Gene therapy aims to free type 1 diabetics from insulin shots
NCT ID NCT07829302
First seen Sep 21, 2026 · Last updated Sep 21, 2026
Summary
Researchers are testing two experimental gene therapies, VV-14299 and VV-14300, in adults with long-standing type 1 diabetes whose blood sugar remains poorly controlled despite using an automated insulin pump. The therapies are injected into muscle and are designed to work together to help the body remove excess sugar from the blood. The study will first check safety and then look at whether the treatment improves blood sugar levels.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- an experimental gene therapy that helps muscle cells take up and store sugar from the blood
- What this could lead to
- If it works, this approach could one day free people with type 1 diabetes from daily insulin injections by giving their muscles a way to clear excess blood sugar.
- What could go wrong
- This is a first-in-human study in a small number of adults, so safety and effectiveness are still unknown. Gene therapy carries risks of immune reactions, and the effect may not last or may not work well enough to replace insulin.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 29 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Jul 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 (T1D) 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 (C-peptide \<0.20 ng/mL \[0.667 nmol/L\] during a mixed meal tolerance test; 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 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. * 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. * Documented anti-AAV1 neutralizing antibody titer above the protocol-specified threshold at Screening. * 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. * 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.
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Kriya Clinical Trial Site
Melbourne, Australia
Contact Email: •••••@•••••
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Other studies related to the condition(s) this trial covers.
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