Safety, Tolerability and Efficacy of Immunomodulation With AT-1501 in Islet Cell Transplantation

Anelixis Therapeutics, LLC

Overview

This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant.

This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant. This study will also provide valuable data with respect to its potential additional uses in autoimmunity and solid organ transplant. This is a single arm open-label study and up to 6 participants will be recruited at a single center in Canada. The objectives include: * To assess the safety and tolerability of immunomodulation with AT-1501, in combination (AT+) with rabbit anti-thymoglobulin (ATG), etanercept and mycophenolate mofetil (MMF/EC-MPS) in adults with T1D undergoing islet cell transplant. * To assess the efficacy of immunomodulation with AT-1501 in adults with T1D undergoing islet cell transplant. The duration of treatment may vary from participant to participant and could be up to 20 months. Participants may receive up to 2 islet cell transplants.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Type 1 Diabetes Mellitus

Interventions

AT-1501DRUG

AT-1501 IV infusion

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Men and women 18-65 years of age 2. A diagnosis of T1D ≥5 years with onset of disease at \<40 years of age 3. Involvement in intensive diabetes management as directed by an endocrinologist or diabetologist with at least 3 clinical evaluations within the 12 months prior to Screening; using an insulin pump or multiple daily injection (MDI) insulin therapy; and, unable to achieve acceptable metabolic control because of the occurrence of severe hypoglycemia 4. At least 2 unexplained SHEs not secondary to a missed meal or dosing error, etc., in the 12 months prior to Screening 5. Glycosylated hemoglobin (HbA1c) level greater than 7% (53 mmol/mol) and less than 9.5% (80 mmol/mol) inclusive 6. Absence of stimulated C peptide (\< 0.3 ng/mL) in response to a mixed meal tolerance test (MMTT) measured at 60 and 90 minutes after the start of consumption 7. Reduced awareness of hypoglycemia as defined by a Clarke Score \[Clarke 1995\] of 4 or more at the time of Screening, during the Screening period and within the last 6 months prior to the transplant Exclusion Criteria: 1. Any previous transplant 2. HbA1c level less than 7% (53 mmol/mol)

Locations (1)

University of Alberta

Edmonton, Alberta, Canada

Outcomes

Primary Outcomes

Safety- Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)

Incidence of adverse events

Time frame: Accessed from date of transplant through 1 year post transplant for approximately 2 years

Efficacy- Insulin independence

Change in the proportion of participants that become insulin independent at Days 75 and 365 post-first, and final transplant

Time frame: Days 75 , Day 365 post-first transplant, and final transplant and 1 year after discontinuation of AT-1501

Secondary Outcomes

Efficacy- Graft failure

Proportion of participants with graft failure

Time frame: Day 365

Efficacy- Durability of insulin independence- long term

Change in the proportion of participants that become insulin independent at year 2 and year 3

Time frame: 2 and 3 years after discontinuation of AT- 1501

Efficacy- HbA1c

* Proportion of participants with HbA1c \<7.0% (53 mmol/mol) and free of serious hypoglycemic events (SHEs) * Proportion of participants with HbA1c ≤6.5% (48 mmol/mol) and free from SHEs

Time frame: Day 365 and free of serious hypoglycemic events from Day 28 to 365 post-first transplant

Data from ClinicalTrials.gov

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