PTG-100 for Patients With Celiac Disease

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

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.

Study Overview

• Status: Completed
• Conditions: Celiac Disease
• Intervention / Treatment: Drug: PTG-100; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Redwood City, California, United States, 94063
      • Stanford University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Nielsen Fernandez-Becker
• Collaborators: Protagonist Therapeutics, Inc.
• Investigators: Principal Investigator: Nielsen Q Fernandez-Becker, MD, PhD, Stanford University

Study record dates

Study Major Dates

• Study Start (Actual): February 26, 2021
• Primary Completion (Actual): April 3, 2022
• Study Completion (Actual): April 3, 2022

Study Registration Dates

• First Submitted: August 19, 2020
• First Submitted That Met QC Criteria: August 19, 2020
• First Posted (Actual): August 24, 2020

Study Record Updates

• Last Update Posted (Actual): May 23, 2022
• Last Update Submitted That Met QC Criteria: May 19, 2022
• Last Verified: May 1, 2022

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: 57613; PTG-100-05-CeD (Other Identifier: Stanford University)

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
• product manufactured in and exported from the U.S.: No