FDA Clarifies Gastroparesis Drugs Trial Expectations

Drugmakers developing treatments for diabetic and idiopathic gastroparesis should study those patient populations in separate trials, the FDA says.

Patients with both forms of gastroparesis tend to experience the same signs and symptoms, but at varying levels, the agency notes in draft guidance issued Wednesday. The guidance covers clinical trial design and assessment of endpoints for gastroparesis drugs.

Sponsors should evaluate five core signs and symptoms of gastroparesis — nausea, vomiting, early fullness, abdominal pain and pre-meal fullness — until well-defined and reliable patient-reported outcomes are available to assess efficacy.

Trials should be randomized, double-blind, placebo-controlled and include a one- to two-week screening period. Treatment should run at least 12 weeks, followed by a two- to four-week randomized withdrawal period to determine if maintenance therapy is needed. Drugmakers also should conduct a 12-month placebo-controlled safety study.

Changes in signs and symptoms from baseline, measured separately or as a summary score, should serve as primary endpoints, the guidance says. Secondary endpoints should measure changes from baseline in individual signs and symptoms that are not assessed as part of the primary endpoint.

Comments on the draft guidance are due Sept. 21. Read Gastroparesis: Clinical Evaluation of Drugs for Treatment at www.fdanews.com/7-15-FDA-Gastroparesis.pdf. — Jonathon Shacat