FDA Approves Amendment to Avita's Vitiligo Trial Design
Avita Medical, Inc. will move forward with an amended pivotal clinical trial evaluating the safety and effectiveness of the RECELL® System for the repigmentation of stable vitiligo lesions to a streamlined single-arm trial design, now that the FDA has approved the trial modification. The Company’s strategic decision to pursue a single cell suspension formulation (1:20 expansion ratio) is based on data from other research efforts that suggest the improbability of meaningful clinical performance differences amongst the three cell suspensions in the initial pivotal clinical trial design.
“The simplified study design and reduced number of study subjects reflects confidence both in the exceptional safety profile of RECELL and in the anticipated high incidence of repigmentation with RECELL treatment, as we have seen in 11 peer-reviewed publications and in the treatment of more than 1,000 patients outside the US,” says Dr. Mike Perry, Chief Executive Officer of Avita Medical. “The design change allows this program to progress in a timely and cost-effective manner toward bringing a novel therapeutic option to an underserved population. Our ongoing multi-media outreach and clinical and advocacy group referral programs are generating significant interest in the trial. The program is on track, and we continue to believe we could be in a position to enter the US market with the vitiligo indication, following successful completion of the clinical trial, as early as the second half of calendar year 2023.”
The multi-center pivotal study includes 15 clinical sites to assess the safety and effectiveness of the RECELL System in treatment of depigmented vitiligo lesions in patients whose vitiligo is stable, meaning they have not had new vitiligo lesions or lesions that have expanded for at least one year. The primary effectiveness evaluation is based on a comparison of the incidence of successful repigmentation with RECELL versus that of a standard of care control. Long-term durability data (assessing sustained repigmentation over 52 weeks) will be collected.
Each site is required to complete a run-in treatment as part of study initiation. Subjects treated in the earlier version of the pivotal clinical trial will be counted as part of the run-in cohort. After run-in, 23 subjects will be treated and evaluated in the final pivotal cohort. As with the previous design, an interim analysis will be conducted on 24-week data for approximately half of the subjects to evaluate sufficiency of the sample size, with a possible increase to sample size as needed (up to 46 subjects).