A Study to Assess the Safety and Tolerability of Different Doses of AG019 Administered Alone or in Combination With Teplizumab in Participants With Recent-onset Diagnosed Type 1 Diabetes (T1D)

NCT ID: NCT03751007

Last Updated: 2023-02-01

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-10-24

Study Completion Date

2021-10-13

Brief Summary

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The purpose of this study is to assess the safety and tolerability of different doses of AG019 administered alone or in combination with teplizumab in participants with recent-onset type 1 diabetes (T1D).

Detailed Description

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This Phase 1b/2a, multi-center study will be conducted in participants with clinical recent-onset type 1 diabetes (T1D).

The primary objective of this study is to assess the safety and tolerability of different doses of AG019 alone as well as AG019 in combination with teplizumab. The secondary objectives of this study are: to obtain pharmacodynamic (PD) data of AG019 alone as well as AG019 in combination with teplizumab; and to determine the potential presence of AG019 in systemic circulation (safety - systemic exposure) and the presence of L. lactis bacteria in fecal excretion (local exposure): Pharmacokinetic (PK) profile.

This study consists of 2 phases:

Phase 1b: this open-label part of the study will investigate the safety and tolerability of 2 different doses of AG019 in 2 age groups (18-40 years of age and 12-17 years of age).

Phase 2a: this randomized, double-blind part of the study will investigate the safety and tolerability of AG019, in combination with teplizumab, in 2 age groups (18-40 years of age and 12-17 years of age).

Conditions

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Diabetes type1

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

The phase 1b part of the study will enroll 4 sequential AG019 cohorts of up to 6 participants, in ascending dose cohorts and descending age cohorts. All participants in these cohorts will be treated with AG019 in an open label fashion.

The phase 2a part of the study will evaluate 2 cohorts of participants administered AG019 and teplizumab. The first 2 participants will be treated with active treatment in an open label fashion. Participants 3-12 will be randomized (4:1) to receive active treatment or placebo in a double-blind fashion.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
For the randomized participants in the combination cohorts, blinding will be accomplished by arranging for AG019 and placebo components as well as teplizumab and placebo components to have identical packaging.

Study Groups

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AG019 Cohort 1 - Low Dose/Adults

Group Type EXPERIMENTAL

AG019 - Low Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 2 capsules per day for 1 day (single dose) or 8 weeks (repeat dose)

AG019 Cohort 2 - High Dose/Adults

Group Type EXPERIMENTAL

AG019 - High Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 6 capsules per day for 1 day (single dose) or 8 weeks

AG019 Cohort 3 - Low Dose/Adolescents

Group Type EXPERIMENTAL

AG019 - Low Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 2 capsules per day for 1 day (single dose) or 8 weeks (repeat dose)

AG019 Cohort 4 - High Dose/Adolescents

Group Type EXPERIMENTAL

AG019 - High Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 6 capsules per day for 1 day (single dose) or 8 weeks

Combination Cohort 1 - Adults

Group Type EXPERIMENTAL

Teplizumab

Intervention Type DRUG

Daily IV infusions of Teplizumab during the first 12 days of AG019 treatment. Total cumulative dose is approximately 17mg (dose calculation based on body surface area).

Placebo-AG019

Intervention Type DRUG

Formulated identically to AG019 with the active ingredient removed.

Placebo-Teplizumab

Intervention Type DRUG

Formulated identically to teplizumab with the active ingredient removed.

AG019 - High Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 6 capsules per day for 8 weeks.

Combination Cohort 2 - Adolescents

Group Type EXPERIMENTAL

Teplizumab

Intervention Type DRUG

Daily IV infusions of Teplizumab during the first 12 days of AG019 treatment. Total cumulative dose is approximately 17mg (dose calculation based on body surface area).

Placebo-AG019

Intervention Type DRUG

Formulated identically to AG019 with the active ingredient removed.

Placebo-Teplizumab

Intervention Type DRUG

Formulated identically to teplizumab with the active ingredient removed.

AG019 - High Dose

Intervention Type BIOLOGICAL

Solid, orally administered capsule - 6 capsules per day for 8 weeks.

Interventions

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AG019 - Low Dose

Solid, orally administered capsule - 2 capsules per day for 1 day (single dose) or 8 weeks (repeat dose)

Intervention Type BIOLOGICAL

Teplizumab

Daily IV infusions of Teplizumab during the first 12 days of AG019 treatment. Total cumulative dose is approximately 17mg (dose calculation based on body surface area).

Intervention Type DRUG

Placebo-AG019

Formulated identically to AG019 with the active ingredient removed.

Intervention Type DRUG

Placebo-Teplizumab

Formulated identically to teplizumab with the active ingredient removed.

Intervention Type DRUG

AG019 - High Dose

Solid, orally administered capsule - 6 capsules per day for 1 day (single dose) or 8 weeks

Intervention Type BIOLOGICAL

AG019 - High Dose

Solid, orally administered capsule - 6 capsules per day for 8 weeks.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Male or non-pregnant, non-lactating females, 18 - 40 years of age (both inclusive) or 12-17 years of age (both inclusive)
* Diagnosis of diabetes according to the American Diabetes Association (ADA) recommended criteria
* Evidence of auto-antibodies to at least 1 β-cell autoantigen
* Stimulated C-peptide measured during 4h Mixed Meal tolerance Test (MMTT) \> 0.2 nmol/L
* The first administration of AG019 should occur no later than 150 days post diagnosis of diabetes
* Body weight ≥ 33kg
* Written informed consent obtained and documented (participant, parent, guardian as applicable)

Exclusion Criteria

* Previous history of serious cytokine release syndrome to teplizumab or other humanized anti-CD3 monoclonal antibodies with no or minimal capacity to bind Fc receptors. (Participants enrolled in the second phase of the trial in either Combination Cohort 1 or Combination Cohort 2, only)
* Use of immunosuppressive or immunomodulatory therapies, including systemic steroids within 1 month prior to randomization
* Participation in another investigational drug trial within 12 weeks prior to the first study drug intake and during participation in this study
* History of recurrent infections, other autoimmune diseases, cardiac disease, malignancy, or any other (chronic) medical condition which, in the investigator's opinion, could compromise participant safety
* Documented history of human immunodeficiency virus (HIV), Hepatitis Virus Type C (HCV), Hepatitis Virus Type B (HBV) infection
* Evidence of active infection with Epstein-Barr Virus (EBV) or cytomegalovirus (CMV)
* Evidence of active or latent tuberculosis (TB)
* Administration of anti-CD3 antibody in past year
* Current therapy with any other anti-diabetic agents other than insulin (MDI, CSII or analogue). Current or planned therapy with experimental (i.e., unapproved) insulin. Patients on therapy for type 2 diabetes (e.g. metformin) should stop their therapy in order to be eligible for study participation.
* Use of medications known to influence glucose tolerance
* Daily use of non-steroidal anti-inflammatory agents
* Compromised GI mucosal integrity or motility, not attributable to T1D (i.e., recent diarrhea, gluten sensitive enteropathy, inflammatory bowel disease, irritable bowel syndrome), or current use of medications known to influence GI motility
* Positive result of SARS-Cov2 PCR test at screening or within 3 days before randomization
Minimum Eligible Age

12 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Intrexon Actobiotics NV, d/b/a Precigen Actobio

UNKNOWN

Sponsor Role collaborator

Precigen Actobio T1D, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Chantal Mathieu, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital of Leuven, Clinical and Experimental Endocrinology

Kevan Herold, MD

Role: PRINCIPAL_INVESTIGATOR

Yale Center for Clinical Investigation; Yale University

Locations

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University of Alabama, Birmingham

Birmingham, Alabama, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

Coastal Metabolic Research Centre

Ventura, California, United States

Site Status

University of Colorado

Aurora, Colorado, United States

Site Status

Yale Center for Clinical Investigation

New Haven, Connecticut, United States

Site Status

University of Miami

Miami, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Barry J Reiner, MD, LLC

Baltimore, Maryland, United States

Site Status

University of Minnesota Health

Minneapolis, Minnesota, United States

Site Status

University of Missouri-Kansas City School of Medicine

Kansas City, Missouri, United States

Site Status

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, United States

Site Status

University Diabetes and Endocrine Consultants

Chattanooga, Tennessee, United States

Site Status

Texas Diabetes & Endocrinology, P.A.

Austin, Texas, United States

Site Status

Research Institute of Dallas

Dallas, Texas, United States

Site Status

Benaroya Research Institute

Seattle, Washington, United States

Site Status

UZ Brussel

Brussels, , Belgium

Site Status

UZ Antwerpen

Edegem, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

Countries

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United States Belgium

References

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Mathieu C, Wiedeman A, Cerosaletti K, Long SA, Serti E, Cooney L, Vermeiren J, Caluwaerts S, Van Huynegem K, Steidler L, Blomme S, Rottiers P, Nepom GT, Herold KC; AG019-T1D-101 Trial Investigators. A first-in-human, open-label Phase 1b and a randomised, double-blind Phase 2a clinical trial in recent-onset type 1 diabetes with AG019 as monotherapy and in combination with teplizumab. Diabetologia. 2024 Jan;67(1):27-41. doi: 10.1007/s00125-023-06014-2. Epub 2023 Oct 2.

Reference Type DERIVED
PMID: 37782353 (View on PubMed)

Alexander LM, van Pijkeren JP. Modes of therapeutic delivery in synthetic microbiology. Trends Microbiol. 2023 Feb;31(2):197-211. doi: 10.1016/j.tim.2022.09.003. Epub 2022 Oct 8.

Reference Type DERIVED
PMID: 36220750 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017-002871-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AG019-T1D-101

Identifier Type: -

Identifier Source: org_study_id

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