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

NCT03513939 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: PROSVA201701
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief 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

Keywords

Type 1 Diabetes Mellitus; Diabetes; Hypoglycemia

Outcome Measures

Primary Outcomes (1)

• To assess the safety of the Cell Pouch following implantation, and islet transplantation, by evaluating the incidence and severity of adverse events (AEs) determined to be probable or highly probable to the Cell Pouch

  Safety will be assessed by evaluating the incidence and severity of adverse events (AEs) determined to be probable or highly probable to the Cell Pouch following initial Cell Pouch implantation, at the time of islet transplantation, and following islet transplantation, and throughout the study up to 365 days ±14 days post-islet transplantation

  365 days ±14 days

Secondary Outcomes (3)

• Survival of endocrine tissue in the Cell Pouch (defined by positive staining of islets during histological analysis)

  90±5 days post-transplant for sentinel Cell Pouch

• Proportion of participants with a reduction in severe hypoglycemic events

  From Day 0 to 90±5 ; Day 90±5 to Day 180±5; Day 180±5 to Day 365±14 following final Cell Pouch transplant and/or last transplant

• Proportion of participants with a reduction in HbA1c >1mg%

  From Day 0 to 90±5 ; Day 90±5 to Day 180±5; Day 180±5 to Day 365±14 following final Cell Pouch transplant and/or last transplant

Study Arms (1)

T1D Cell Pouch Recipients

EXPERIMENTAL

Eligible Type 1 Diabetes Mellitus (T1D) subjects with hypoglycemia unawareness and a history of severe hypoglycemic episodes undergoing Sernova Cell Pouch intervention

Combination Product: Sernova Cell Pouch

Interventions

Sernova Cell Pouch COMBINATION_PRODUCT

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).

T1D Cell Pouch Recipients

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female patients 18 to 65 years of age.
• Ability to provide written informed consent.
• Mentally stable and able to comply with the procedures of the study protocol.
• Clinical history compatible with Type 1 Diabetes Mellitus (T1DM) with onset of disease at \<40 years of age, insulin-dependence for ≥5 years at the time of consent, and a sum of patient age and insulin dependent diabetes duration of ≥28.
• Absent stimulated c-peptide (\<0.3 ng/mL) in response to a mixed meal tolerance test (MMTT; measured during the 4 hour test).
• Involvement in intensive diabetes management defined as self-monitoring of glucose values no less than a mean of three times each day averaged over each week and by the administration of three or more insulin injections each day or insulin pump therapy. Such management must be under the direction of an endocrinologist, diabetologist, or diabetes specialist with at least 3 clinical evaluations during the 12 months prior to study consent.
• At least one episode of severe hypoglycemia in the 12 months prior to study consent.
• Reduced awareness of hypoglycemia. More information about this criterion, including specific definitions of hypoglycemia unawareness, is in the protocol.

You may not qualify if:

• Body mass index (BMI) \>30 kg/m2
• Insulin requirement \>1.0 IU/kg/day
• Glycated Haemoglobin (HbAlc) \>13%.
• Untreated proliferative diabetic retinopathy.
• Blood Pressure: Systolic blood pressure (SBP) \>160 mmHg or Diastolic Blood Pressure (DBP) \>100 mmHg.
• Measured glomerular filtration rate \<70 mL/min/1.73m2 (More information about this criterion is in the protocol
• Presence or history of macroalbuminuria (\>300 mg/g creatinine).
• Presence or history of panel-reactive anti-HLA antibodies \>30%
• For female subjects of child bearing potential: Positive pregnancy test, presently breast-feeding, or unwillingness to use effective contraceptive measures for the duration of the study and 4 months after discontinuation. For male subjects: intent to procreate during the duration of the study or within 4 months after discontinuation or unwillingness to use effective measures of contraception. More information about this criterion is in the protocol.
• Presence or history of active infection including hepatitis B, hepatitis C, HIV, or tuberculosis (TB). Subjects with laboratory evidence of active infection are excluded even in the absence of clinical evidence of active infection.
• Patients with negative screen for Epstein Barr Virus by Immunoglobulin G (IgG) determination. More information about this criterion is in the protocol,
• Invasive aspergillus, histoplasmosis, or coccidioidomycosis infection within one year prior to study consent.
• Any history of malignancy except for completely resected squamous or basal cell carcinoma of the skin.
• Active smoking, vaping or marijuana use or known active alcohol or substance abuse.
• Baseline Hb below the lower limits of normal at the local laboratory for patients initially being enrolled into study.

Sponsors & Collaborators

• Sernova Biotherapeutics Inc.: lead
• ICON plc: collaborator
• University of Chicago: collaborator
• Breakthrough T1D: collaborator

Study Sites (1)

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus; Hypoglycemia

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Piotr Witkowski, MD, PhD

  University of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Prospective, non-randomized, single arm

Study Record Dates

• First Submitted: April 6, 2018
• First Posted: May 2, 2018
• Study Start: February 7, 2019
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: February 25, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)