ReFocus202

20 evidence topics · 5 sources

Objective

Registered purpose of the trial

“The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma) harboring FGFR2 fusion or rearrangement.” (opens the source at this quote in a new tab)

Objective stated in the trial-in-progress abstract

“The ELE-4008-202, phase 2, open-label, single-arm study (NCT07359820) is designed to further evaluate the efficacy and safety of lirafugratinib 70 mg in 20-30 subjects across 20 sites with FGFR2 f/r, previously treated, unresectable, locally advanced or metastatic, non-CCA solid tumors who have not received prior FGFR inhibitor therapy.” (opens the source at this quote in a new tab)

Sponsor-stated regulatory purpose

“ReFocus202 aims to confirm the tumor-agnostic potential of lirafugratinib in patients with FGFR2 fusion/rearrangement.” (opens the source at this quote in a new tab)

Location and study date

Study dates and enrollment: registry record

Milestone	Registry record
Study start (actual)	“2026-06-04”
Primary completion (estimated)	“2027-09”
Study completion (estimated)	“2028-12”
Enrollment (estimated)	“30”

Registered sites

“This study has 19 locations” (opens the source at this quote in a new tab)

First participants dosed and participating countries

“The first patient was dosed at Samsung Medical Center in Seoul, South Korea, and a second patient at Moffitt Cancer Center in Tampa, Florida. The multi-site study is set to take place in the U.S., Korea, the UK, Spain, and France.” (opens the source at this quote in a new tab)

Study design

Registered design details

Design feature	Registry record
Primary purpose	“Treatment”
Allocation	“N/A”
Interventional model	“Single Group Assignment”
Masking	“None (Open Label)”

Pooling with the ReFocus Group 3 cohort

“An interim analysis of the pooled efficacy dataset from ELE-4008-202 and Group 3 of RLY-4008-101 will be conducted once approximately 65 evaluable subjects with non-CCA tumors have been enrolled across ≥7 tumor types (≥5 subjects per tumor type). Enrollment of a tumor type may be capped once it has reached approximately 20% of the total subjects in the pooled data from the two studies.” (opens the source at this quote in a new tab)

Eligibility criteria

Key inclusion criteria: registry record

Criterion	Quoted registry text
Diagnosis	“Unresectable, locally advanced, or metastatic solid tumor (other than CCA).”
FGFR2 alteration	“Documented FGFR2 gene fusion or rearrangement per local testing of blood and/or tumor.”
Prior FGFR inhibitor	“Subject has not received prior treatment with an FGFRi.”

Key exclusion criteria: registry record

“An uncontrolled comorbidity.” (opens the source at this quote in a new tab)
“Patient does not have adequate organ function (defined in protocol).” (opens the source at this quote in a new tab)
“Clinically significant, uncontrolled cardiovascular disease.” (opens the source at this quote in a new tab)
“CNS metastases or primary CNS tumor that is associated with progressive neurologic symptoms.” (opens the source at this quote in a new tab)

Exclusion of tumors with another targetable driver

“Cancer other than FGFR2 f/r and has a known primary driver alteration that is amenable to approved targeted therapy (e.g., EGFR, ALK, ROS1, RET, HER2, BRAF, IDH1, KRAS). However, the subject may be eligible after consultation with the Sponsor” (opens the source at this quote in a new tab)

Treatment

Registered intervention

Arm	Registry description
“Lirafugratinib”	“Treatment with standard dose of lirafugratinib”

Dose and cycle length

“Lirafugratinib 70 mg QD in 28-day cycles” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using RECIST v1.1 objective response rate as the primary endpoint

Registered primary outcome measure

Outcome measure	Time frame
“Objective Response Rate (ORR) assessed by Independent Review Committee per RECIST v1.1.”	“Approximately every 8 weeks during treatment and every 12 weeks after the last dose in the absence of progressive disease, approximately 36 months.”

Endpoints stated in the trial-in-progress abstract

“The primary endpoint is objective response rate (ORR) by independent review committee (IRC) per RECIST v1.1; key secondary endpoints include duration of response (DOR) by IRC, investigator-assessed efficacy endpoints, safety, pharmacokinetic (PK) parameters, and quality of life (QoL) per EORTC QLQ-C30.” (opens the source at this quote in a new tab)

Response threshold for the tissue-agnostic analysis

“With 65 subjects, observing at least 21 subjects with a complete response (CR) or partial response (PR) (ORR ≥32%), will result in a 95% CI (lower bound >20%) with the probability of observing ≥21 responders assuming a true ORR of 40% is approximately 92%.” (opens the source at this quote in a new tab)

Regulatory basis for ORR in single-arm studies

“When defined in this manner, ORR is a direct measure of a drug antitumor activity, which can be evaluated in a single-arm study.” (opens the source at this quote in a new tab)
“The significance of ORR is assessed by its magnitude and duration, and the percentage of CRs.” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment

“Approximately 20 to 30 patients will be enrolled in this study to reach the target patient population for the proposed interim analysis (i.e., at least 7 tumor types with ≥5 patients in each tumor type when pooled with 46 patients from Group 3 of Study RLY-4008-101).” (opens the source at this quote in a new tab)

Participants analyzed

No evidence found.

Description of analysis sets

Descriptive analysis and pooling of the two studies

“Statistical analyses of efficacy, safety, pharmacokinetic, and pharmacodynamic endpoints will be primarily descriptive in nature.” (opens the source at this quote in a new tab)
“All data from Study ELE-4008-202 will be pooled with the data of 46 patients from Group 3 of Study RLY-4008-101 for analysis.” (opens the source at this quote in a new tab)

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

ReFocus202 is an ongoing single-arm, open-label phase 2 study that began on June 4, 2026 and plans to enroll 20 to 30 participants with FGFR2 fusion- or rearrangement-positive solid tumors other than cholangiocarcinoma; primary completion is estimated for September 2027 and no results have been reported. It does not evaluate the approved cholangiocarcinoma indication. Its efficacy analysis pools its participants with the 46 participants of ReFocus Group 3, whose objective response rate was 33.3% (95% CI 19.6 to 49.5), so the planned interim analysis of approximately 65 evaluable participants is not an independent replication. Analyses are planned as primarily descriptive, with a threshold of at least 21 responders among 65 participants, and enrollment of any one tumor type may be capped at approximately 20% of the pooled total, which limits precision within individual tumor types.