Investigators outline how phase 1 safety data could inform a phase 2 efficacy study and eventual pediatric enrollment down to age 4 years.
Brian Vickery, MD, principal investigator of a first-in-human phase 1 trial testing a microneedle
Vickery, who also serves as vice chair of clinical research in the department of pediatrics at Emory University School of Medicine, has an ownership interest in Moonlight Therapeutics, Inc., the company developing the device.¹ The ascending-dose safety study is enrolling an initial adult cohort before proceeding through an age-de-escalating design toward younger participants.¹
"I think there's a real opportunity for a treatment like this to gain traction because there is still a lot of unmet need in the space," Vickery told HCPLive. Many patients in his clinic remain ineligible for existing food allergy therapies or have tried them and experienced an adverse effect.
The current study is designed to test safety and tolerability, with an additional goal of identifying a dose to carry into a phase 2 trial, according to Vickery. A phase 2 study would introduce repeated dosing over a period of months, potentially up to 52 weeks, to test for the first time whether the treatment changes reactivity to peanuts.
Efficacy studies in food allergy typically include oral food challenges at baseline and at the end of treatment to measure how reactivity shifts with immunotherapy. The current phase 1 protocol does not include food challenges, leaving this as a component reserved for later-stage studies.
If a phase 2 trial shows a signal toward efficacy, Vickery said the data would support the design of a properly powered, pivotal phase 3 study. This sequence, moving from safety to a repeated-dose efficacy signal to a pivotal trial, follows a conventional path for early-phase immunotherapy development.¹
The phase 1 trial is enrolling young adults and adults aged 18 to 26 years at Children's Healthcare of Atlanta and adults aged 27 to 55 years at Emory University in its initial cohort.¹ Following review by a data safety and monitoring committee, investigators plan to open a second cohort enrolling 10 adolescents, repeating the review process before any further expansion.¹ If the adolescent cohort is cleared, a third cohort would enroll 20 children as young as 4 years old, following an additional safety review by the committee.¹
Enrollment spans 5 sites nationally: Children's and Emory University in Atlanta, the University of North Carolina at Chapel Hill, the University of Arkansas, the University of Michigan, and a clinical research organization in Nebraska.¹ Vickery said the design lets investigators characterize safety in adults before extending exposure to pediatric participants.
Looking 5 to 10 years ahead, Vickery said he would like to see many treatment options for food allergies.
“As an allergist seeing patients, I would love to have a toolkit of 4, 5, 6 options to offer patients because we recognize that one size does not fit all in this condition,” he added.
Watch part 1 of our interview with Vickery here:
Reported disclosures for Vickery include Genentech USA, AQUESTIVE THERAPEUTICS, and Novartis Pharmaceuticals Corporation.
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