What side effects are associated with this type of intervention?

Common treatments for Type 1 Diabetes, particularly those involving islet transplantation and immunosuppressive agents like tacrolimus, can lead to side effects such as inflammation, oxidative stress, and graft loss due to toxicity. Tacrolimus is known for its diabetogenic effects and potential to impair graft function. Insulin therapy, another common treatment, can cause hypoglycemia and weight gain.

What prior FDA approvals exist?

The FDA has approved several treatments for Type 1 Diabetes, primarily focusing on insulin therapy and adjunctive treatments to improve glycemic control. However, specific treatments that offer cytoprotection against inflammation, oxidative stress, and Tac toxicity, similar to the investigational drug PKX-001, are not explicitly mentioned in the provided context. PKX-001 is unique in its approach to protecting islet cells during transplantation, addressing challenges like inflammation and oxidative stress, which are not typically the primary focus of standard Type 1 Diabetes treatments.

What other types of research exist?

Promising novel alternatives to Antiaging glycopeptide (PKX-001) for Type 1 Diabetes patients include the use of rapamycin and its derivatives, such as sirolimus, which have shown efficacy in reducing disease progression and protecting cells from oxidative stress and inflammation. Additionally, metformin has demonstrated potential in improving disease parameters and providing cytoprotection in various models of kidney disease, which may translate to benefits in Type 1 Diabetes management.

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PKX-001 for Type 1 Diabetes

Phase 1

Recruiting

Led By James Shapiro, MD, PhD

Research Sponsored by University of Alberta

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant must have had type 1 diabetes mellitus (T1DM) for more than 5 years

All control participants will be included according to the immunosuppression / engraftment regimen used in this pilot, specifically the current standard of care islet transplant at the University of Alberta Hospital: Alemtuzumab/Basiliximab, Anakinra, Etanercept, Mycophenolate Mofetil and Tacrolimus

Must not have

Clinical suspicion of nephritic or rapidly progressing renal impairment

Use of coumadin or other anticoagulant therapy (except aspirin) or patient with prothrombin time (PT) / international normalized ratio (INR) > 1.5

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year post-transplant

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While PKX-001 itself may not be given directly to participants as medication, potential side effects could relate to the transplantation process such as inflammation at the site of injection in the liver, immune reactions, or complications related to immunosuppressive therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have had type 1 diabetes for over 5 years.

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am suspected to have rapidly worsening kidney problems.

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

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

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

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

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I have Celiac disease that has not been treated.

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

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

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

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

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I have a serious heart condition.

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

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My cholesterol levels are very high and not under control.

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I am either younger than 18 or older than 68.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year post-transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse event morbidity

Serious adverse event morbidity

Secondary study objectives

Beta-2 score

Engraftment index

Glucose stimulated insulin release

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 17Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The primary treatment for Type 1 Diabetes (T1D) is insulin therapy, which replaces the insulin that the body can no longer produce. Advanced treatments like islet transplantation involve transplanting insulin-producing cells into the liver to help regulate blood glucose levels. However, these transplanted cells often face challenges such as inflammation, oxidative stress, and toxicity from immunosuppressive drugs like tacrolimus (Tac). PKX-001, an antiaging glycopeptide, is being studied for its potential to protect these islet cells from such harmful conditions, thereby improving their survival and function. This cytoprotective approach is crucial for T1D patients as it aims to enhance the longevity and efficacy of islet transplants, potentially reducing the need for frequent insulin injections and improving overall glucose control.

Potential of PKM2 as a drug target in mouse models with type 1 diabetes mellitus.The PKCβ-p66shc-NADPH oxidase pathway plays a crucial role in diabetic nephropathy.Protection of pancreatic β-cells against glucotoxicity by short-term treatment with GLP-1.