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Safety and Tolerability Study of Larazotide Acetate in Celiac Disease Subjects

Phase 2
Completed
Conditions
Celiac Disease
Interventions
Registration Number
NCT00362856
Lead Sponsor
9 Meters Biopharma, Inc.
Brief Summary

This study was run to determine the safety, tolerance, and efficacy of multiple doses of larazotide acetate in subjects with celiac disease following a gluten challenge.

Detailed Description

CLIN1001-004 was a randomized, double-blind, placebo controlled, dose-ranging, 7-arm, multicenter study with a gluten challenge. The objects were multiple dose safety and tolerance; efficacy (intestinal permeability \[change in urinary LAMA ratio\] and disease signs and symptoms) following gluten challenge.

Following a 21-day screening period, subjects were randomized to one of seven treatments groups: four groups received larazotide acetate (0.25 mg, 1 mg, 4 mg or 8 mg TID) along with an 800 mg gluten challenge, one group received placebo with an 800 mg gluten challenge, a safety control arm received the highest dose of larazotide acetate (8 mg TID) and gluten placebo and the last group received drug placebo and gluten placebo. The gluten challenge was administered as capsules (800 mg TID) with each main meal for a total of 2.4 g daily. Drug or drug placebo was administered TID 15 minutes prior to each main meal. Subjects received their assigned treatments for two weeks (Day 0 through Day 14) and came to clinic for a follow-up visit one week later (Day 21). Subjects remained on their gluten-free diet for the duration of the study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
80
Inclusion Criteria
  • Must have been diagnosed with celiac disease by biopsy for ≥ 6 months.
  • Have a Anti-Tissue Transglutaminase (tTG) ≤ 10 EU as measured by serology.
  • Must be on a gluten-free diet for at least the past 6 months.
Exclusion Criteria
  • Have any chronic active GI disease other than celiac disease (e.g., IBS, Crohn's, Colitis).
  • Have diabetes (Type 1 or Type 2).
  • Chronically consumes non-steroidal anti-inflammatory agents ("NSAIDs") or takes proton-pump inhibitors.
  • Consuming oral corticosteroids or immune suppressants.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Larazotide acetate 8 mg + Gluten placebolarazotide acetateSafety Control Arm. Larazotide acetate 8 mg TID + gluten placebo TID administered orally in capsules
Larazotide acetate 8 mg + Glutenlarazotide acetateLarazotide acetate 8 mg TID + gluten 800 mg TID administered orally in capsules
Placebo + GlutenPlaceboplacebo TID + gluten 800 mg TID administered orally in capsules
Placebo + Gluten placeboPlaceboplacebo TID + gluten placebo TID administered orally in capsules
Larazotide acetate 4 mg + Glutenlarazotide acetateLarazotide acetate 4 mg TID + gluten 800 mg TID administered orally in capsules
Larazotide acetate 0.25 mg + Glutenlarazotide acetateLarazotide acetate 0.25 mg TID + gluten 800 mg TID administered orally in capsules
Larazotide acetate 1 mg + Glutenlarazotide acetateLarazotide acetate 1 mg TID + gluten 800 mg TID administered orally in capsules
Primary Outcome Measures
NameTimeMethod
To demonstrate the safety and tolerability of multiple, oral doses of larazotide acetate in celiac disease subjects that maintain a gluten-free diet.Safety measurements were performed at Screening and at Day 0, 7, 14, and 21 ('End of Study'). Any change from baseline value was calculated at each subsequent visit until "End of Study" (Day 21).

Safety endpoints assessed in this study were adverse events, vital signs, hematology, clinical chemistry, urinalysis and ECG

To evaluate the efficacy of multiple dose levels of larazotide acetate in preventing intestinal permeability changes induced by gluten challengeOn Days 0, 6, 13, and 20 subjects drank a solution of lactulose and mannitol. Subject's urine was collected during the day on Day 0 and overnight prior to subsequent visits and analyzed for LAMA recoveries via standardized methodologies.

The primary efficacy outcome was the Day 0-to-Day 14 change in urinary LAMA ratio ( a measure of intestinal permeability) as a response to gluten

Secondary Outcome Measures
NameTimeMethod
Changes in daily and weekly reported health outcomesSymptom diary - daily; PGWBI - weekly; GSRS - weekly

Health outcomes were assessed using a daily symptom diary; a weekly PGWBI and a weekly GSRS

Changes in urinary mannitol fractional excretion between Day 0 to Day 7 to Day 14See Primary Outcome Measure No. 2

See Primary Outcome Measure No. 2

Changes in urinary LAMA ratios between Day 0 to Day 7See Primary Outcome Measure No. 2

See Primary Outcome Measure No. 2

Changes in urinary lactulose fractional excretion between Day 0 to Day 7 to Day 14See Primary Outcome Measure No. 2

See Primary Outcome Measure No. 2

Change in anti-tTG levels from Screening to Day 21Screening and Day 21

Changes between screening and Day 21 anti-tTG levels were assessed for correlation to the measures of intestinal permeability

Change in urinary nitrite / nitrate levels from Day 0 to Day 14Day 0 and Day 14

Nitrite/nitrate levels were assessed for correlation to the measures of intestinal permeability

Change in cell markers and cytokines from PBMCsDays 0, 7, 14 and 21

Serum cytokine and cell surface marker determinations were assessed for correlation to the measures of intestinal permeability

Changes in zonulin levelDays 0, 7, 14 and 21

Serum zonulin levels were assessed for correlation to the measures of intestinal permeability

Trial Locations

Locations (1)

Research Site

🇺🇸

Richmond, Virginia, United States

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