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VV-14300 for the Treatment of Type 1 Diabetes
Bu Faz 1/2 (Phase 1/2) çalışma, otomatik insülin verme (automated insulin delivery, AID) sistemi kullanmasına rağmen kan şekeri yeterince kontrol altında olmayan, uzun süreli tip 1 diyabetli (type 1 diabetes, T1D) yetişkinlerde VV-14300'ü test ediyor. VV-14300, kas içine (intramuscular) enjekte edilen ve kandaki fazla şekerin uzaklaştırılmasına yardımcı olmak üzere tasarlanmış araştırma aşamasındaki bir gen tedavisidir. Katılımcıların 18–65 yaş arasında olması, beden kitle indeksinin 20,0 ile 30,0 kg/m² altı arasında olması, en az 5 yıldır tip 1 diyabet tanısı bulunması, en az 3 aydır AID sistemi kullanması ve HbA1c değerinin %7'nin üzerinde olması gerekmektedir.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Able to provide signed informed consent. * 18 to 65 years of age (inclusive) at Screening. * Body mass index (BMI) of 20.0 to \<30.0 kg/m². * Clinical diagnosis of Type 1 Diabetes for at least 5 years prior to Screening, currently managed with an Automated Insulin Delivery (AID) system for at least 3 months prior to Screening. * Suboptimal glycemic control (HbA1c \>7% and \<10%), and on a stable insulin regimen at Screening. * Adequate kidney function (estimated glomerular filtration rate \[eGFR\] \>60 mL/min/1.73m²) at Screening. * Females of child-bearing potential must have a negative pregnancy test at Screening and prior to dosing; participants must agree to use highly effective contraception during and for at least 12 months after study intervention administration. * Agree to refrain from donating blood, plasma, platelets, eggs, or sperm during the 12-month Post-Treatment Follow-up Period. * Willing and able to complete all study visits, procedures, and required use of study-provided monitoring devices for the duration of the study. Exclusion Criteria: * Type 2 diabetes or other condition requiring exogenous insulin that is not due to autoimmunity, or when insulin delivery is not managed through an AID system. * Diabetic complications (e.g., severe/proliferative retinopathy or neuropathy) or a clinically significant medical, cognitive, or psychiatric condition that, in the Investigator's opinion, poses additional risk or would make consistent study follow-up unlikely. * Recurrent diabetic ketoacidosis (2 or more events in the 12 months prior to Screening). * Pregnant or breastfeeding. * History of malignancy requiring chemotherapy and/or radiation within the 12 months prior to Screening, except for successfully treated non-melanoma skin cancers (e.g., basal cell, squamous cell carcinomas), cervical intraepithelial neoplasia, and localized prostate cancer. * Clinically significant cardiovascular or cerebrovascular disease, uncontrolled blood pressure, family history of Long QT syndrome, or clinically significant ECG abnormality. * Significant history of alcohol or drug abuse, or positive alcohol breath test or urine drug screen at the Screening or Run-in visit. * Plans to implement new strenuous physical activity (e.g., significantly increased running pace/duration, high-intensity interval training, heavy weightlifting, vigorous cycling) during the study. * Impaired awareness of hypoglycemia. * Active hepatitis B or C infection, positive HIV serology, or any latent or active infection that would interfere with study procedures or be exacerbated by study medications. * Clinically significant abnormal Screening laboratory or other diagnostic findings (including hepatic, hematologic, thyroid, muscle-enzyme, or tuberculosis screening) rendering the participant unsuitable for the study. * Current or recent use of medications that could interfere with glucose metabolism or confound study assessments (e.g., glucocorticoids, systemic beta-blockers, growth hormone, or other antidiabetic medications \[e.g., glucagon-like peptide 1 (GLP-1) receptor agonists and/or sodium-glucose cotransport 2 (SGLT2) inhibitors\]). * Vaccination within 30 days prior to dosing or planned vaccination within 8 weeks post-dosing. * Known hypersensitivity to tocilizumab, or Screening laboratory findings indicating undue risk of a tocilizumab-related adverse event. * History of bariatric surgery within the 12 months prior to Screening. * History of trauma to, or other findings affecting the suitability of, the muscles intended for study intervention administration. * Participation in another investigational drug or biologic trial within 6 months prior to Screening, or participation in any previous gene therapy trial