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Safety, Tolerability, and Efficacy of Immunomodulation With AT-1501 in Combination With Transplanted Islet Cells in Adults With Brittle T1D

Phase 1
Withdrawn
Conditions
Brittle Type 1 Diabetes Mellitus
Interventions
Biological: AT-1501 IV Infusion
Biological: Isolated cadaveric islet cells
Registration Number
NCT05480657
Lead Sponsor
Eledon Pharmaceuticals
Brief Summary

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.

Detailed Description

This study will evaluate the safety, tolerability and efficacy of transplantation of experimental islet cells as a potential treatment for brittle type 1 diabetes mellitus. It will also assess the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with brittle T1D undergoing an islet cell transplant. This is a single arm open-label study and up to 6 participants will be recruited at a single center in the United States.

The objectives include:

* To assess the safety and tolerability of transplanted islet cells and 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 transplanted islet cells and immunomodulation with AT-1501 in adults with brittle T1D undergoing islet cell transplant.

The duration of treatment may vary from participant to participant and could be up to 2 years. Participants may receive up to 2 islet cell transplants.

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  • Men and women 18-65 years of age
  • A diagnosis of T1D ≥5 years with onset of disease at <40 years of age
  • Involvement in appropriate diabetes management in accordance with the standard of care, 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 unexplained SHEs
  • At least 3 unexplained SHEs not secondary to a missed meal or dosing error, in the 12 months prior to Screening
  • Glycosylated hemoglobin (HbA1c) level greater than 7% (53 mmol/mol) and less than 9.5% (80 mmol/mol) inclusive
  • 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
  • Impaired awareness of hypoglycemia (IAH) 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
  • Any previous solid organ or islet allotransplant
  • Body mass index (BMI) >30 kg/m2
  • Insulin requirement >1.0 unit/kg/day or <15 units/day

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Single Arm AT-1501Isolated cadaveric islet cellsSingle-arm, open-label trial
Single Arm AT-1501AT-1501 IV InfusionSingle-arm, open-label trial
Primary Outcome Measures
NameTimeMethod
Efficacy - Insulin independenceDate of transplant through Day 364 post- final transplant

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

Safety - Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)Accessed from date of transplant through Day 364 post final transplant for approximately 2 years

Incidence of adverse events

Secondary Outcome Measures
NameTimeMethod
Efficacy - Graft failureProportion of participants with graft failure at Day 364 post final transplant

Date of transplant through Day 364 post final transplant

Efficacy - HbA1cDate of transplant through Day 364 post-final transplant

Proportion of participants with HbA1c \<7.0% (53 mmol/mol) and free of serious hypoglycemic events (SHEs) from Day 28 to Day 364 post first and final transplant.

Efficacy - Durability of insulin independence - long term2 and 3 years after discontinuation of AT- 1501

The proportion of participants that become insulin independent at year 2 and year 3

Efficacy - Durability of insulin independenceDate of transplant through Day 364 post final transplant

The proportion of participants that become insulin independent at Day 364 post-transplant

Trial Locations

Locations (1)

University of Chicago

🇺🇸

Chicago, Illinois, United States

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