Golidocitinib for Refractory Immune-related Hematologic Toxicities of Advanced Lung Cancer

NCT07303881 | Not Yet Recruiting Phase 2

• 1 other identifier: ES-2025-244-02
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a single-arm clinical trial designed to evaluate the safety and efficacy of golidocitinib in patients with refractory, immune-related hematologic toxicity in advanced lung cancer.

Conditions

Lung Cancer (Locally Advanced or Metastatic); IrAE; Hematologic Disorder

Keywords

Immune-related hematologic toxicities; irAE; Lung cancer

Outcome Measures

Primary Outcomes (1)

• Clinical remission rate of immune-related hematologic toxicities

  The proportion of subjects who achieved clinical remission of immune-related hematologic toxicities treated with golidocitinib (clinical remission is defined as a degraded hematologic toxicity grade within 1 week of golidocitinib use, a hematologic toxicity grade ≤1 within 2 weeks, and no relapse before discontinuation of treatment). This was assessed by the investigator based on laboratory indicators and the subject's clinical condition.

  12 weeks

Secondary Outcomes (4)

• Negative conversion rate of SSA/Ro52 antibody

  12 weeks

• Progression free survival

  2 years

• Overall survival

  2 years

• Adverse events

  12 weeks

Study Arms (1)

golidocitinib

EXPERIMENTAL

Based on the BOIN dose escalation and Simon two-stage efficacy assessment design, if the dose of golidocitinib used in Part B is 75 mg, the total number of subjects to be included in Parts A and B is 10; if the dose of golidocitinib used in Part B is 150 mg, the total number of subjects to be included in Parts A and B is 13-16.

Drug: golidocitinib

Interventions

golidocitinib DRUG

Golidocitinib treatment can be discontinued once clinical remission is achieved, with a maximum maintenance period of 12 weeks.

golidocitinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to provide a signed and dated informed consent form, including compliance with the Informed Consent Form (ICF) and the requirements and limitations listed in this protocol.
• The subject is ≥18 years of age at the time of signing the ICF.
• Has not experienced disease progression in the past two weeks and has a Eastern Cooperative Oncology Group (ECOG) score of 0-2, with a predicted survival of ≥12 weeks.
• Pathologically or cytologically confirmed locally advanced (American Joint Committee on Cancer, AJCC 8th edition IIIB and IIIC stages) or metastatic (stage IV) non-small cell lung cancer, or extensive-stage small cell lung cancer (AJCC 8th edition TNM staging of lung cancer stage IV, or T3-4 disease caused by multiple pulmonary nodules with excessive disease spread, or tumor/nodule size too large for a tolerable radiation therapy plan).
• Patients confirmed by an accredited local laboratory to lack any therapeutically targeted driver gene mutations, i.e., driver gene-negative subjects.
• Patients experiencing grade ≥3 refractory hematologic toxicity (white blood cell count \<2.0 × 10⁹/L or/and absolute neutrophil count \<1.0 × 10⁹/L or/and platelet count \<50 × 10⁹/L or/and hemoglobin \<8.0 g/dL, specific hematologic parameters are detailed in Appendix D), during treatment with immune checkpoint inhibitors (including monotherapy or combination therapy), and the toxicity is considered to be immune-related. Refractory toxicity is defined in two main ways: ① Resistance to conventional treatment: No significant improvement in hematologic toxicity after at least 3 days of supportive care including the use of hormones (e.g., methylprednisolone ≥1 mg/kg/d), hematopoietic growth factors (e.g., G-CSF, TPO), and/or blood transfusions; ② Positive antinuclear antibody profile indicating anti-SSA antibodies and/or Ro52 antibodies, suggesting an immune-mediated mechanism.
• Patients with brain metastases must be asymptomatic or have been treated and have stable disease after discontinuation of steroids and anticonvulsants. Patients suspected of having brain metastases at screening should undergo brain CT/MRI before study enrollment.
• At least one measurable lesion (as defined in RECIST 1.1): a lesion that has not undergone radiotherapy, has a long diameter ≥10 mm (short diameter ≥15 mm for lymph node lesions), and can be accurately and repeatedly measured from baseline on CT or MRI; and a measurable lesion outside the central nervous system.
• Adequate organ system functional reserve, summarized as follows:
• Total bilirubin ≤1.5×ULN; if Gilbert's syndrome (unconjugated hyperbilirubinemia) is present, total bilirubin should be ≤3×ULN.
• ALT and AST ≤2.5×ULN. For patients with documented liver metastases, AST and ALT levels ≤5×ULN.
• Creatinine clearance, calculated using the Cockcroft-Gault method, \>60 ml/min for patients treated with cisplatin and \>45 ml/min for patients treated with carboplatin.
• Urinalysis shows less than 2+ protein in urine, or 24-hour urine protein quantification \<1g.
• Good coagulation function, defined as International Normalized Ratio (INR) and/or Prothrombin Time (PT) ≤1.5 times the ULN and/or Activated Partial Thromboplastin Time (APTT) ≤1.5 of the upper limit of normal; if the subject is receiving anticoagulation therapy, PT is acceptable as long as it is within the range intended for use with the anticoagulant.
• Serum amylase ≤1.5 times the ULN and/or serum lipase ≤1.5 times the ULN.

You may not qualify if:

• a. An active autoimmune disease requiring systemic treatment (e.g., use of disease-modifying medications, corticosteroids, or immunosuppressants) within 2 years prior to the first dose. Replacement therapies (e.g., thyroxine, insulin, or physiological corticosteroids for adrenal or pituitary insufficiency) are not considered systemic treatment.
• b. Prior to the first dose, any other form of immunosuppressive therapy other than corticosteroids (e.g., TNF-α inhibitors, mycophenolate mofetil, gamma globulin, rituximab, other JAK inhibitors, etc.) was received for hematologic toxicity.
• c. Spinal cord compression or meningeal metastases are present. d. Any of the following medical histories:
• Currently participating in an interventional clinical trial, or having received other investigational drugs or used investigational devices within 4 weeks prior to the first dose; any drug still in development requires a 5-half-life washout (or discussion with the research team);
• Underwent major surgery (excluding diagnostic or biopsy, excluding vascular access) within 4 weeks prior to the first dose, or is expected to undergo major surgery during the study;
• Received palliative radiation therapy within 2 weeks prior to the first dose;
• Have experienced a serious arterial/venous thrombotic event within 6 months prior to the first dose, including cerebrovascular accidents (e.g., history of stroke or intracranial hemorrhage), deep vein thrombosis, and pulmonary embolism;
• Currently receiving (or unable to discontinue at least 1 week prior to the first dose) any known potent inducer or inhibitor of CYP3A, herbal supplements, or foods;
• Have experienced a grade CTCAE \> 1 adverse event (excluding any degree of alopecia and hematologic toxicity) due to prior treatment prior to the first dose. e. Has received a solid organ or blood system transplant (e.g., a previous allogeneic bone marrow transplant).
• f. Has a history of interstitial lung disease, drug-induced interstitial lung disease, radiation pneumonitis requiring corticosteroid therapy, or currently has active interstitial lung disease (including interstitial lung changes), or immune-mediated pneumonitis caused by immunotherapy.
• g. Has been diagnosed with another malignancy within 5 years prior to the first dose, excluding clinically cured basal cell carcinoma, squamous cell carcinoma, and/or radically resected carcinoma in situ.
• h. Has received a live vaccine, including attenuated live vaccines, excluding inactivated vaccines, within 30 days prior to the first dose.
• i. Has known active tuberculosis, such as a positive tuberculin (PPD) test (induration diameter \> 10 mm), a positive T-SPOT test, tuberculous lesions on chest X-ray/CT, or other positive results found according to routine clinical screening (excluding those cured by investigator assessment after standard anti-tuberculosis treatment).
• j. Subjects with pre-existing, uncontrollable severe infectious diseases must be excluded. If an infectious disease occurred within two months prior to the first dose, its control must be assessed by the research team to determine eligibility for enrollment.
• k. Subjects with active infections, including but not limited to hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV) (see table below), and active COVID-19 infection (determined by the investigator to be clinically significant, with signs or symptoms). COVID-19 testing will be based on local practice.

Sponsors & Collaborators

• The First Affiliated Hospital of Guangzhou Medical University: lead

Study Sites (1)

First Affiliated Hospital of Guangzhou Medical College

Guangzhou, Guangdong, China

Location

MeSH Terms

Conditions

Hematologic Diseases; Lung Neoplasms; Neoplasm Metastasis

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Wenhua Liang, MD, PhD

CONTACT

86-20-83337792; 550627660@qq.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Head of Thoracic Surgery, Principal Investigator, Clinical Professor

Study Record Dates

• First Submitted: December 12, 2025
• First Posted: December 26, 2025
• Study Start: February 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: December 26, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)