What side effects are associated with this type of intervention?

Common treatments for Type 1 Diabetes, including advanced therapies like stem cell-derived treatments such as VX-880, may have side effects such as gastrointestinal symptoms, potential for adverse proliferation, and immune rejection. Conventional treatments like insulin therapy can cause hypoglycemia, weight gain, and injection site reactions. Continuous glucose monitoring systems may lead to skin irritation or sensor-related issues. Overall, while advanced therapies show promise in improving glycemic control and reducing insulin dependency, they require careful monitoring for potential adverse effects.

What prior FDA approvals exist?

Currently, there are no FDA-approved stem cell-derived therapies specifically for Type 1 Diabetes that aim to restore insulin production, similar to VX-880. The primary treatments for T1D include insulin therapy, which involves various insulin analogs to manage blood glucose levels. Advanced treatments under investigation, like VX-880, are still in clinical trial phases and have not yet received FDA approval.

What other types of research exist?

Promising alternatives to VX-880 for Type 1 Diabetes patients include: 1) Sensor-augmented pump (SAP) therapy, which combines insulin pumps with continuous glucose monitoring to improve glycemic control. 2) Hybrid closed-loop (HCL) systems, which automate insulin delivery based on real-time glucose readings. 3) Inhaled insulin, offering rapid absorption and improved post-prandial glucose control. 4) Islet cell transplantation, an emerging treatment that aims to restore insulin production, though it is limited by the availability of donor cells.

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Stem Cell Therapy

VX-880 for Type 1 Diabetes

Q: What is the mechanism of action of this treatment?

The most common treatments for Type 1 Diabetes (T1D) include insulin therapy, stem cell therapy, and investigational approaches like gene therapy. Insulin therapy involves the administration of exogenous insulin to manage blood glucose levels, as T1D patients lack endogenous insulin production. Stem cell therapy, such as the investigational VX-880, aims to restore insulin production by differentiating stem cells into insulin-producing pancreatic beta cells, potentially offering a more permanent solution. Gene therapy introduces modified genes to produce the missing insulin. These treatments are crucial for T1D patients as they address the fundamental issue of insulin deficiency, helping to maintain glycemic control and prevent complications.

Phase 1 & 2

Recruiting

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment

Clinical history of T1D with > 5 years of duration

Must not have

Prior islet cell transplant, organ transplant, or cell therapy

Timeline

Screening 3 days

Treatment Varies

Follow Up from vx-880 infusion to end of study (up to 5 years)

Awards & highlights

No Placebo-Only Group

Summary

This trial will test the safety and effectiveness of VX-880 infusion in people with Type 1 diabetes who have trouble sensing low blood sugar and experience severe low blood sugar episodes. The treatment aims to help manage their blood sugar levels better.

Who is the study for?

This trial is for people with Type 1 diabetes who've had it for over 5 years and have trouble sensing when their blood sugar gets too low. They should have had at least two serious low blood sugar events in the past year and must be using a continuous glucose monitor (CGM) for three months before, and during, the study.

What is being tested?

The trial is testing VX-880's safety and effectiveness. Participants will receive an infusion of VX-880 to see if it helps manage Type 1 diabetes, especially for those who often don't notice when their blood sugar drops dangerously low.

What are the potential side effects?

Possible side effects include reactions at the infusion site, changes in blood sugar levels that could lead to hypoglycemia or hyperglycemia, immune responses against the infused cells, or other unforeseen complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have had at least two severe low blood sugar episodes in the last year.

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Timeline

Screening ~ 3 days1 visit

Treatment ~ Varies

Follow Up ~ from vx-880 infusion to end of study (up to 5 years)

Screening ~ 3 days

Treatment ~ Varies

Follow Up ~from vx-880 infusion to end of study (up to 5 years)

This trial's timeline: 3 days for screening, Varies for treatment, and from vx-880 infusion to end of study (up to 5 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of Participants Free of Severe Hypoglycemic Events With Either a Glycosylated Hemoglobin (HbA1c) <7.0% or a ≥ 1% Reduction in HbA1c From Baseline

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary study objectives

Changes in Stimulated C-peptide

Proportion of Participants who are Insulin Independent

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VX-880Experimental Treatment1 Intervention

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Type 1 Diabetes (T1D) include insulin therapy, stem cell therapy, and investigational approaches like gene therapy. Insulin therapy involves the administration of exogenous insulin to manage blood glucose levels, as T1D patients lack endogenous insulin production. Stem cell therapy, such as the investigational VX-880, aims to restore insulin production by differentiating stem cells into insulin-producing pancreatic beta cells, potentially offering a more permanent solution. Gene therapy introduces modified genes to produce the missing insulin. These treatments are crucial for T1D patients as they address the fundamental issue of insulin deficiency, helping to maintain glycemic control and prevent complications.

Efficacy and Safety of Stem Cell Therapy for T1DM: An Updated Systematic Review and Meta-Analysis.Evaluating preclinical efficacy.