VCTX211 for Type 1 Diabetes

Recruiting at 1 trial location

Overseen By Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: CRISPR Therapeutics AG

Must not be taking: Immunosuppressants

Disqualifiers: Transplant history, Hypoglycemic events, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called VCTX211 for individuals with Type 1 Diabetes (T1D). Researchers aim to determine if this treatment is safe and effective in managing T1D. Individuals who have had T1D for at least 5 years and maintained a stable diabetes routine for 3 months might be suitable candidates. However, the trial excludes those who have recently experienced severe low blood sugar episodes or have undergone organ transplants related to diabetes. As a Phase 1, Phase 2 trial, this research focuses on understanding how VCTX211 works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have a stable diabetes regimen for at least 3 months before joining. You cannot be on immunosuppressant therapy 30 days before or during the study.

Is there any evidence suggesting that VCTX211 is likely to be safe for humans?

Research shows that VCTX211 might be safe for humans. In earlier studies, initial results indicated that the treatment was safe and well tolerated, especially at the lowest dose. Patients did not experience serious side effects when taking small amounts of the treatment.

Additionally, safety has improved over time. For example, the need for extra procedures, like reintroducing insulin-producing cells, has decreased. This suggests that VCTX211 is safer now compared to previous years.

However, since VCTX211 remains in the early testing stages, not all possible side effects are known yet. Prospective trial participants can use this information to understand the current safety of VCTX211. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about VCTX211 for Type 1 Diabetes because it represents a novel approach to managing the condition. Unlike existing treatments that primarily focus on insulin replacement or modulation of the immune system, VCTX211 aims to address the root cause by using stem cell-derived islet cells to restore the body's natural insulin production. This innovative method has the potential to provide a more sustainable and long-term solution for patients, reducing or even eliminating the need for daily insulin injections. If successful, VCTX211 could revolutionize the way Type 1 Diabetes is treated, offering hope for improved quality of life.

What evidence suggests that VCTX211 might be an effective treatment for Type 1 Diabetes?

Research shows that VCTX211, the treatment under study in this trial, is being developed to help people with Type 1 Diabetes by supporting the immune system and reducing inflammation. This treatment aims to protect the remaining beta cells in the pancreas, which are crucial for insulin production. Early results suggest that VCTX211 might help maintain the health of these cells, potentially improving blood sugar control. Although still in early testing stages, the treatment uses CRISPR technology and other promising methods to address Type 1 Diabetes. Overall, VCTX211 represents an innovative approach to better manage the disease. ^{1 3 5 6 7}

Who Is on the Research Team?

Manasi Jaiman, MD, MPH

Principal Investigator

ViaCyte

Sandeep Soni, MD

Principal Investigator

CRISPR Therapeutics

Are You a Good Fit for This Trial?

This trial is for people who have had Type 1 Diabetes (T1D) for at least 5 years and are on a stable diabetes treatment plan. They can't join if they've used immunosuppressants recently, had certain organ transplants, other types of diabetes, gene therapy treatments, or frequent severe unexplained drops in blood sugar.

Inclusion Criteria

My diabetes treatment has been the same for the last 3 months.

I have been diagnosed with Type 1 Diabetes for at least 5 years.

Exclusion Criteria

I have had gene therapy or a gene-edited treatment before.

My diabetes is not caused by Type 1 diabetes.

I haven't taken immunosuppressants in the last 30 days and don't need them during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the VCTX211 combination product, which includes genetically modified pancreatic endoderm cells and a delivery device

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VCTX211

Trial Overview The study is testing VCTX211, a new combination product to see how safe and effective it is for treating T1D. It's an early-stage trial where everyone gets the same treatment without any placebos or comparison groups.

How Is the Trial Designed?

Group I: VCTX211 unit Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

CRISPR Therapeutics AG

Lead Sponsor

Trials

9

Recruited

640+

ViaCyte

Industry Sponsor

Trials

7

Recruited

200+

Published Research Related to This Trial

In a study of 412 adults undergoing islet (ITx) and pancreas (PTx) transplantation, PTx recipients experienced higher rates of insulin independence (92.5%) compared to ITx recipients (78.6%), indicating better overall graft function for PTx.

However, PTx was associated with higher mortality rates, procedure-related complications, and hospital readmissions compared to ITx, suggesting that while PTx offers better insulin independence and glycemic control, it also carries greater risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pancreas and Islet Transplantation: Comparative Outcome Analysis of a Single-centre Cohort Over 20-years. Marfil-Garza, BA., Hefler, J., Verhoeff, K., et al.[2023]

Islet transplantation outcomes for type 1 diabetes have improved over time, with insulin independence rates increasing from 27% in 1999-2002 to 44% in 2007-2010, indicating enhanced efficacy of the procedure.

Safety outcomes also improved, as evidenced by a lower rate of islet reinfusion (48% in 2007-2010 compared to 60-65% in earlier years) and a reduction in adverse events, suggesting a more favorable risk profile for recent transplant recipients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improvement in outcomes of clinical islet transplantation: 1999-2010. Barton, FB., Rickels, MR., Alejandro, R., et al.[2022]

In the Czech Republic, simultaneous pancreas and kidney transplantation (SPKTx) shows strong patient survival rates of 92% at 1 year and 81% at 5 years, with pancreas graft survival rates of 78% and 66% over the same periods, indicating its effectiveness for type 1 diabetes patients with kidney issues.

Islet transplantation (ITx) is emerging as a complementary treatment, with 60% of recipients showing islet function after 12 months, although only 3 patients were completely free of insulin, highlighting challenges in organ availability and islet viability.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The IKEM pancreas and islet transplant program as part of healthcare for type 1 diabetes patients: retrospective analysis of outcome from 1983 to 2010. Girman, P., Saudek, F.[2021]

Stem cell therapy significantly increased C-peptide levels and reduced glycated hemoglobin levels in patients with type 1 diabetes mellitus (T1DM), indicating improved insulin production and blood sugar control based on a meta-analysis of 22 studies.

The most effective outcomes were observed in patients aged 18 and older or those with a medical history of less than 3 months, particularly when higher doses of stem cells (≥107 IU/kg/day) were used, suggesting that patient characteristics and treatment protocols can influence the efficacy of stem cell transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem cell transplantation for the treatment of patients with type 1 diabetes mellitus: A meta-analysis. Gan, J., Wang, Y., Zhou, X.[2020]

Islet transplantation is a promising alternative treatment for type 1 diabetes, which affects an estimated 300,000 to 500,000 people in the U.S., potentially improving health outcomes compared to traditional insulin therapy.

The North American Collaborative Islet Transplant Registry (CITR) is collecting and analyzing data from islet transplants across North America to enhance the safety and efficacy of these procedures, with participation from over 22 transplant centers to identify key factors influencing patient and graft survival.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

NIH-Supported National Islet Transplantation Registry. Close, N., Anand, R., Hering, B., et al.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05210530

NCT05210530 | An Open-Label, FIH Study Evaluating the ... A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11663981/

Advancing type 1 diabetes therapy: autologous islet ... CRISPR Therapeutics are currently involved in an open-label, phase 1/2 study evaluating the efficacy of VCTX211 in people living with T1D ( ...

3. synthego.com synthego.com/blog/crispr-clinical-trials

CRISPR Clinical Trials to Follow CTX211 (VCTX210A)​​ The phase I/II CRISPR clinical trial of safety and efficacy expects to test the therapy in up to 40 patients and is estimated ...

4. thejdca.org thejdca.org/publications/report-library/archived-reports/2024-reports/2024-t1d-practical-cure-projects-in-human-trials.html

2024 T1D Practical Cure Projects in Human Trials The drug was given orally to promote sustained immune regulation, reduce inflammation, and support residual beta cell mass.

5. crisprmedicinenews.com crisprmedicinenews.com/clinical-trial/type-1-diabetes-t1d-nct05565248/

Clinical trial: Type 1 Diabetes, T1D, (NCT05565248) This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D.

6. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/377782

An Open-Label, FIH Study Evaluating the Safety, ... This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in ...

7. withpower.com withpower.com/trial/phase-2-immune-system-diseases-10-2022-bd2b8

VCTX211 for Type 1 Diabetes Safety outcomes also improved, as evidenced by a lower rate of islet reinfusion (48% in 2007-2010 compared to 60-65% in earlier years) and a reduction in ...