Study of the Safety, Tolerability, Pharmacokinetics and Biomarker of DONQ52 in Celiac Disease Patients

A Phase I, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate The Safety, Tolerability, Pharmacokinetics, and Biomarkers of DONQ52 in Celiac Disease Patients (LILY Study)

This study is to characterize the safety and tolerability of an investigational drug called DONQ52 and consists of a single ascending dose part (Part A) and a multiple ascending dose part (Part B) in well-controlled celiac disease patients.

Study Overview

• Status: Recruiting
• Conditions: Celiac Disease
• Intervention / Treatment: Drug: Placebo; Drug: DONQ52

• Study Type: Interventional
• Enrollment (Estimated): 56
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Clinical trials information; Phone Number: only use Email; Email: clinical-trials@chugai-pharm.co.jp

Study Locations

• Australia
  • New South Wales
    • Campbelltown, New South Wales, Australia, 2560
      • Recruiting
      • Campbelltown Hospital
  • Queensland
    • Morayfield, Queensland, Australia, 4506
      • Recruiting
      • University of Sunshine Coast Clinical Trials Centre - Morayfield
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Recruiting
      • Linear Clinical Research
• United States
  • Alabama
    • Anniston, Alabama, United States, 36207
      • Recruiting
      • Pinnacle Research Group
  • California
    • Walnut Creek, California, United States, 94598
      • Withdrawn
      • Diablo Clinical Research, Inc
  • Colorado
    • Denver, Colorado, United States, 80209
      • Recruiting
      • Mountain View Clinical Research
  • Florida
    • Jacksonville, Florida, United States, 32216
      • Recruiting
      • Jacksonville Center for Clinical Research
    • Leesburg, Florida, United States, 34748
      • Recruiting
      • Clinical Site Partners
    • Winter Park, Florida, United States, 32789
      • Recruiting
      • Clinical Site Partners - Orlando
  • Idaho
    • Meridian, Idaho, United States, 83642
      • Recruiting
      • Velocity Clinical Research - Boise
  • Louisiana
    • Marrero, Louisiana, United States, 70072
      • Recruiting
      • Tandem Clinical Research
  • Minnesota
    • Rochester, Minnesota, United States, 55095
      • Recruiting
      • Mayo Clinic - Rochester
  • New York
    • Great Neck, New York, United States, 11023
      • Recruiting
      • Long Island Gastrointestinal Research Group
    • New York, New York, United States, 10019
      • Recruiting
      • Columbia University Medical Center
  • North Carolina
    • Morehead City, North Carolina, United States, 28557
      • Recruiting
      • Lucas Research - Diabetes & Endocrinology Consultants, PC
    • Raleigh, North Carolina, United States, 27607
      • Recruiting
      • North Carolina Clinical Research
  • Ohio
    • Columbus, Ohio, United States, 43213
      • Recruiting
      • Aventiv Research, Inc.
    • Dayton, Ohio, United States, 45414
      • Recruiting
      • Digestive Specialists Inc
  • South Carolina
    • Anderson, South Carolina, United States, 29621
      • Recruiting
      • Velocity Clinical Research, Anderson
  • Tennessee
    • Knoxville, Tennessee, United States, 37920
      • Recruiting
      • Alliance for Multispecialty Research
  • Texas
    • Waco, Texas, United States, 76712
      • Recruiting
      • Digestive Research of Central Texas
  • Utah
    • Ogden, Utah, United States, 84403
      • Recruiting
      • Care Access Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• History of medically diagnosed celiac disease based on biopsies and positive celiac serology.
• Be on a GFD for at least 12 months
• HLA-DQ2.5 genotype
• Experienced at most mild symptoms of celiac disease

Exclusion Criteria:

• Refractory celiac disease
• Positive for any of the 3 serology (-Tissue transglutaminase-2,- Deamidated gliadin peptide-IgA, and deamidated gliadin peptide-IgG)

Study Plan

How is the study designed?

Design Details

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

Number of Arms

7

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SAD Cohort 1; All randomized patients will receive one dose of either DONQ52 Dose A or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: SAD Cohort 2; All randomized patients will receive one dose of either DONQ52 Dose B or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: SAD Cohort 3; All randomized patients will receive one dose of either DONQ52 Dose C or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: SAD Cohort 4; All randomized patients will receive one dose of either DONQ52 Dose D or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: MAD Cohort 1; All randomized patients will receive multiple dose of either DONQ52 Dose E or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: MAD Cohort 2; All randomized patients will receive multiple dose of either DONQ52 Dose F or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection
Experimental: MAD Cohort 3; All randomized patients will receive multiple dose of either DONQ52 Dose G or placebo	Drug: Placebo; Subcutaneous (SC) injection; Drug: DONQ52; Subcutaneous (SC) injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of treatment-emergent adverse events (TEAEs) as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 or higher	Incidence and severity of TEAEs and its relationship to the study drugs	Up to 246 days
Safety as assessed by Vital signs (blood pressure, body temperature, pulse rate, respiratory rate, percutaneous oxygen saturation)	Abnormality in vital signs	Up to 246 days
Safety as assessed by Electrocardiograms (ECGs; QT interval, heart rate)	Abnormality in Electrocardiograms (ECGs)	Up to 246 days
Safety as assessed by Laboratory tests (hematology, blood chemistry, coagulation and urinalysis)	Incidence of laboratory abnormalities, based on clinical laboratory tests	Up to 246 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics; Serum DONQ52 concentration	Serum DONQ52 concentrations over time	Up to 246 days
Pharmacokinetics; Maximum serum concentration [Cmax]	Cmax of DONQ52	Up to 246 days
Pharmacokinetics; Time to maximum serum concentration [Tmax]	Tmax of DONQ52	Up to 246 days
Pharmacokinetics; Area under the serum concentration time curve [AUC]	AUC of DONQ52	Up to 246 days
Pharmacokinetics; Half life [T1/2]	T1/2 of DONQ52	Up to 246 days
Immunogenicity	Prevalence and incidence of anti-drug antibodies (ADAs) to DONQ52	Up to 246 days

Collaborators and Investigators

• Sponsor: Chugai Pharmaceutical
• Investigators: Study Director: Sponsor Chugai Pharmaceutical Co. Ltd, clinical-trials@chugai-pharm.co.jp

Study record dates

Study Major Dates

• Study Start (Actual): September 19, 2022
• Primary Completion (Estimated): May 31, 2024
• Study Completion (Estimated): May 31, 2024

Study Registration Dates

• First Submitted: June 13, 2022
• First Submitted That Met QC Criteria: June 16, 2022
• First Posted (Actual): June 21, 2022

Study Record Updates

• Last Update Posted (Actual): November 28, 2023
• Last Update Submitted That Met QC Criteria: November 26, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Metabolic Diseases; Gastrointestinal Diseases; Intestinal Diseases; Malabsorption Syndromes; Celiac Disease
• Other Study ID Numbers: DQB101US

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to individual patient level data through the clinical study data request platform. For further details on Chugai's Data Sharing Policy and how to request access to related clinical study documents, see here (www.chugai-pharm.co.jp/english/profile/rd/ctds_request.html).

Drug and device information, study documents

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