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

Trial Summary

What is the purpose of this trial?

This trial tests a new treatment for people with Type 1 Diabetes using special insulin-making cells and a delivery device. The cells are edited to avoid immune attacks, and the device helps place them in the body.

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.

What data supports the effectiveness of the treatment VCTX211 for Type 1 Diabetes?

Islet transplantation, which is similar to the VCTX211 treatment, has shown promise in treating Type 1 Diabetes by reducing the need for insulin and improving blood sugar control. Studies have shown that islet function was maintained in 60% of recipients after 12 months, and stem cell therapy, which may be related, has been effective in increasing C-peptide levels and reducing insulin requirements. ^{1 2 3 4 5}

Research Team

Manasi Jaiman, MD, MPH

Principal Investigator

ViaCyte

Sandeep Soni, MD

Principal Investigator

CRISPR Therapeutics

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

References

1. Greece pubmed.ncbi.nlm.nih.gov

Stem cell transplantation for the treatment of patients with type 1 diabetes mellitus: A meta-analysis. [2020]

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

Pancreas and Islet Transplantation: Comparative Outcome Analysis of a Single-centre Cohort Over 20-years. [2023]

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

Improvement in outcomes of clinical islet transplantation: 1999-2010. [2022]

4. Singapore 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. [2021]

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

NIH-Supported National Islet Transplantation Registry. [2019]