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

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Celiac Disease

Intervention / Treatment
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* Drug: DONQ52
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

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

What is the study measuring?
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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 |