A Randomized Phase 1/2 Trial of Low Dose Anti-thymocyte Globulin (ATG) With Subsequent Adalimumab or Verapamil in New Onset Type 1 Diabetes
NCT: NCT07061574 ·
Status: RECRUITING ·
Phase: Phase 2
· Sponsor: City of Hope Medical Center
· Started: 2026-03-30
· Est. Completion: 2031-04-15
Official Summary
This multi-center randomized controlled trial will assess the safety and efficacy of ATG followed by either adalimumab or verapamil in preserving insulin secretion 2 years from randomization in persons aged 9 to \<21 with recent-onset stage 3 T1D.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: RANDOMIZED
- Model: PARALLEL
- Masking: TRIPLE
- Enrollment: 120 participants
Interventions
- DRUG: Anti-thymocyte globulin (ATG) — ATG (brand name Thymoglobulin) a polyclonal T cell antibody preparation. The first dose (0.5 mg/kg) will be infused on day 0, during a period of 6 hours. The second dose (2 mg/kg) will be given on day 1, over a period of 4 to 6 hours.
- DRUG: verapamil extended-release capsule — Daily oral (pill) administration at 60, 120, 240 or 360 mg based on weight and ECG findings.
- DRUG: Adalimumab — 40 mg administered subcutaneously every other week beginning 6 weeks after the last dose of ATG until the 156-week visit.
Primary Outcomes
- Stimulated C-peptide AUC (Week 104)
Secondary Outcomes
- Severe Adverse Events (week13, week 26, week 52, week 78, and week 104)
- Adverse Events (Week 104)
- Severe Hypoglycemia (Week 104)
- Diabetic ketoacidosis (DKA) (Week 104)
- Number of Participants with CD4 count <500 mm3 (Week 104)
Eligibility Criteria
Key Inclusion Criteria: Recent-onset stage 3 T1D diagnosed by standard ADA criteria, with the ability to be randomized within 6 months from the date of T1D diagnosis and within 37 days of Screening Visit. * At least one positive T1D auto-antibody. * If clearly positive (≥20% above local lab's ULN) at screening, repeat antibody testing for central lab is not required. * Insulin auto-antibodies are only considered if exogenous insulin use is \<10 days when blood is drawn. * Must have stimulated C-peptide levels ≥0.2 pmol/mL measured during MMTT conducted prior to randomization. * Age 9 to \<21 years at the time of randomization. * Body weight \>30kg. * BMI \<95th percentile for age and gender. * Willing to comply with intensive diabetes management. * Female participants with childbearing potential are not currently pregnant, are willing to avoid pregnancy and breastfeeding, and to undergo pregnancy testing prior to MMTTs for the duration of the study. * Women of childbearing potential (WOCBP) must use an acceptable form of birth control. Acceptable forms include oral/injection contraceptives, transdermal contraceptives, diaphragm, intrauterine devices, condoms with spermicide, documented surgical sterilization of either the participant or their partner or abstinence. * Male participants with potential to father children must be willing to use abstinence or adequate contraceptive methods for the duration of the study. * Males must agree to be sexually abstinent or use a condom and agree not to donate sperm for the treatment period and for a minimum of 1 spermatogenesis cycle (90 days after last dose of study drug) after last treatment. * Willing to provide informed consent and child assent as applicable. * Sufficient cognitive ability, per investigator judgment, to provide informed consent for study participation on an IRB approved consent form. * Able to read and understand English or Spanish (both participant and legally authorized representative, if applicable). * Must be fully vaccinated for age. * Must have been vaccinated for flu (if currently in flu season). * Must be willing to not receive live vaccines throughout the treatment period. * Must be willing to not use any non-insulin glucose-lowering agents such as GLP-1 agonists (including for weight loss indication), symlin, DPP-4 inhibitors, SGLT-2 inhibitors, biguanides, sulfonylureas) for the duration of study treatment. Participants are required to go off these drugs at least 30 days prior to screening. Key Exclusion Criteria: * Prior treatment with ATG or known allergy to ATG or rabbit-derived products. * Local lab draw at screening: * Immunodeficient or have clinically significant chronic lymphopenia: Leukopenia (\<3,000 leukocytes /μL), neutropenia (\<1,500 neutrophils/μL), lymphopenia (\<800 lymphocytes/μL). * Thrombocytopenia (\<100,000 platelets/μL) or anemia (hemoglobin \< 10g/dL). * Leukocytosis (\>14,000/mL) * Infections: * Ongoing infection or had recently had a major infection requiring hospitalization or intravenous antibiotics.within 30 days prior to randomization. * Have active signs or symptoms of acute infection at the time of randomization. * Have evidence of prior or current tuberculosis infection as assessed interferon gamma release assay (QuantiFERON), or a positive test for latent tuberculosis. * Have evidence of current or past HIV or Hepatitis B or current Hepatitis C infection. * History of serious bacterial, viral, fungal, or other opportunistic infections. * Have active signs or symptoms of CMV or EBV compatible illness lasting more than 7 days within 30 days of randomization. * Have positive CMV and/or EBV PCR test within 30 days prior to randomization. * Have positive COVID-19 self-antigen test within 3 days of randomization. * History of underlying cardiac disease (ex. left ventricular dysfunction, hypertrophic cardiomyopathy), certain arrhythmias (e.g. AV block, accessory pathway such as Wolff- Parkinson-White or Lown-Ganong-Levine syndromes) or abnormal ECG (unless cleared by cardiology). * Blood pressure (either systolic or diastolic) \<5th percentile for age, gender, and height on two out of three measurements. * Pulse \<2nd percentile for age and gender on two out of three measurements. * History of vasovagal syncopal episodes related to hypotension. * History of malignancies other than of skin. * Use of medications likely to interfere with study results: * Any immunomodulators, including systemic steroids or participation in prior research study in which a potential participant received an immunomodulatory agent (may participate if received placebo only). * Current or previous use of Teplizumab. * Ongoing use of medications known to influence glycemia or glucose tolerance. Only topical steroids are allowed. * Need to use of any of the following medications during the study: beta blocker, seizure medication (carbamazepine, phenobarbital, phenytoin), other antihypertensive medications, HMG-CoA reductase inhibitors
Trial Locations
- UCSF, San Francisco, California, United States
- Barbara Davis Center for Diabetes University of Colorado Anschutz, Aurora, Colorado, United States
- Yale University, New Haven, Connecticut, United States
- University of Florida, Gainesville, Florida, United States
- University of Miami, Miami, Florida, United States
- Indiana University, Indianapolis, Indiana, United States
- University of Minnesota, Minneapolis, Minnesota, United States
- University of Buffalo, Buffalo, New York, United States
- University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Baylor College of Medicine, Houston, Texas, United States
- ...and 1 more locations
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