A Safety, Tolerability and Efficacy Study of Sernova's Cell Pouch™ for Clinical Islet Transplantation

Summary

The Cell Pouch™ is a novel implantable device, that is transplanted with therapeutic cells such as insulin producing islets. This combination product is designed for the treatment of Type 1 Diabetes Mellitus (T1D) with hypoglycemia unawareness and a history of severe hypoglycemic episodes. Upon implantation, the Cell Pouch is designed to form a natural environment, rich in tissue and microvessels for the transplant and function of therapeutic cells. The Cell Pouch is designed as a scaffold made of non-degradable polymers, formed into small cylindrical chambers which, when implanted against the abdominal muscle, becomes incorporated with vascularized tissue to the circumference of removable plugs within as early as two weeks as demonstrated in preclinical studies. After the tissue incorporation, the plugs are removed, leaving fully formed tissue chambers with central void spaces for the transplantation of therapeutic cells including Islets of Langerhans (islets). Tissue integration within and around the Cell Pouch forms a natural environment, rich in microvessels that allows the transplanted islets to engraft, resulting in a functional biohybrid organ. It is believed this engraftment will enable long-term survival and function of transplanted islets. This study aims to demonstrate the safety and tolerability of islet transplantation into the Cell Pouch for the treatment of T1D in subjects with hypoglycemia unawareness and a history of severe hypoglycemic episodes. The study also aims to establish islet release criteria that accurately characterize the islet product and are predictive of clinical transplant outcomes into the Cell Pouch, which will be demonstrated through defined efficacy measures.

Conditions

• Type 1 Diabetes Mellitus

Interventions

COMBINATION_PRODUCT

Sernova Cell Pouch

The Sernova Cell Pouch will be implanted against the rectus abdominis. The patient will receive either an 8-plug or 10-plug Cell Pouch configuration depending on Cohort assignment. A minimum of three weeks after Cell Pouch implantation, immunosuppression will be initiated and optimized for another 3 weeks. This will allow for proper vascularization of the Cell Pouch chambers and the patient to be stabilized on immunosuppression prior to islet transplantation. A mass of highly purified islets will be transplanted in the Cell Pouch. Patients who elect to retain the Cell Pouch after completion of the study will be asked to consent to long term safety follow-up, with data collected beyond the study scheduled visits every 3 months for up to 5 years until the last Cell Pouch explant (and then for a minimum of 3 months thereafter).

Sponsors & Collaborators

• ICON plc

  collaborator INDUSTRY

• University of Chicago

  collaborator OTHER

• Breakthrough T1D

  collaborator OTHER

• Sernova Biotherapeutics Inc.

  lead INDUSTRY

Principal Investigators

• Piotr Witkowski, MD, PhD · University of Chicago

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

65 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-02-07

Primary Completion

2026-10-31

Completion

2026-10-31

FDA Drug

Yes

Countries

• United States

Study Locations