PKX-001 for Type 1 Diabetes

Palo Alto (17 mi)

Overseen byJames Shapiro, MD, PhD

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: University of Alberta

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests a new drug, PKX-001, to help insulin-producing cells survive and function better when transplanted into people with hard-to-control Type 1 Diabetes. The drug protects the cells from damage and helps them produce insulin. The study aims to confirm the safety and effectiveness of this approach.

Eligibility Criteria

This trial is for people over 18 and under 68 with Type 1 Diabetes who've had it for more than 5 years, have trouble sensing low blood sugar or unstable blood sugar levels despite using insulin. They must understand the study's risks, agree to not get pregnant or father a child, and can't have certain diseases like uncontrolled thyroid issues or infections.

Inclusion Criteria

I have had type 1 diabetes for over 5 years.

I am following the specific immunosuppression regimen for islet transplant at the University of Alberta Hospital.

I often don't notice when my blood sugar is too low, despite using insulin carefully.

Exclusion Criteria

I am suspected to have rapidly worsening kidney problems.

I am taking blood thinners other than aspirin, or my blood clotting time is longer than normal.

I do not have an active infection like Hepatitis C, B, HIV, or TB.

My hemoglobin level is below the normal range for my gender.

I have an untreated eye condition where new blood vessels grow abnormally.

I have Celiac disease that has not been treated.

My kidney function is reduced with a GFR less than 60.

I am on long-term steroids for another health issue.

I need more than 1 unit of insulin per kilogram of my body weight daily.

I have had cancer before, but it was not skin cancer.

I have a serious heart condition.

I do not have a psychiatric condition that would prevent me from undergoing transplantation.

My cholesterol levels are very high and not under control.

I am either younger than 18 or older than 68.

Treatment Details

The trial tests if PKX-001 treated islet cells (insulin-producing cells) from donors can survive better when transplanted into patients' livers. This drug mimics antifreeze proteins to protect cells during transplant against damage from immune system drugs that prevent rejection.

1Treatment groups

Experimental Treatment

Group I: Treatment GroupExperimental Treatment1 Intervention

PKX-001 will be supplemented to islet preservation CMRL-1066 medium at final concentration of 3 mg/mL during islet isolation process. On the day of transplantation, preserved islets supplemented with PKX-001 are collected and washed with Transplant Media, which does not contain PKX-001, as a standard procedure. The isolation team will evaluate the final islet product based on standard assays. Islets are maintained for minimal 6 hours up to 72 hours in supplemented CMRL1066-based media containing PKX-001 until the time of transplant. When product release minimal criteria are met, islets will be clinically transplanted into patients intraportally.