VC-02 for Type 1 Diabetes

No longer recruiting at 10 trial locations

Overseen By Corporate Communications

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: ViaCyte

Disqualifiers: Transplants, Hypoglycemic events, Thyroid disease, Diabetic complications, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new cell replacement therapy called VC-02, which aims to offer a functional cure for people with Type 1 Diabetes who struggle with hypoglycemia unawareness (the inability to sense low blood sugar). The treatment involves implanting small units that help the body manage blood sugar levels. The trial includes two groups testing different combinations of these implants. Ideal participants have had Type 1 Diabetes for at least five years, experience frequent blood sugar swings, and are willing to use a continuous glucose monitor. As a Phase 1/Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Do I need to stop my current medications for the trial?

The trial protocol does not specify if you need to stop your current medications. However, it mentions that participants should have a stable diabetic treatment, which might mean you can continue your current regimen.

Is there any evidence suggesting that the VC-02 Combination Product is likely to be safe for humans?

Research has shown that the VC-02 product has been tested for safety in people with Type 1 Diabetes. This product includes implants designed to replace insulin-producing cells. Earlier studies with these implants found that they can be placed under the skin and are generally well-tolerated.

In one study with 17 participants, the implants did not cause major issues, indicating they are safe to use. Participants did not experience serious side effects, which is a positive sign of safety. Since these studies have been conducted with people and the treatment has progressed to this stage, it suggests that the treatment is safe enough for further testing.

As always, those considering joining a trial should consult a healthcare professional. They can help explain how this treatment might work for individual needs. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard insulin therapies and continuous glucose monitoring systems used for Type 1 Diabetes, the VC-02 Combination Product is unique because it involves implantable devices that release insulin-producing cells directly into the body. This approach aims to mimic the body’s natural insulin production more closely, potentially reducing the need for frequent insulin injections and constant blood sugar monitoring. Researchers are excited about VC-02 because it offers a new method of delivering treatment that could significantly improve blood sugar control and quality of life for patients.

What evidence suggests that VC-02 could be an effective treatment for Type 1 Diabetes?

Research shows that the VC-02 Combination Product could be promising for treating Type 1 Diabetes. In a study with 17 people who have this condition, implanted cells made from stem cells helped produce insulin and C-peptide, indicating insulin production. This suggests the treatment might enable the body to produce its own insulin. In this trial, participants will join different cohorts receiving varying configurations of the VC-02 implants. The cells used in VC-02 also demonstrated the ability to react to sugar levels in the body. These results suggest that VC-02 might help control blood sugar levels and reduce the need for insulin injections. ^{1 3 4 5 6}

Who Is on the Research Team?

Gautham Marigowda

Principal Investigator

Vice President, Clinical Development, Vertex

Are You a Good Fit for This Trial?

Inclusion Criteria

You have been diagnosed with type 1 diabetes for at least five years.

You are physically suitable for a surgical implantation.

Only men and women who are not pregnant can participate.

Exclusion Criteria

Detectable stimulated serum C-peptide during screening period assessment.

You don't follow your current diabetes treatment plan.

Six (6) or more severe, unexplained hypoglycemic events within six (6) months of enrollment

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the VC-02 combination product implants and are monitored for safety and efficacy

26 weeks

Regular visits for monitoring and assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

• VC-02 Combination Product

How Is the Trial Designed?

Group I: Cohort 2 Experimental Treatment 1 Intervention

VC-02 Combination Product; Up to twelve units implanted of which up to ten (10) are VC-02-300 implants and the rest are VC-02-20 implants.

Group II: Cohort 1 Experimental Treatment 1 Intervention

VC-02 Combination Product: Up to ten (10) VC-02-20 implants and up to two (2) VC-02-300 implants

Who Is Running the Clinical Trial?

ViaCyte

Lead Sponsor

Trials

7

Recruited

200+

Vertex Pharmaceuticals Incorporated

Industry Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

California Institute for Regenerative Medicine (CIRM)

Collaborator

Trials

70

Recruited

3,300+

Horizon 2020 - European Commission

Collaborator

Trials

35

Recruited

14,200+

Published Research Related to This Trial

In a 52-week study involving 1,398 adults with type 1 diabetes, adding liraglutide to insulin therapy significantly reduced HbA1c levels and insulin requirements, with reductions in body weight observed across all liraglutide doses compared to placebo.

However, the addition of liraglutide was associated with increased rates of symptomatic hypoglycemia and a significant rise in hyperglycemia with ketosis for the highest dose, which may limit its clinical use in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Liraglutide Added to Insulin Treatment in Type 1 Diabetes: The ADJUNCT ONE Treat-To-Target Randomized Trial. Mathieu, C., Zinman, B., Hemmingsson, JU., et al.[2022]

In the Phase III DEFEND-2 trial involving 179 patients (including 54 adolescents), otelixizumab did not significantly preserve C-peptide secretion in patients with new-onset Type 1 diabetes, showing a change from baseline that was not statistically significant (P=0.051).

The study found that the efficacy and tolerability of otelixizumab at a 3.1 mg dose were similar to previous results from DEFEND-1, indicating that this dose was ineffective for both adults and adolescents, and further research is needed to explore higher doses and their mechanisms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of low-dose otelixizumab anti-CD3 monoclonal antibody in preserving C-peptide secretion in adolescent type 1 diabetes: DEFEND-2, a randomized, placebo-controlled, double-blind, multi-centre study. Ambery, P., Donner, TW., Biswas, N., et al.[2018]

In a study of 50 children and adolescents with poorly controlled type 1 diabetes, switching to the IDegAsp insulin co-formulation led to a significant reduction in self-reported mild to moderate hypoglycemic episodes after one year.

While the overall hemoglobin A1c levels remained unchanged, the frequency of diabetic ketoacidosis (DKA) attacks decreased from 11 to 4, suggesting that IDegAsp may enhance clinical management for patients struggling with frequent hypoglycemia and DKA.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of the Novel Degludec/Aspart Insulin Co-formulation in Children and Adolescents with Type 1 Diabetes: A Real-life Experience with One Year of IDegAsp Therapy in Poorly Controlled and Non-compliant Patients Kırkgöz, T., Eltan, M., Kaygusuz, SB., et al.[2022]

Citations

1. beyondtype1.org beyondtype1.org/trial-pec-direct/

A Safety, Tolerability, and Efficacy Study of VC-02 ... Learn about the phase 1 + 2 clinical trial by ViaCyte testing its VC-02 device implant for safety and effectiveness over two years.

2. ctv.veeva.com ctv.veeva.com/study/a-safety-tolerability-and-efficacy-study-of-vc-02-combination-product-in-subjects-with-type-1-dia

A Safety, Tolerability, and Efficacy Study of VC-02 ... The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and ...

3. nature.com nature.com/articles/s41587-023-02055-5

Encapsulated stem cell–derived β cells exert glucose ... This trial is a first-in-human, phase 1/2, open-label study on safety, tolerability and efficacy of VC-02 in patients with T1D and hypoglycemia ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03163511

NCT03163511 | A Safety, Tolerability, and Efficacy Study of ... The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and Hypoglycemia Unawareness ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8714853/

Insulin expression and C-peptide in type 1 diabetes subjects ... In this study of 17 subjects aged 22-57 with type 1 diabetes, PEC-01 cells were implanted subcutaneously in VC-02 macroencapsulation devices.

6. hpscreg.eu hpscreg.eu/browse/trial/5

A Safety and Tolerability Study of VC-02™ Combination ... The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for up ...