A Study of CNSA-001 in Women With Diabetic Gastroparesis

Phase 2 Completed 21 enrolled PTC Therapeutics

RandomizedParallel-groupDouble-blindTreatment

Summary

This study evaluates CNSA-001 (sepiapterin) in the treatment of women with moderate to severe diabetic gastroparesis. Participants will be randomized in a ratio of 1:1 to receive CNSA-001 20 (milligrams) mg/kilogram (kg)/day or placebo. All participants will receive the standard of care for diabetic gastroparesis.

Timeline

Start: 2019-02-27
Primary completion: 2019-09-08
Completion: 2019-10-08

Outcome

Mixed primary results

registry analysis (other test); CNSA-001 vs Placebo; p = 0.9030; Least Square (LS) Mean Difference -6.44 (95% CI -116.18 to 103.31); ANCOVA NCT03712124 ↗

registry analysis (other test); CNSA-001 vs Placebo; p = 0.0042; LS Mean Difference 98.45 (95% CI 35.81 to 161.08); ANCOVA NCT03712124 ↗

Publications

Abell TL, Garcia LM, Wiener GJ, Wo JM, Bulat RS, Smith N. Effect of Oral CNSA-001 (sepiapterin, PTC923) on gastric accommodation in women with diabetic gastroparesis: A randomized, placebo-controlled, Phase 2 trial. J Diabetes Complications. 2021 Sep;35(9):107961. doi: 10.1016/j.jdiacomp.2021.107961. Epub 2021 Jun 17.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sepiapterin	Small molecule	10 mg/kg	Oral