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PTG-100 for Patients With Celiac Disease

Phase 1
Completed
Conditions
Celiac Disease
Interventions
Drug: Placebo
Registration Number
NCT04524221
Lead Sponsor
Nielsen Fernandez-Becker
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
12
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PTG-100PTG-100Patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days.
PlaceboPlaceboPatients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days.
Primary Outcome Measures
NameTimeMethod
Percentage change in villous height-to crypt ratio42 days

Percentage change from baseline to Week 6 in villous height-to crypt depth (Vh:Cd) ratio

Secondary Outcome Measures
NameTimeMethod
Celiac symptom index (CSI)42 days

Changes in validated celiac symptom index (CSI)

CD3- positive intraepithelial lymphocyte density42 days

CD3- positive intraepithelial lymphocyte density

Changes in celiac disease antibodies42 days

Changes in anti-DGP antibodies from baseline

Change in celiac disease antibodies42 days

Changes in anti-tTG antibodies from baseline

Characterization of immune modulators underlying mechanism of action in celiac disease.42 days

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.

Trial Locations

Locations (1)

Stanford University

🇺🇸

Redwood City, California, United States

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