The MMTT was performed in the morning and blood samples for C-peptide collected at baseline (pre-meal) and 15, 30, 60, 90, 120, 150, 180, 210, and 240 minutes post-meal. C-peptide mAUC was calculated using the trapezoidal rule over the 2-hour time period. Larger numbers are preferable (better) in these mAUC results: more insulin being produced reflects less severe disease. C-peptide levels in the serum (e.g., mAUC following a standardized MMTT) compared to control group at 1 year post treatment initiation for the evaluation of investigational products intended to preserve endogenous beta-cell function in T1DM trials is recognized by the Center for Drug Evaluation and Research (CDER) at the FDA as a valid efficacy endpoint., Baseline (Pre-treatment) to Week 52 | Secondary: Change From Baseline in 2-Hour C-peptide Mean Area Under the Curve (mAUC), C-peptide is a substance released by the pancreas into the bloodstream in equal amounts to insulin and reflects how much insulin pancreatic beta cells are making. The standardized MMTT evaluates whether beta cells are producing endogenous insulin. The MMTT was performed in the morning and blood samples for C-peptide collected at baseline (pre-meal) and 15, 30, 60, 90, 120, 150, 180, 210, and 240 minutes post-meal. C-peptide mAUC was calculated using the trapezoidal rule over the 2-hour time period. Larger numbers are preferable (better) in these mAUC results: more insulin being produced reflects less severe disease. C-peptide levels in the serum (e.g., mAUC following a standardized MMTT) compared to control group at 1 year post treatment initiation for the evaluation of investigational products intended to preserve endogenous beta-cell function in T1DM trials is recognized by the Center for Drug Evaluation and Research (CDER) at the FDA as a valid efficacy endpoint., Baseline (Pre-treatment) to Weeks 24, 52, and 104|2-Hour C-peptide Mean Area Under the Curve (mAUC), Mixed Model, C-peptide is a substance released by the pancreas into the bloodstream in equal amounts to insulin and reflects how much insulin pancreatic beta cells are making. The standardized MMTT evaluates whether beta cells are producing endogenous insulin. The MMTT was performed in the morning and blood samples for C-peptide collected at baseline (pre-meal) and 15, 30, 60, 90, 120, 150, 180, 210, and 240 minutes post-meal. C-peptide mAUC was calculated using the trapezoidal rule over the 2-hour time period. Larger numbers are preferable (better) in these mAUC results: more insulin being produced reflects less severe disease. C-peptide levels in the serum (e.g., mAUC following a standardized MMTT) compared to control group at 1 year post treatment initiation for the evaluation of investigational products intended to preserve endogenous beta-cell function in T1DM trials is recognized by the Center for Drug Evaluation and Research (CDER) at the FDA as a valid efficacy endpoint., Baseline (Pre-treatment), Weeks 12, 24, 39, 52, 78, and 104|Change From Baseline in 4-Hour C-peptide Mean Area Under the Curve (mAUC), C-peptide is a substance released by the pancreas into the bloodstream in equal amounts to insulin and reflects how much insulin pancreatic beta cells are making. The standardized MMTT evaluates whether beta cells are producing endogenous insulin. The MMTT was performed in the morning and blood samples for C-peptide collected at baseline (pre-meal) and 15, 30, 60, 90, 120, 150, 180, 210, and 240 minutes post-meal. C-peptide mAUC was calculated using the trapezoidal rule over the 4-hour time period. Larger numbers are preferable (better) in these mAUC results: more insulin being produced reflects less severe disease. C-peptide levels in the serum (e.g., mAUC following a standardized MMTT) compared to control group at 1 year post treatment initiation for the evaluation of investigational products intended to preserve endogenous beta-cell function in T1DM trials is recognized by the Center for Drug Evaluation and Research (CDER) at the FDA as a valid efficacy endpoint., Baseline (Pre-treatment) to Weeks 52 and 104|Change From Baseline in Average Insulin Use in Units Per Kilogram Body Weight Per Day, The need to use exogenous insulin is an indication that the body is not producing enough endogenous insulin. Higher amounts of insulin use indicate higher disease activity. Insulin use was collected each day for 5 days prior to the visit. Average insulin use per kg is the average insulin use over the 5 days prior to the visit divided by the participant's weight in kg., Baseline (Pre-treatment) to Weeks 24, 52, and 104|Change From Baseline in Average Insulin Use Per Kg, Mixed Model, The need to use exogenous insulin is an indication that the body is not producing enough endogenous insulin. Higher amounts of insulin use indicate higher disease activity. Insulin use was collected each day for 5 days prior to the visit. Average insulin use per kg is the average insulin use over the 5 days prior to the visit divided by the participant's weight in kg., Baseline (Pre-treatment) to Weeks 12, 24, 39, 52, 78, and 104|Change From Baseline in Hemoglobin A1c, Glycosylated hemoglobin (HbA1c) is a measure of the average plasma concentration of blood sugar (glucose) over the previous three months and measures the level of optimal management of underlying disease. An HbA1c of 5.6% or less is considered normal. HbA1c of 6.5% or higher is typical for individuals with Type 1 Diabetes mellitus (T1DM). The closer HbA1c levels are to normal, the better controlled the disease is., Baseline (Pre-treatment) to Weeks 24, 52, and 104|Change From Baseline in Hemoglobin A1C (HbA1c) Level in Participants, Mixed Model, Glycosylated hemoglobin (HbA1c) is a measure of the average plasma concentration of blood sugar (glucose) over the previous three months and measures the level of optimal management of underlying disease. An HbA1c of 5.6% or less is considered normal. HbA1c of 6.5% or higher is typical for individuals with Type 1 Diabetes mellitus (T1DM). The closer HbA1c levels are to normal, the better controlled the disease is., Baseline (Pre-treatment) to Weeks 12, 24, 39, 52, 78, and 104|Number of Participants Who Experienced at Least One Major Hypoglycemic Event After Treatment Initiation, Major hypoglycemic adverse events are defined as: Blood glucose concentration \< 40 mg/dL (Grades 3-5, NCI-CTCAE version 4.03\*), or hypoglycemic events involving seizure or loss of consciousness (coma) or requiring assistance from another individual in order to recover. \*NCI-CTCAE: National Cancer Institute Common Terminology Criteria for Adverse Events, Day 0 (Treatment Initiation) to Weeks 52 and 104|Number of Participants Who Experienced Infusion-Related Adverse Events, An infusion/dose reaction is defined as an adverse event occurring during and within 24 hours after the infusion or subcutaneous injection of tocilizumab. This may include hypersensitivity reactions or anaphylactic reactions., Day 0 (Treatment Initiation) to Week 52|Number of Participants Who Experienced Hypersensitivity Adverse Events, Signs of a possible hypersensitivity reaction to the study drug include but are not limited to: * Fever, chills, pruritus, urticaria, angioedema, and skin rash * Cardiopulmonary reactions, including chest pain, dyspnea, hypotension or hypertension, Day 0 (Treatment Initiation) to Week 52
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| Sponsor/Collaborators: |
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Collaborators: Immune Tolerance Network (ITN)|PPD DEVELOPMENT, LP|Rho Federal Systems Division, Inc.
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| Gender: |
ALL
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| Age: |
CHILD
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| Phases: |
PHASE2
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| Enrollment: |
136
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| Study Type: |
INTERVENTIONAL
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| Study Designs: |
Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
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| Start Date: |
2015-03-12
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| Completion Date: |
2020-08-31
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| Results First Posted: |
2021-08-17
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| Last Update Posted: |
2021-09-08
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| Locations: |
University of California San Francisco, San Francisco, California, 94143, United States|Stanford University, Stanford, California, 94305, United States|Yale University School of Medicine: Diabetes Endocrinology Research Center, New Haven, Connecticut, 06519, United States|University of Florida, Gainesville, Florida, 32610, United States|University of Miami: Diabetes Research Institute, Miami, Florida, 33136, United States|University of South Florida: Diabetes Center, Tampa, Florida, 33612, United States|Indiana University Health - Riley Hospital for Children, Indianapolis, Indiana, 46202, United States|University of Iowa, Iowa City, Iowa, 52242, United States|Harvard University, Joslin Diabetes Center, Boston, Massachusetts, 002215, United States|University of Minnesota, Minneapolis, Minnesota, 55455, United States|Children's Mercy Hospital, Kansas City, Missouri, 64111, United States|Columbia University, Naomi Berrie Diabetes Center, New York, New York, 10032, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States|Sanford Research, Sioux Falls, South Dakota, 57104, United States|Vanderbilt University, Nashville, Tennessee, 37232, United States|University of Texas Southwestern Medical Center, Dallas, Texas, 75390, United States|Benaroya Research Institute, Seattle, Washington, 98101, United States|The Children's Hospital at Westmead: Kids Research Institute, Westmead, New South Wales, Westmead 2145, Australia|Lady Cilento Children's Hospital: Department of Endocrinology, South Brisbane, Queensland, 4101, Australia
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| URL: |
https://clinicaltrials.gov/show/NCT02293837
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