VX-264 for Type 1 Diabetes

Not currently recruiting at 17 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Islet transplant, Organ transplant, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VX-264 to determine its safety and effectiveness for people with type 1 diabetes (T1D). The main goal is to assess how well VX-264 works and its impact on individuals with T1D. Suitable candidates have had T1D for at least five years, use continuous glucose monitoring (CGM) regularly, and maintain a stable diabetes treatment plan. Those with a history of islet cell, organ, or cell therapy transplants are not eligible. As a Phase 1, Phase 2 trial, this research aims to understand VX-264's function in people and measure its effectiveness in an initial group, offering a chance to contribute to groundbreaking diabetes treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it mentions that participants should be on a stable diabetic treatment. It seems likely that you can continue your current diabetes medications.

Is there any evidence suggesting that VX-264 is likely to be safe for humans?

Research has shown that VX-264 is generally safe and well tolerated in people with type 1 diabetes. An earlier study did not meet its effectiveness goals, but it raised no major safety concerns. Participants experienced increases in C-peptide, indicating insulin production, without major side effects. This suggests the treatment is well tolerated. However, as this trial is still in the early stages, further research is needed to confirm these findings. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about VX-264 for Type 1 Diabetes because it represents a potential new approach to managing the condition. Unlike the current standard treatments that often rely on insulin injections to control blood sugar levels, VX-264 is designed to target the underlying cause of the disease by preserving or restoring insulin-producing cells in the pancreas. This innovative mechanism could mean less reliance on constant glucose monitoring and insulin administration, offering a more sustainable and potentially transformative solution for patients.

What evidence suggests that VX-264 might be an effective treatment for type 1 diabetes?

Research has shown that VX-264, the treatment under study in this trial, has not been very effective in treating type 1 diabetes in previous studies. These studies did not achieve their goals, meaning the treatment did not work as expected. Although some increases in C-peptide levels, which indicate insulin production, were observed, these results were not strong enough. The clinical trial for VX-264 was even stopped because it wasn't giving the desired results. Overall, the evidence suggests that VX-264 has not been successful in managing type 1 diabetes symptoms. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for people with Type 1 Diabetes who have been managing their condition for over 5 years and are on a stable treatment plan. Participants must be using continuous glucose monitoring (CGM) consistently for at least a month before the study starts and agree to use it throughout the trial. Those who've had an islet cell or organ transplant, or cell therapy cannot join.

Inclusion Criteria

My diabetes treatment has not changed recently.

I have had Type 1 Diabetes for 5 years or more.

Consistent use of continuous glucose monitoring (CGM) for at least 4 weeks before Screening and willingness to use CGM for the duration of the study

Exclusion Criteria

I have had an islet cell, organ transplant, or cell therapy before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VX-264 to evaluate safety, tolerability, and efficacy

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VX-264

Trial Overview The study tests VX-264's safety, how well participants can tolerate it, and its effectiveness in individuals with Type 1 Diabetes. The main goal is to see if this new intervention could potentially improve diabetes management.

How Is the Trial Designed?

Group I: VX-264 Experimental Treatment 1 Intervention

VX-264 is already approved in United States, Canada for the following indications:

Approved in United States as VX-264 for:

• Type 1 Diabetes (under investigation)

Approved in Canada as VX-264 for:

• Type 1 Diabetes (under investigation)

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead 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

Published Research Related to This Trial

Empagliflozin, at doses of 10 mg and 25 mg, significantly improved glycemic control and reduced weight in patients with type 1 diabetes without increasing the risk of severe hypoglycemia, based on a study involving 1,707 participants across two phase 3 trials.

The 2.5 mg dose of empagliflozin showed a comparable rate of diabetic ketoacidosis to placebo, while higher doses (10 mg and 25 mg) had increased rates, suggesting that lower doses may be safer and effective for managing type 1 diabetes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Empagliflozin as Adjunctive to Insulin Therapy in Type 1 Diabetes: The EASE Trials. Rosenstock, J., Marquard, J., Laffel, LM., et al.[2022]

In a study of 30 patients with type 1 diabetes, adding exenatide to insulin treatment significantly reduced body weight, insulin dosage, and glycemic variability over 4 weeks, indicating improved glycemic control.

Exenatide effectively inhibited glucagon secretion during normal blood sugar levels but did not affect counter-regulatory hormones during hypoglycemia in patients without functional residual islet cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effects of add-on exenatide to insulin on glycemic variability and hypoglycemia in patients with type 1 diabetes mellitus. Jiang, LL., Wang, SQ., Ding, B., et al.[2022]

In a study of 40 normal-weight patients with poorly controlled type 1 diabetes, liraglutide 1.2 mg did not significantly lower HbA1c compared to placebo, indicating it may not provide additional glycemic control when added to insulin treatment.

However, liraglutide led to a significant reduction in body weight and insulin requirements, suggesting it could be beneficial for managing weight and insulin dosage in these patients, despite an increase in gastrointestinal side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Twelve-Week Treatment With Liraglutide as Add-on to Insulin in Normal-Weight Patients With Poorly Controlled Type 1 Diabetes: A Randomized, Placebo-Controlled, Double-Blind Parallel Study. Frandsen, CS., Dejgaard, TF., Holst, JJ., et al.[2018]

GLP-1 receptor agonists (like exenatide and liraglutide) can significantly lower hemoglobin A1C levels, plasma glucose, and body weight in patients with type 1 diabetes who are already on insulin, without increasing the risk of hypoglycemia.

The most common side effects of GLP-1 RAs are mild and transient gastrointestinal issues, making them a safe option for patients struggling with insulin side effects or those needing to lose weight.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adjunctive Role of Glucagon-Like Peptide-1 Receptor Agonists in the Management of Type 1 Diabetes Mellitus. Harris, KB., Boland, CL.[2022]

The dual-functional peptide PGLP-1-VP has been shown to improve hyperglycemia and increase insulin sensitivity in a mouse model, indicating its potential as an effective treatment for type 1 diabetes.

In the NOD mouse model, PGLP-1-VP significantly reduced morbidity and mortality while also decreasing pancreatic inflammation, outperforming treatments with PGLP-1 or VP alone, suggesting its dual anti-inflammatory and immunomodulatory effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Continuous stimulation of dual-function peptide PGLP-1-VP inhibits the morbidity and mortality of NOD mice through anti-inflammation and immunoregulation. Gao, H., Zhao, Q., Tang, S., et al.[2021]

Citations

1. investors.vrtx.com investors.vrtx.com/news-releases/news-release-details/vertex-announces-program-updates-type-1-diabetes-portfolio

Vertex Announces Program Updates for Type 1 Diabetes ... VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint. Increases in C-peptide, a marker of insulin ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05791201

A Safety, Tolerability, and Efficacy Study of VX-264 in ... The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).

3. type1strong.org type1strong.org/blog-post/vertexs-vx-264-clinical-trial-fails-to-produce-results-ends-abruptly

Vertex's VX-264 Clinical Trial Fails to Produce Results & ... Vertex Pharmaceuticals announced in a press release that its VX-264 clinical trial had been discontinued because it wasn't producing significant results.

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-vx-264-for-patients-with-type-1-diabetes/

Study on the Safety and Effects of VX-264 for Patients with ... The purpose of this study is to evaluate the safety, tolerability, and effectiveness of the VX-264 Implant in people with Type 1 Diabetes.

5. beyondtype1.org beyondtype1.org/vx-264-a-diabetes-breakthrough-that-didnt-work-as-planned/

VX-264: A Diabetes Breakthrough That Didn't Work as ... Vertex's VX-264 didn't work out, but zimislecel and UP421 show promise in type 1 diabetes treatment. Learn how these therapies could change ...

6. diatribe.org diatribe.org/diabetes-research/vertex-releases-new-data-potential-cure-type-1-diabetes

Vertex Releases New Data on Potential Cure for Type 1 ... Key takeaways: Zimislecel (VX-880) is an investigational islet cell therapy by Vertex that shows promise as a functional cure for type 1 diabetes.

7. cgtlive.com cgtlive.com/view/vertex-doses-first-patient-phase-1-2-trial-vx-264-type-1-diabetes

Vertex Doses First Patient in Phase 1/2 Trial of VX-264 ... Vertex Pharmaceuticals has announced amid its quarterly financial results that it has dosed the first patient in its newest cell therapy program.