PTG-100 for Patients With Celiac Disease

Study Overview
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Brief Summary
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The goal of this study is to learn whether or not the drug PTG-100 can reduce or prevent inflammatory injury to the small intestine that occurs when people with celiac disease eat food products containing gluten. This is a clinical research study to determine the safety and efficacy of PTG-100 in preventing gluten-induced inflammatory injury to the small intestine in patients with celiac disease. 30 patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days. They will also receive a gluten challenge twice daily in the form of a cookie or equivalent. An upper gastrointestinal endoscopy and exam including small bowel mucosa biopsy will be performed at the start of the treatment period and again at the end. Blood samples will be routinely taken to evaluate safety and the drug's mechanism of action throughout the study, and symptoms will be recorded using the celiac symptoms index (CSI) survey.

Official Title
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A Phase 1b Study of PTG-100 in Patients With Celiac Disease

Conditions
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Celiac Disease

Intervention / Treatment
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* Drug: PTG-100
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of celiac disease Exclusion Criteria: Active GI disease or history of clinically significant diseases Diagnosis of Crohn's disease or ulcerative colitis.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 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
Interventional Model Description: Randomized, double-blind, placebo-controlled
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PTG-100<br>Patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days. | Drug: PTG-100<br>* PTG-100, 600mg taken twice daily in capsule form for 42 days<br>|
| Placebo Comparator: Placebo<br>Patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days. | Drug: Placebo<br>* Placebo taken twice daily in capsule form for 42 days<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage change in villous height-to crypt ratio | Percentage change from baseline to Week 6 in villous height-to crypt depth (Vh:Cd) ratio | 42 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in celiac disease antibodies | Changes in anti-tTG antibodies from baseline | 42 days | 
| Changes in celiac disease antibodies | Changes in anti-DGP antibodies from baseline | 42 days | 
| CD3- positive intraepithelial lymphocyte density | CD3- positive intraepithelial lymphocyte density | 42 days | 
| Celiac symptom index (CSI) | Changes in validated celiac symptom index (CSI) | 42 days | 
| Characterization of immune modulators underlying mechanism of action in celiac disease. | Identification of immune cell populations targeted by PTG-100 to better understand mechanism of action in celiac disease in both blood and tissue temporally following exposure to drug. | 42 days |