Phase Ib Study of Oral R01 Monotherapy in Advanced Solid Tumors

Conditions

• Esophageal Cancer
• Gastric Cancer
• Squamous Cell Lung Cancer

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• R01 — DRUG
  The study consists of two parts: (1) Dose Escalation Phase: Three prespecified escalating dose levels will be evaluated: 240 mg BID, 320 mg BID, and 400 mg BID. (2) Dose Expansion Phase: Subjects will receive the expansion dose determined in the dose escalation phase. All subjects will receive R01 orally, twice daily, with each treatment cycle consisting of 21 days. Treatment will continue until the discontinuation criteria specified in the study protocol are met.

Study Details

This is a Phase I（Ib）, open-label, single-arm clinical study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of R01 administered orally as monotherapy twice daily (BID) in patients with advanced solid tumors, including squamous cell lung cancer, gastric cancer , and esophageal cancer. Patients with advanced squamous cell lung cancer, gastric cancer , and esophageal cancer will be enrolled. All subjects will receive oral R01 monotherapy and will undergo physical examinations, laboratory tests, radiographic tumor response assessments, biological sample collection, and safety follow-ups as specified in the protocol. The study comprises two consecutive sub-stages: a dose-bridging escalation stage (i.e., a dose escalation stage designed to bridge from the highest dose evaluated in Phase Ia) and a dose expansion stage. The dose escalation follows a classic '3+3' design at three dose levels (240, 320, and 400 mg BID). The 400 mg dose may be initiated after the Safety Review Committee (SRC) review based on safety and PK data. The primary objectives of this stage are to characterize dose-limiting toxicities (DLTs), determine the maximum tolerated dose (MTD), and establish the recommended Phase II dose (RP2D). The dose expansion stage will be conducted at the RP2D in selected indication cohorts, with a planned enrollment of at least 6 subjects per cohort (including subjects with the corresponding indications enrolled at the same dose level during the dose escalation stage), to further evaluate the safety, tolerability, PK characteristics, and preliminary antitumor efficacy at this dose level. With respect to study endpoints: during the dose escalation stage, the primary endpoints are the incidence of DLTs, the MTD, and/or the determination of the expansion dose (RP2D); secondary endpoints include PK parameters of R01 and its metabolites, objective response rate (ORR), disease control rate (DCR), progression-free survival (PFS), and overall survival (OS). During the dose expansion stage, the primary endpoints are ORR and the incidence and severity of treatment-related adverse events (TRAEs); secondary endpoints include duration of response (DOR), time to response (TTR), DCR, PFS, OS, and steady-state PK parameters.

Key Dates

First listed

Sep 8, 2026

Start date

Sep 30, 2026

Status verified

Sep 2026

Primary completion

Sep 30, 2027

Completion

Sep 30, 2027

Study Design

Enrollment

18 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Experimental: R01 Oral Monotherapy
  This is a single-arm, open-label study consisting of a dose escalation phase and a dose expansion phase. The investigational product R01 is an oral formulation for the treatment of patients with advanced solid tumors.The dose escalation phase will evaluate three prespecified dose levels (240 mg BID, 320 mg BID, and 400 mg BID). Participants will be enrolled sequentially to determine the maximum tolerated dose (MTD) and/or the recommended dose for expansion. In the dose expansion phase, participants will uniformly receive the expansion dose determined during the dose escalation phase to further evaluate safety and preliminary efficacy.All participants will receive oral R01 twice daily, with each treatment cycle consisting of 21 days. Treatment will continue until disease progression, unacceptable toxicity, withdrawal of informed consent, or other discontinuation criteria specified in the protocol.

Primary Outcome Measure

Number of Participants with Dose-Limiting Toxicities (DLTs) [ Time Frame: Cycle 1 (21 days) ]

Central Contacts

• Haiyong Wang, PhD
  86+18911251079
  [email protected]

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