VX-880 for Type 1 Diabetes

Recruiting at 11 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Vertex Pharmaceuticals Incorporated

Must be taking: Immunosuppressants

Disqualifiers: Prior islet transplant, 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: 12 weeks

10 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 for individuals with Type 1 Diabetes who have undergone a kidney transplant. The study aims to evaluate the effectiveness, safety, and impact of VX-880 on those who regularly require insulin shots. It seeks participants who have managed their diabetes with insulin for at least five years and are on a stable medication plan to prevent organ rejection. Participants should consistently use a continuous glucose monitor, which tracks sugar levels in real-time. Those who have had more than one kidney transplant or certain other organ transplants may not qualify for this trial. As a Phase 3 trial, this study represents 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 requires that you continue taking a stable immunosuppression regimen, which includes medications like tacrolimus and mycophenolate mofetil, for at least 4 weeks before joining the study.

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

Research has shown that VX-880, a treatment for Type 1 Diabetes, is generally well-tolerated. Studies indicate that the safety profile of VX-880 aligns with expectations for drugs that aid in treatment acceptance and the administration process. This means the side effects resemble those of other immune system management treatments. Although specific safety numbers are not provided, this similarity to known treatments can be reassuring. Discussing any concerns with a healthcare provider is advisable to understand the potential risks and benefits. ^{1 2 3 4 5}

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

Unlike the standard treatments for Type 1 Diabetes, which often involve insulin therapy and glucose monitoring, VX-880 is exciting because it uses lab-grown islet cells to restore the body's ability to produce insulin naturally. The treatment targets the root cause of the disease by introducing functional islet cells, potentially reducing or even eliminating the need for regular insulin shots. Researchers are thrilled about VX-880's potential to offer a more sustainable and long-term solution by addressing the underlying problem rather than just managing symptoms.

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

Research shows that VX-880, also known as zimislecel, could be a promising treatment for Type 1 Diabetes. Studies have found that some people no longer needed insulin shots after receiving this treatment. VX-880 uses stem cells to create islet cells, enabling the body to produce insulin independently. Early results suggest that the benefits of VX-880 are consistent and long-lasting, with many patients experiencing significant improvement. Overall, evidence supports VX-880 as a potentially transformative option for managing Type 1 Diabetes. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for people with Type 1 Diabetes who have had a kidney transplant. Participants must have been insulin-dependent for at least 5 years, be on a stable immunosuppression regimen for at least 4 weeks, and consistently use a continuous glucose monitor (CGM) for the same duration.

Inclusion Criteria

I have been dependent on insulin for 5 years or more due to Type 1 Diabetes.

I've been on a steady dose of specific immunosuppressants for over 4 weeks.

I have been using a CGM for at least 4 weeks and am willing to continue using it.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

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

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 study tests VX-880's effectiveness, safety, and tolerability in individuals with Type 1 Diabetes who also had a kidney transplant. It aims to see how well VX-880 works as a biological/vaccine intervention in this specific patient group.

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. 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.

2. news.vrtx.com news.vrtx.com/news-releases/news-release-details/vertex-presents-positive-data-zimislecel-type-1-diabetes

Vertex Presents Positive Data for Zimislecel in Type 1 ... Results from the study continue to demonstrate the transformative potential of zimislecel with consistent and durable patient benefit –.

3. 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 ...

4. 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).

5. 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.

6. 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).

7. breakthrought1d.org breakthrought1d.org/news-and-updates/vertex-launches-pivotal-trial-for-stem-cell-derived-islet-therapy/

Vertex launches pivotal trial for stem-cell derived islet ... These data show that VX-880 can restore insulin production in people with type 1 diabetes. The primary endpoint for this study will be the ...