VX-880 for Type 1 Diabetes

Recruiting at 30 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Travel: May Be Covered

Trial Phase: Phase 3

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Prior transplants, cell therapy, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

52 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VX-880 (also known as STx-02) for individuals with Type 1 diabetes who struggle to sense low blood sugar and frequently experience severe episodes. The main goal is to assess the safety and effectiveness of VX-880 in managing these issues. Suitable candidates have relied on insulin for over five years, experienced at least two severe low blood sugar events in the past year, and regularly use a continuous glucose monitor. As a Phase 3 trial, this is the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that VX-880 is likely to be safe for humans?

Research has shown that VX-880 has been promising in earlier studies. By Day 90 after receiving VX-880, all patients experienced positive results. The cells integrated well into the body and produced insulin in response to sugar. This suggests that VX-880 is well-tolerated, functioning as expected without major issues.

Although specific safety details were not highlighted, the study's progression to a later phase indicates that the treatment has passed several safety checks. This phase typically suggests the treatment is safe enough for testing in more people.

For those considering joining the trial, this information may provide reassurance about the treatment's safety so far. However, discussing all details and concerns with a healthcare provider remains essential. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Type 1 diabetes?

VX-880 is unique because it's a cell therapy aimed at treating Type 1 diabetes by replacing the insulin-producing cells that the body has lost. Unlike traditional treatments that focus on insulin injections or pumps to manage blood sugar levels, VX-880 directly addresses the root cause by using stem cell-derived islet cells to restore the body's ability to produce insulin naturally. Researchers are excited about this approach because it has the potential to offer a long-term solution, potentially reducing or even eliminating the need for frequent insulin administration.

What evidence suggests that VX-880 might be an effective treatment for Type 1 Diabetes?

Research has shown that VX-880, the treatment under study in this trial, could help manage Type 1 diabetes by aiding the body in controlling blood sugar levels. In studies, some patients no longer needed insulin shots. Specifically, out of 12 people who received full doses, 3 stopped needing insulin, and 7 more are expected to follow. VX-880 restores the function of pancreatic cells that regulate blood sugar. Overall, this treatment has the potential to reduce or even eliminate the need for insulin. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening and willingness to use CGM for the duration of the study

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

I have been using a CGM for 3 months and am willing to continue.

Exclusion Criteria

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

There may be other requirements to participate in the study that are not listed here.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VX-880 infusion to evaluate safety, tolerability, and efficacy

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VX-880

Trial Overview 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.

How Is the Trial Designed?

Group I: VX-880 Experimental Treatment 1 Intervention

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

Citations

1. investors.vrtx.com investors.vrtx.com/news-releases/news-release-details/vertex-announces-positive-results-ongoing-phase-12-study-vx-880

Vertex Announces Positive Results From Ongoing Phase 1 ... VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04786262

A Safety, Tolerability, and Efficacy Study of VX-880 in ... This study will evaluate the safety, tolerability and efficacy of VX-880 infusion in participants with Type 1 diabetes (T1D) and impaired awareness of ...

3. thejdca.org thejdca.org/publications/report-library/archived-reports/2024-reports/vx-880-update-3-insulin-independent-with-7-expected-to-follow.html

VX-880 Update: 3 Insulin Independent with 7 Expected to ... 12 people received full-dosing in Vertex's T1D trial, VX-880. 3 individuals were declared insulin independent (from a clinical standpoint). 7 ...

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa2506549

Stem Cell–Derived, Fully Differentiated Islets for Type 1 ... Zimislecel is an allogeneic stem cell–derived islet-cell therapy. Data on the safety and efficacy of zimislecel in persons with type 1 diabetes are needed.

5. diabetes.org diabetes.org/newsroom/press-releases/expanded-forward-trial-demonstrates-continued-potential-stem-cell-derived

Expanded FORWARD Trial Demonstrates Continued ... The results demonstrated that VX-880 reduced or eliminated the need for insulin use in patients with type 1 diabetes (T1D).

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04786262

A Safety, Tolerability, and Efficacy Study of VX-880 in ... This study will evaluate the safety, tolerability and efficacy of VX-880 infusion in participants with Type 1 diabetes (T1D) and impaired awareness of ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40544428/

Stem Cell-Derived, Fully Differentiated Islets for Type 1 ... Zimislecel is an allogeneic stem cell-derived islet-cell therapy. Data on the safety and efficacy of zimislecel in persons with type 1 diabetes are needed.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT06832410

An Efficacy, Safety, and Tolerability Study of VX-880 in ... This study will evaluate the efficacy, safety, and tolerability of VX-880 in participants with Type 1 Diabetes (TID) with a kidney transplant. Official Title. A ...