Postmarketing requirement 5057-1 randomized dosage trial

4 evidence topics · 1 source

Objective

Required trial and its purpose

“Conduct a randomized clinical trial to further characterize known serious risks with lirafugratinib including severe stomatitis, palmar-plantar erythrodysesthesia syndrome, retinal pigment epithelial detachment, and nail toxicities, and to compare the safety and activity of lirafugratinib 70 mg once daily versus a lower dosage in patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or other rearrangements, who have not received prior FGFR inhibitors.” (opens the source at this quote in a new tab)

Location and study date

Timetable submitted by the sponsor

Milestone	Approval letter
Draft protocol submission	“Draft Protocol Submission: 01/2027”
Final protocol submission	“Final Protocol Submission: 05/2027”
Trial completion	“Trial Completion: 11/2031”
Final report submission	“Final Report Submission: 05/2032”

Registry record and trial sites

No evidence found.

Study design

Basis for requiring a clinical trial

“Finally, we have determined that only a clinical trial (rather than a nonclinical or observational study) will be sufficient to assess these serious risks.” (opens the source at this quote in a new tab)

Eligibility criteria

No evidence found.

Treatment

No evidence found.

Study outcomes

Rationale for using safety and activity at two dosages as the primary endpoint

No evidence found.

Statistical analysis description

Number of participants (Planned and analyzed)

No evidence found.

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

This randomized comparison of lirafugratinib 70 mg once daily against a lower dosage is a postmarketing requirement stated in the September 23, 2026 approval letter. No protocol, registry record, planned sample size, or endpoints were located; the sponsor's timetable schedules the draft protocol for January 2027, trial completion for November 2031, and the final report for May 2032. Until it reports, the dose-dependence of the stomatitis, palmar-plantar erythrodysesthesia, retinal pigment epithelial detachment, and nail toxicities seen with the approved dose remains uncharacterized in a randomized setting.