A Study of Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KAN-101 in Celiac Disease (ACeD-it)

A Phase 1B Open-label/Phase 2 Double-blind Placebo- Controlled Study for Pharmacodynamic (PD) Activity, Pharmacokinetics (PK), Safety, and Tolerability of KAN-101 In Patients With Celiac Disease (CeD)

This study is to evaluate the Pharmacodynamic (PD), safety, tolerability, Pharmacokinetic (PK), and plasma biomarker response of KAN-101 in participants with Celiac Disease (CeD).

Study Overview

• Status: Terminated
• Conditions: Celiac Disease
• Intervention / Treatment: Drug: Cohort 1 in Part A; Drug: Cohort 2 in Part A; Other: Placebo: Group 1 in Part B and Part C; Drug: Group 2 in Part B and Part C; Drug: Group 3 in Part B and Part C; Drug: Group 4 in Part B and Part C

Detailed Description

The study is a 3-part, multicenter Phase 1b/2 study of KAN-101 in participants with Celiac Disease (CeD) on a gluten free diet (GFD). The 3 parts include:

• Part A - Open-label, multiple ascending dose
• Part B - Double-blind, placebo-controlled, parallel design
• Part C - Double-blind, placebo-controlled, parallel design

Part A is a Phase 1b, open-label, multiple ascending dose (MAD) study design to assess the safety, tolerability, and pharmacokinetics (PK) of KAN-101 in adult participants (18 to 70 years inclusive) with histology-confirmed CeD. Up to 12 participants who meet study inclusion/exclusion criteria will receive 1 of 2 dose levels of KAN-101. The overall study duration will be about 56 days, including up to 28 days of screening, 7 days of treatment and 21 days of follow up. There will be a gluten challenge test (GC) on Day 15.

Parts B and C are Phase 2, double-blind, placebo-controlled, parallel design study to characterize the biomarker response following GC, safety, tolerability, and PK of KAN-101 in adult participants with histology-confirmed CeD. Approximately 16 participants (4 participants per dose group) will be enrolled in Part B and 104 participants (26 participants per dose group) enrolled into Part C. Participants will be randomized 1:1:1:1 and stratified by participation in a biopsy substudy to 4 treatment groups: placebo and 3 treatment groups with KAN-101 doses based on information obtained from Part A.

• Study Type: Interventional
• Enrollment (Actual): 128
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Campbelltown, New South Wales, Australia, 2560
      • Campbelltown Hospital
  • Queensland
    • Auchenflower, Queensland, Australia, 4066
      • Wesley Research Institute
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
  • Victoria
    • Box Hill, Victoria, Australia, 3128
      • Box Hill Hospital
    • Parkville, Victoria, Australia, 3050
      • The Royal Melbourne Hospital
  • Western Australia
    • Midland, Western Australia, Australia, 6056
      • St John of God Midland Public and Private Hospitals
• New Zealand
  • Auckland
    • Auckland, Auckland, New Zealand, 1023
      • Optimal Clinical Trials
    • Takapuna, Auckland, New Zealand, 0622
      • PCRN Trials
  • Bay of Plenty
    • Tauranga, Bay of Plenty, New Zealand, 3110
      • P3 Research - Tauranga
  • Hamilton
    • Hamilton, Hamilton, New Zealand, 3204
      • Waikato Hospital
  • Otago
    • Dunedin, Otago, New Zealand, 9016
      • P3 Research - Dunedin
  • Wellington Region
    • Paraparaumu, Wellington Region, New Zealand, 5032
      • P3 Research - Palmerston North
    • Wellington, Wellington Region, New Zealand, 6021
      • P3 Research - Wellington
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham
  • California
    • Anaheim, California, United States, 92801
      • Anaheim Clinical Trials, LLC
  • Florida
    • St. Petersburg, Florida, United States, 33705
      • GCP Research
  • Georgia
    • Sandy Springs, Georgia, United States, 30328
      • Agile Clinical Research Trials
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
    • Normal, Illinois, United States, 61761
      • Sneeze, Wheeze & Itch Associates, LLC
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Health University Hospital
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
    • Saint Paul, Minnesota, United States, 55114
      • Prism Research LLC dba Nucleus Network
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • Nebraska
    • Omaha, Nebraska, United States, 68114
      • Quality Clinical Research
  • New York
    • New York, New York, United States, 10032
      • Celiac Disease Center at Columbia University
  • North Carolina
    • Raleigh, North Carolina, United States, 27607
      • North Carolina Clinical Research
  • Ohio
    • Columbus, Ohio, United States, 43213
      • Aventiv Research, Inc. d/b/a Centricity Research
    • Mentor, Ohio, United States, 44060
      • Great Lakes Gastroenterology Research, LLC
    • Westlake, Ohio, United States, 44145
      • NorthShore Gastroenterology Research, LLC
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas Health Science Center at Houston
    • Waco, Texas, United States, 76712
      • Digestive Research of Central Texas
  • Utah
    • Ogden, Utah, United States, 84405
      • Advanced Research Institute
    • West Jordan, Utah, United States, 84088
      • Velocity Clinical Research, Salt Lake City

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Previous diagnosis of celiac disease based on histology and positive celiac serology
• HLA-DQ2.5 genotype
• Gluten-free diet for at least 12 months
• Negative or weak positive for transglutaminase IgA and negative or weak positive for DGP-IgA/IgG during screening

Exclusion Criteria:

• Refractory celiac disease
• HLA-DQ8 genotype
• Previous oral gluten challenge within 12 months
• Selective IgA deficiency
• Diagnosis of Type-1 diabetes
• Active gastrointestinal diseases
• History of dermatitis herpetiformis

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1 in Part A; All eligible Part A participants will receive 3 intravenous (IV) infusions of KAN-101 Dose 1	Drug: Cohort 1 in Part A; Dose 1 KAN-101 Intravenous (IV) infusion; Other Names: KAN-101
Experimental: Cohort 2 in Part A; All eligible Part A participants will receive 3 intravenous (IV) infusions of KAN-101 Dose 2	Drug: Cohort 2 in Part A; Dose 2 KAN-101 Intravenous (IV) infusion; Other Names: KAN-101
Placebo Comparator: Group 1 in Part B and Part C; All eligible Part B and Part C participants will receive 3 intravenous (IV) infusions of placebo	Other: Placebo: Group 1 in Part B and Part C; Placebo Intravenous (IV) infusion; Other Names: Placebo
Experimental: Group 2 in Part B and Part C; All eligible Part B and Part C participants will receive 3 intravenous (IV) infusions of KAN-101 Dose 3	Drug: Group 2 in Part B and Part C; Dose 3 KAN-101 Intravenous (IV) infusion; Other Names: KAN-101
Experimental: Group 3 in Part B and Part C; All eligible Part B and Part C participants will receive 3 intravenous (IV) infusions of KAN-101 Dose 4	Drug: Group 3 in Part B and Part C; Dose 4 KAN-101 Intravenous (IV) infusion; Other Names: KAN-101
Experimental: Group 4 in Part B and Part C; All eligible Part B and Part C participants will receive 3 intravenous (IV) infusions of KAN-101 Dose 5	Drug: Group 4 in Part B and Part C; Dose 5 KAN-101 Intravenous (IV) infusion; Other Names: KAN-101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and Severity of TEAEs as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) in Part A	An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious and all non-serious adverse events. SAE was defined as any untoward medical occurrence that, at any dose resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event. According to NCI CTCAE version 5: Grade 1= mild AE; Grade 2= moderate AE; Grade 3=severe AE; Grade 4= life-threatening consequences and urgent intervention indicated; Grade 5= death related to AE.	From screening until the safety follow-up visit on Day 28
Change in Pre- and Post-Gluten Challenge (GC) IL-2 Response From Baseline to Day 15	CeD increases the circulating level of IL-2. Plasma samples were collected to assess the magnitude of biomarker response of IL-2 at the baseline screening visit pre-GC and again post-GC on Day 15 after 3 doses of KAN-101, the IL-2 response to GC is the difference of IL-2 between pre-GC and post-GC.	From Baseline screening to Day 15
Change in IL-2 Response From Day 15 Pre-GC to Day 15 Post GC	CeD increases the circulating level of IL-2. Plasma samples were collected to assess the magnitude of biomarker response of IL-2 at the baseline screening visit pre-GC and again post-GC on Day 15 after 3 doses of KAN-101, the IL-2 response to GC is the difference of IL-2 between pre-GC and post-GC.	0 (pre-GC) and 4 hours post-GC on Day 15

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
KAN-101 Plasma Exposure in Part A: AUCinf	PK sample collection at pre- dose and post dose timepoints in Part A.	Pre-dose, end of infusion, 45 minutes, 1 hour, 1.5 hour, 2.5 hours, 4.5 hours and 7 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part A: AUClast	PK sample collection at pre- dose and post dose timepoints in Part A.	Pre-dose, end of infusion, 45 minutes, 1 hour, 1.5 hour, 2.5 hours, 4.5 hours and 7 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part A: Cmax	PK sample collection at pre- dose and post dose timepoints in Part A.	Pre-dose, end of infusion, 45 minutes, 1 hour, 1.5 hour, 2.5 hours, 4.5 hours and 7 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part A: Tmax	PK sample collection at pre- dose and post dose timepoints in Part A.	Pre-dose, end of infusion, 45 minutes, 1 hour, 1.5 hour, 2.5 hours, 4.5 hours and 7 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part A: t½	PK sample collection at pre- dose and post dose timepoints in Part A.	Pre-dose, end of infusion, 45 minutes, 1 hour, 1.5 hour, 2.5 hours, 4.5 hours and 7 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part B and Part C: AUCinf	PK sample collection at pre- dose and post dose timepoints in Part B and Part C.	Pre-dose, end of infusion, 2.5 hours, and 4 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part B and Part C: AUClast	PK sample collection at pre- dose and post dose timepoints in Part B and Part C.	Pre-dose, end of infusion, 2.5 hours, and 4 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part B and Part C: Cmax	PK sample collection at pre- dose and post dose timepoints in Part B and Part C.	Pre-dose, end of infusion, 2.5 hours, and 4 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part B and Part C: Tmax	PK sample collection at pre- dose and post dose timepoints in Part B and Part C.	Pre-dose, end of infusion, 2.5 hours, and 4 hours after infusion start on Day 1 and Day 7
KAN-101 Plasma Exposure in Part B and Part C: t½	PK sample collection at pre- dose and post dose timepoints in Part B and Part C.	Pre-dose, end of infusion, 2.5 hours, and 4 hours after infusion start on Day 1 and Day 7
Incidence and Severity of TEAE as Assessed by the CTCAE in Part B	An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious and all non-serious adverse events. SAE was defined as any untoward medical occurrence that, at any dose resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event. According to NCI CTCAE version 5: Grade 1= mild AE; Grade 2= moderate AE; Grade 3=severe AE; Grade 4= life-threatening consequences and urgent intervention indicated; Grade 5= death related to AE.	From the time the participant provided informed consent through Week 52
Incidence and Severity of TEAE as Assessed by the CTCAE in Part C	An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious and all non-serious adverse events. SAE was defined as any untoward medical occurrence that, at any dose resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event. According to NCI CTCAE version 5: Grade 1= mild AE; Grade 2= moderate AE; Grade 3=severe AE; Grade 4= life-threatening consequences and urgent intervention indicated; Grade 5= death related to AE.	From the time the participant provided informed consent through Week 52

Collaborators and Investigators

• Sponsor: Kanyos Bio, Inc., a wholly-owned subsidiary of Anokion SA
• Collaborators: Pfizer
• Investigators: Study Director: Study Director, Anokion SA

Publications and helpful links

General Publications

• Murray JA, Wassaf D, Dunn K, Arora S, Winkle P, Stacey H, Cooper S, Goldstein KE, Manchanda R, Kontos S, Grebe KM. Safety and tolerability of KAN-101, a liver-targeted immune tolerance therapy, in patients with coeliac disease (ACeD): a phase 1 trial. Lancet Gastroenterol Hepatol. 2023 Aug;8(8):735-747. doi: 10.1016/S2468-1253(23)00107-3. Epub 2023 Jun 14.

Study record dates

Study Major Dates

• Study Start (Actual): November 15, 2022
• Primary Completion (Actual): November 29, 2024
• Study Completion (Actual): May 19, 2025

Study Registration Dates

• First Submitted: October 7, 2022
• First Submitted That Met QC Criteria: October 7, 2022
• First Posted (Actual): October 10, 2022

Study Record Updates

• Last Update Posted (Actual): March 10, 2026
• Last Update Submitted That Met QC Criteria: March 9, 2026
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: celiac disease; gluten free diet; HLA-DQ2.5
• Additional Relevant MeSH Terms: Metabolic Diseases; Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Malabsorption Syndromes; Nutritional and Metabolic Diseases; Celiac Disease
• Other Study ID Numbers: KAN-101-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No