A Phase I Clinical Study of YN003 in the Treatment of Glioma

A Phase 1, Open-Label, Single-Arm, Dose-Escalation and Dose-Expansion Study of YN003 Injection in Patients With Glioma

This is a Phase 1, open-label, single-arm, dose-escalation and dose-expansion study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of YN003 Injection in patients with recurrent or progressive glioma. The study consists of Part A (dose escalation using a Bayesian Optimal Interval design) and Part B (dose expansion at the recommended Phase 2 dose).

Study Overview

• Status: Recruiting
• Conditions: Glioma; Glioblastoma; Brain Neoplasms
• Intervention / Treatment: Drug: YN003 Injection

Detailed Description

YN003 is an investigationalliposome injection designed to deliver the active drug preferentially to specific glioma tissue. Part A will evaluate ascending doses of YN003 administered by intravenous infusion once every 4 weeks to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D). The dose-limiting toxicity (DLT) observation period is 28 days. Approximately 21 to 30 DLT-evaluable subjects will be enrolled in Part A. Part B will enroll approximately 10 to 20 additional subjects at the RP2D to further evaluate safety, pharmacokinetics, and preliminary efficacy. The study will be conducted in the United States and China.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Kaiqi Zong, Master; Phone Number: 13683178623; Email: zongkaiqi@innovmedicine.com
• Study Contact Backup: Name: Yaqian Cui, Master; Phone Number: 18811320289; Email: cuiyaqian@innovmedicine.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 102218
      • Recruiting
      • Beijing Tsinghua Changgung Hospital
      • Contact: Xuejun Yang, Ph.D; Phone Number: 13011329950; Email: yxja03728@btch.edu.cn
    • Beijing, Beijing Municipality, China
      • Recruiting
      • Sanbo Brain Hospital, Capital Medical University
      • Contact: Junping Zhang, Ph.D; Phone Number: 13811099759; Email: doczhjp@hotmail.com
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Not yet recruiting
      • University of California, Los Angeles (UCLA)
      • Contact: Timothy Cloughesy, Ph.D; Email: John.deGroot@ucsf.edu
    • San Francisco, California, United States, 94158
      • Not yet recruiting
      • University of California, San Francisco (UCSF)
      • Contact: Nazanin Majd, Ph.D; Email: NKMajd@mdanderson.org
  • Texas
    • Houston, Texas, United States, 77030
      • Not yet recruiting
      • The University of Texas MD Anderson Cancer Center
      • Contact: Nazanin Majd, Ph.D; Email: TCloughesy@mednet.ucla.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Voluntarily sign the informed consent form, understand the study, and be willing to comply with the protocol and complete all study procedures.
• Male or female subjects 18 years ≤ aged ≤ 70 years.
• Histologically confirmed recurrent or progressive glioma, including glioblastoma, as defined by the 2021 WHO Classification of Tumors of the Central Nervous System, Grade 3 to Grade 4, following standard therapy.Excluding re-irradiation for same lesions
• Karnofsky Performance Status (KPS) ≥60.
• At least one measurable lesion per RANO 2.0 criteria.
• Have adequate hematologic, cardiac, hepatic, renal, coagulation, and pulmonary function at final screening.
• Have completed the required washout period for prior therapies before dosing on Day 1.
• Corticosteroid dose must be stable or tapering within 7 days prior to dosing.

Exclusion Criteria:

• Participation in another interventional clinical study within 1 month prior to the date when subject signed the consent form.
• Receipt of a live vaccine within 30 days prior to study treatment.
• Pregnant or breastfeeding women.
• Clinically significant intracranial hypertension (e.g., impending brain herniation or requiring urgent palliative intervention) or uncontrolled seizures. Subjects requiring antiepileptic drugs are eligible only if the dose has been stable for ≥ 14 days prior to first dosing
• Toxicities from prior anticancer therapy that have not recovered to CTCAE v6.0 Grade ≤1, except for alopecia or other events considered tolerable by the investigator.
• Contraindications to MRI, including but not limited to pacemakers, epicardial pacing leads, infusion pumps, aneurysm clips, metal fragments, metallic prostheses, magnetic implants, intraocular metal objects, or non-removable metallic dental implants.
• Uncontrolled or severe cardiovascular or cerebrovascular disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Study Drug： YN003 Injection; Single-arm, open-label study of YN003 administered by intravenous infusion once every 4 weeks.

Drug: YN003 Injection; YN003 is an investigational liposome injection designed to deliver the drug preferentially to specific glioma tissue. Part A will evaluate ascending doses of YN003 administered by intravenous infusion once every 4 weeks to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D). The dose-limiting toxicity (DLT) observation period is 28 days. Approximately 21 to 30 DLT-evaluable subjects will be enrolled in Part A. Part B will enroll approximately 10 to 20 additional subjects at the RP2D to further evaluate safety, pharmacokinetics, and preliminary efficacy. The study will be conducted in the United States and China.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To Incidence and characteristics of AEs/SAEs；	Assessed and graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 6.0.	From first dose of YN003 to 30 days after the last dose (or initiation of new antitumor therapy, whichever occurs first)
Incidence of clinically significant abnormalities in laboratory tests	Percentage of participants with clinically significant abnormalities in hematology, blood chemistry, urinalysis, and coagulation lipid, Thyroid function.	From first dose of YN003 to 30 days after the last dose (or initiation of new antitumor therapy, whichever occurs first)
Incidence of clinically significant abnormalities in vital signs	Percentage of participants with clinically significant abnormalities in body temperature, respiratory rate, blood pressure, pulse rate, and oxygen saturation.	From first dose of YN003 to 30 days after the last dose (or initiation of new antitumor therapy, whichever occurs first)
Incidence of clinically significant abnormalities in physical examinations	Percentage of participants with clinically significant abnormalities in general physical examinations and neurological examinations.	From first dose of YN003 to 30 days after the last dose (or initiation of new antitumor therapy, whichever occurs first)
Change from baseline in 12-lead Electrocardiogram (ECG) parameters	To Incidence and characteristics of Clinically significant changes of ECG The ECG evaluation must include heart rate, QT interval, QTcF, and PR interval.	From first dose of YN003 to 30 days after the last dose (or initiation of new antitumor therapy, whichever occurs first)
To assess the tolerability of YN003 in subjects with recurrent/progressive glioma (WHO grade 3 - 4)	Dose-limiting toxicity (DLT) Maximum tolerated dose (MTD)	First 28 days after the first dose of YN003 MTD：During dose escalation (Part A), approximately 21-30 DLT-evaluable subjects]

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Peak Plasma Concentration (Cmax) and Time to Peak Plasma Concentration (Tmax) of YN003	Maximum observed plasma concentration (Cmax) and time to reach maximum observed plasma concentration (Tmax).	Up to 28 days of post initiation of last dose
Area Under the Plasma Concentration-Time Curve (AUC) of YN003	Area under the plasma concentration versus time curve from time zero to the last quantifiable time point (AUC0-t) and from time zero to infinity (AUC0-∞).	Up to 28 days of post initiation of last dose
Terminal Half-life (t1/2), Clearance (CL), and Volume of Distribution (Vd) of YN003	Terminal elimination half-life, total body clearance, and apparent volume of distribution.	Up to 28 days of post initiation of last dose
Overall Survival (OS)	OS rates at 6 and 12 months.	From 1st dose to death from any cause, assessed at 6 and 12 months after first dose.
Progression-Free Survival (PFS)	PFS rates at 6 and 12 months, assessed by the investigator per RANO 2.0 criteria.	From 1st dose until the date to first documented disease progression or death from any cause, whichever comes first, assessed at 6 and 12 months after first dose.
Objective Response Rate (ORR)	Percentage of participants with a confirmed complete response (CR) or partial response (PR) as assessed by the investigator per RANO 2.0 criteria.	From first dose until disease progression or initiation of new antitumor therapy (estimated up to 2 years).
Duration of Response (DoR)	Time from the first documented objective response to the date of first documented disease progression or death from any cause, whichever comes first.	From the date of 1st dose to the date of first documented disease progression or death (estimated up to 1 year).
Disease Control Rate (DCR)	Percentage of participants with a confirmed complete response (CR), partial response (PR), or stable disease (SD) as assessed by the investigator per RANO 2.0 criteria.	From first dose until disease progression or initiation of new antitumor therapy (estimated up to 2 years).

Collaborators and Investigators

• Sponsor: Beijing Inno Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): October 27, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: October 19, 2026
• First Submitted That Met QC Criteria: November 4, 2026
• First Posted (Actual): November 10, 2026

Study Record Updates

• Last Update Posted (Actual): November 10, 2026
• Last Update Submitted That Met QC Criteria: November 4, 2026
• Last Verified: November 1, 2026

More Information

Terms related to this study

• Keywords: glioma; progressive; recurrent; BOIN; YN003; liposomal injection
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Neoplasms by Site; Neoplasms; Disease Attributes; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Astrocytoma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Nervous System Neoplasms; Central Nervous System Neoplasms; Pathological Conditions, Signs and Symptoms; Recurrence; Glioblastoma; Glioma; Brain Neoplasms
• Other Study ID Numbers: YN003-101; Beijing Inno Medicine Co., Ltd (Registry Identifier: IND181709)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No