POLARIS: A Phase 1, Single Dose, Open-label Study of GNTI-122 in Adults With Recently Diagnosed Type 1 Diabetes (T1D)
This is a 78-week single arm, multi-center, Phase 1 study to evaluate the safety, tolerability, cellular kinetics, and biomarker changes in C-peptide over time of GNTI-122, an investigational cell therapy manufactured from a participant's own blood cells in adult participants with recently diagnosed T1D. After assessment of eligibility, participants who qualify for the study will be enrolled sequentially in 1 of 3 cohorts. Cohort 1 participants (n=3) receive a low dose of GNTI-122 . Cohort 2 participants (n=3) receive a high dose of GNTI-122. Cohort 3 participants (n=10) receive a high dose of GNTI-122 in combination with rapamycin. Participants are followed for 78 weeks after the administration of GNTI-122 during which safety and efficacy assessments are made, including vital signs, ECG, physical exam, clinical labs, and monitoring of adverse events and concomitant medications. Disease markers (e.g., MMTT-stimulated C-peptide, HbA1c) and pharmacodynamic activity (e.g., lymphocyte subsets and phenotypes, effector T cell responses to islet antigens ex vivo, T1D autoantibodies) will be monitored serially throughout the study. The study will include sentinel dosing and a Safety Review Committee to ensure participant safety. Visit https://www.polarisstudy.com to learn more!
• Male and female participants aged ≥18 to ≤55 years with recently diagnosed (within 180 days of Screening) T1D according to American Diabetes Association criteria.
• Participant has residual β-cell function during Screening, defined as random C-peptide ≥ 0.2 nmol/L.
• Positive for at least one T1D-associated autoantibody.
• Able and willing to provide written, informed consent as approved by the IRB.
• Is confirmed positive for the HLA-DRB1\*04:01 allele.
• Has adequate vascular access to undergo leukapheresis with no known contraindications.
⁃ 8\. Female participants of childbearing potential must have a negative serum pregnancy test at Screening, must be not lactating, and must agree to protocol-specified contraception.
⁃ 9\. Male participants of childbearing potential must agree to protocol specified contraception.
⁃ 10\. Other than T1D, participant is in good general health.