VX-264 for Type 1 Diabetes

Recruiting at 15 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

7 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug VX-264 for Type 1 Diabetes?

Research on similar treatments, like GLP-1 receptor agonists such as exenatide and liraglutide, shows they can help reduce blood sugar levels, lower insulin doses, and decrease the risk of low blood sugar in people with Type 1 Diabetes. These drugs work by helping the body use insulin more effectively and reducing the release of glucagon, a hormone that raises blood sugar. ^{1 2 3 4 5}

What is the purpose of this trial?

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

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Empagliflozin as Adjunctive to Insulin Therapy in Type 1 Diabetes: The EASE Trials. [2022]

2. England 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. [2021]

3. United States 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. [2018]

4. Italy 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. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

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