KSD-201 Treatment for Advanced Clear Cell Renal Cell Carcinoma: an Early Exploratory Clinical Study

A Single-arm, Open-label, Single-center, Multiple-dose Early Exploratory Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of KSD-201 in Patients With Advanced Clear Cell Renal Cell Carcinoma

The main purpse of this study is to evaluate the safety and tolerability of KSD-101 in patients with advanced clear cell renal cell carcinoma, to evaluate the initial clinical outcomes , immune response, and to evaluate the impact of KSD-201 on the quality of life in patients with advanced clear cell renal cell carcinoma.

Study Overview

• Status: Withdrawn
• Conditions: Clear Cell Renal Cell Carcinoma (ccRCC)
• Intervention / Treatment: Biological: KSD-201

Detailed Description

This study is a single-center, single-arm, open-label, dose-confirmation clinical trial designed to evaluate the safety, tolerability, immune response, and preliminary clinical efficacy of KSD-201 in the treatment of patients with advanced clear cell renal cell carcinoma. The study is conducted in two phases: the first phase is for dose-limiting toxicity (DLT) observation and dose confirmation; the second phase is an expansion study, where the investigators may add 3 to 6 subjects based on the safety and efficacy signals obtained from the first phase.

Enrolled subjects will undergo peripheral blood mononuclear cell collection. The collected material is transported via cold chain logistics to the dendritic cell vaccine preparation site, where the dendritic cell vaccine (KSD-201) is prepared using the subject's own monocytes. Once KSD-201 is successfully prepared, subjects will return to the hospital for KSD-201 injection according to the following method.

1. KSD-201 treatment dose: The provisional dosing is set at 5.0 × 10^6 cells/dose.
2. KSD-201 treatment frequency: a total of 3-5 times. The 4th and 5th times are booster treatments, which need to be decided by the investigator according to the condition of the subjects.
3. Dosing interval: 2 weeks. The interval may be adjusted based on the subject's condition during the intensive treatment phase.
4. KSD-201 route of administration: subcutaneous injection.
5. Administration site: near the axillary lymph node or inguinal lymph node.

• Study Type: Interventional
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430000
      • Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 1. Subjects and/or their legal guardians agree to participate and sign the ICF. 2. Male or female subjects aged 18 years and above on the day of ICF signing. 3. Patients with advanced clear cell renal cell carcinoma, confirmed by pathological tissue examination, who have failed standard therapy (failure of standard therapy refers to disease progression confirmed by imaging during or after at least one systemic regimen, or intolerance to such regimen, which includes tyrosine kinase inhibitors such as sunitinib, axitinib, pazopanib, sorafenib, among others, as well as everolimus and immune checkpoint inhibitors), and for whom no effective treatment measures are available, with the condition posing a serious threat to their lives.

  4. According to RECIST v1.1 criteria, there should be at least one measurable lesion.

  5. Eastern Cooperative Oncology Group (ECOG) score of 0-1. 6. Eligible for leukapheresis and has no other contraindications for cell collection.

  7. Must have adequate organ function：

  • Hematology test: Monocyte count ≥ 0.1 × 10^9/L, neutrophil count ≥ 1.5 × 10^9/L, hemoglobin ≥ 90 g/L, platelet count ≥ 100 × 10^9/L， and no blood transfusion or blood products within 28 days prior to leukapheresis, and no use of hematopoietic growth factors or other drugs to correct blood cells.
  • Liver function: ALT, AST ≤ 2.5 × ULN and TBIL ≤ 1.5 × ULN [for patients with liver metastases: ALT, AST ≤ 5 × ULN and TBIL ≤ 2 × ULN]
  • Kidney function: Creatinine ≤ 1.5 × ULN
  • Cardiac function: Left ventricular ejection fraction (LVEF) ≥ 50%
  • pulmonary function：Pulse oximetry saturation ≥ 94%.

  • Coagulation function: Fibrinogen ≥ 1.0 g/L, activated partial thromboplastin time (APTT) ≤ 1.5 × ULN, prothrombin time (PT) ≤ 1.5 × ULN 8. Patient's corresponding lymph node regions meet the requirements for subcutaneous injection.

    9. Male and female subjects of reproductive age agree to take non-pharmacological contraceptive measures from signing the ICF until 6 months after the last dose.

Exclusion Criteria:

• 1. Within 4 weeks prior to apheresis, patients have received any chemotherapy, immunosuppressive drugs, or other medicinal treatments, or have undergone less than 5 drug half-lives (whichever is shorter), and within 2 weeks prior to apheresis, they have undergone systemic radiation therapy.

  2. Underwent allogeneic transplantation prior to enrollment. 3. Received (attenuated) live vaccines within 4 weeks prior to enrollment. 4. Participated in other clinical studies within 4 weeks prior to enrollment and received at least one dose of the investigational product.

  5. Underwent therapeutic surgery within 4 weeks prior to enrollment, or plan to undergo major surgery during the study, except diagnostic, biopsy and drainage procedures.

  6. Presence of uncontrolled infectious disease within 4 weeks prior to enrollment.

  7. Receiving systemic corticosteroid therapy prior to screening and require long-term systemic corticosteroids during the treatment period (except inhalation or topical application) as judged by the investigator, or received systemic corticosteroid therapy (except inhalation or topical application) within 72 hours prior to administration.

  8. Active central nervous system metastases/lesions (e.g., brain edema requiring hormone intervention or brain metastases).

  9. Severe cardiovascular diseases:

  • Grade ≥ 3 cardiovascular diseases according to the New York Heart Association (NYHA) classification within 6 months prior to enrollment.
  • Unstable angina or severe arrhythmias requiring medication.

  • Other significant ECG abnormalities, including second-degree type 2 atrioventricular block, third-degree atrioventricular block, bradycardia (ventricular rate < 50 beats/min with clinical symptoms), etc.

    10. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and increased peripheral blood hepatitis B virus (HBV) DNA titer above the ULN; positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody; positive for syphilis-specific antibodies.

    11. Have not recovered to normal or Grade ≤ 1 from prior treatment-induced AEs prior to enrollment, except alopecia (any grade) and peripheral neuropathy (Grade ≤ 2).

    12. Other active malignancies within the past 3 years, except for curable cancers that have been markedly cured, such as basal or squamous cell carcinoma, cervical or breast cancer in situ.

    13. Prior history of severe drug allergies or history of penicillin allergy. 14. Substance abuse/addiction. 15. Women who are pregnant or nursing. 16. Other serious medical conditions, including liver disease, kidney disease, neurological/psychiatric disorders, endocrine disorders, hematologic disorders, and immune system disorders, which will render a subject unsuitable for participation in the study as judged by the investigator.

    17. Other conditions that render a subject unsuitable for enrollment as judged by the investigator.

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: KSD-201; Biological: Dendritic Cell Vaccine.Autologous monocyte-derived DCs pulsed with clear cell renal cell carcinoma-associated antigen.	Biological: KSD-201; Patients will receive approximately 5x10^6 cells/dose DC vaccine via subcutaneous injections bi-weekly,total 3-5 times.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety endpoint	Adverse events will be graded according to the NCI-CTCAE 5.0 grading criteria throughout the study period, except for injection site (localized) adverse events, which will be graded with reference to the Guidelines for Grading Criteria for Adverse Events in Clinical Trials of Vaccines for Prophylaxis. Monitor and assess the incidence and relevance to study drug and severity of all adverse events, vital signs, physical examination and laboratory findings.	1 year after DC Vaccines injection
DLT	Incidence and number of dose-limiting toxicities	From the infusion (Day 0) to Day 28

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate (ORR)	The percentage of participants who achieved PR or better response	1 year after DC Vaccines injection
Disease control rate (DCR)	The percentage of participants who achieved SD or better response	1 year after DC Vaccines injection
Duration of response (DOR)	DOR will be calculated among responders (with a PR or better response) from the date of initial documentation of a response (PR or better) to the date of first documented evidence of progressive disease	1 year after DC Vaccines injection
Progression-free survival (PFS)	The time from the start of CAR-GPRC5D treatment for the participants to the first time of disease progression or death for any reason	1 year after DC Vaccines injection
Overall survival (OS)	OS is measured from the date of the initial injection of DC Vaccines to the date of the participant's death	1 year after DC Vaccines injection
Levels of various lymphocyte subsets	total T lymphocytes, activated T lymphocytes, helper T lymphocytes, cytotoxic T lymphocytes, regulatory T lymphocytes, total B lymphocytes and total NK cells in peripheral blood will be assessed to monitor changes	1 year after DC Vaccines injection
EQ-5D-5L	The change from baseline in scores of EQ-5D-5L endpoints will be analyzed by age group or indication group	Up to 1 year
QLQ-C30	The change from baseline in scores of QLQ-C30 endpoints will be analyzed by age group or indication group	Up to 1 year

Collaborators and Investigators

• Sponsor: Kousai Bio Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2026
• Primary Completion (Actual): March 17, 2026
• Study Completion (Actual): March 17, 2026

Study Registration Dates

• First Submitted: November 25, 2024
• First Submitted That Met QC Criteria: November 25, 2024
• First Posted (Actual): November 29, 2024

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 5, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Urologic Neoplasms; Carcinoma; Kidney Neoplasms; Carcinoma, Renal Cell
• Other Study ID Numbers: KSD-201-CR001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No