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Novel prebiotic improves abdominal symptoms in patients with lactose intolerance
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RP-G28, a non-digestible oligosaccharide designed to stimulate the gut microbiota to metabolize lactose, improved abdominal symptoms in patients with lactose intolerance in a phase 2b/3 trial, the manufacturer announced.
However, the trial did not meet its primary endpoint until researchers at one site excluded about 20% of the study population due to “significant irregularities.” The developer [Ritter Pharmaceuticals] believes the results support phase 3 development, which could be adequate to support FDA approval, according to a press release.
“I believe the totality of the results and the multitude of positive outcome measures demonstrates there is a clear treatment benefit for subjects taking RP-G28,” William Sandborn, MD, chief of the division of gastroenterology at University of California, San Diego, said in the press release. “The responder composite score instrument, which we worked with the FDA to establish, performed as anticipated to best quantify and evaluate clinically meaningful treatment benefit for lactose intolerance sufferers. This study was groundbreaking for both its size in lactose intolerance and in its design to identify clinically meaningful benefit to patients in the context of an evolving regulatory framework to demonstrate effect.”
William J. Sandborn
At 16 U.S. sites, Sandborn and colleagues randomly assigned 377 individuals with lactose intolerance to receive one of two doses of RP-G28 or placebo for 30 days, after which they entered a 30-day post-treatment dairy inclusion assessment.
The proportion of abdominal symptom responders served as the pre-specified primary endpoint, but after a recent Type C informational meeting with the FDA, the company changed the primary endpoint to a composite symptom score including abdominal pain, cramping, bloating and gas before unmasking the study data.
Among the 368 patients who received at least one dose, 40% of the treatment group vs. 31% of the placebo group were responders, which did not reach statistical significance. However, one study center demonstrated “significant irregularities,” and after excluding these patients, an analysis of the remaining 296 participants showed 40% of the pooled dosing group vs. 26% of the placebo group were responders, which reached significance (P = .0159). The low and high dose groups also showed significant benefits over placebo individually, and individual symptoms improved “both immediately post-dosing and 30 days post-dosing.”
The investigators observed no significant adverse events and safety measurements were comparable between treatment and placebo groups.
Additional endpoints, including gut microbiome outcomes, are being further reviewed, and the company plans to meet with the FDA to discuss the study results, the irregularities at the excluded center, and the design of a phase 3 program, according to the press release. – by Adam Leitenberger
Disclosures: Sandborn reports he serves on the medical advisory board for Ritter Pharmaceuticals.