Registry of an Integrated Longitudinal Multimodal Biospecimen Biobank for Primary CNS Lymphoma in China
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-100
SponsorXiaohui Ren
About this trial
This project aims to establish and prospectively register a longitudinal, multimodal biobank for primary central nervous system lymphoma (PCNSL). The study will primarily enroll patients undergoing needle biopsy or surgical resection of lesions involving the brain, spinal cord, or meninges, with a pathological diagnosis of central nervous system lymphoma. To ensure population homogeneity, patients with PCNSL will constitute the core analysis cohort. Patients with imaging findings suggestive of PCNSL but a final pathological diagnosis other than PCNSL will serve as disease controls, and matched healthy volunteers will be recruited as baseline controls.
At baseline, biopsy tissue, cerebrospinal fluid (CSF), peripheral blood, urine, saliva, and stool samples will be collected. Follow-up visits will be scheduled at 1 month after biopsy and every 3-6 months thereafter, with contrast-enhanced brain magnetic resonance imaging (MRI) and serial collection of CSF and peripheral blood. After the requirements of routine pathological diagnosis have been met, biopsy specimens will undergo tiered multi-omics profiling according to sample availability and freshness, including whole-exome sequencing, bulk RNA sequencing, single-cell RNA sequencing, metabolomics, spatial transcriptomics, and spatial metabolomics. Analyses of CSF and blood samples will focus on circulating tumor DNA and cell-free RNA (ctDNA/cfRNA), supplemented by conventional cytology, flow cytometry, and other clinical data for longitudinal disease monitoring.
The project will develop an integrated database incorporating clinical, imaging, pathological, molecular, and biospecimen data. It will characterize the relationships of baseline tissue multi-omics features and longitudinal changes in CSF- and blood-derived ctDNA/cfRNA with treatment response, recurrence prediction, and prognosis. The study will also develop diagnostic and risk-stratification models applicable to patients at our institution, across the region, and potentially throughout mainland China, while establishing standardized operating procedures for biospecimen collection and processing. This work is expected to provide high-quality evidence and a sustainable translational research platform for elucidating the biological heterogeneity of PCNSL, validating liquid-biopsy biomarkers, and optimizing precision follow-up strategies.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
General Criteria for Patient Participants
Age ≥18 years.
Radiological findings suspicious for primary central nervous system lymphoma (PCNSL).
Scheduled to undergo needle biopsy or surgical resection of a lesion involving the brain, spinal cord, or meninges.
Disqualifiers
Exclusion Criteria for Patient Cohorts
PCNSL is suspected based on imaging findings, but no definitive histopathological diagnosis can be obtained.
Estimated life expectancy of less than 3 months and, in the investigator's judgment, inability to complete the minimum required clinical data or biospecimen collection.
Refusal to provide written informed consent or withdrawal of informed consent.
Trial population
The study population will be drawn from patients evaluated for suspected primary central nervous system lymphoma at participating tertiary referral hospitals in mainland China, including Beijing Tiantan Hospital, Shandong Provincial Hospital, and Tianjin Huanhu Hospital. Participants will be identified through departments of neurosurgery, hematology, neuro-oncology, radiology, and pathology. Healthy controls will be recruited from the participating institutions and surrounding communities to provide reference biospecimens and molecular profiles. The study will include a core PCNSL cohort, a non-PCNSL disease-control cohort, and a nested longitudinal cerebrospinal fluid/peripheral blood subcohort.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
High-dose methotrexate-based therapy
DrugStandard-of-care treatment containing high-dose methotrexate as the backbone regimen, administered alone or in combination with other anticancer agents according to the treating physician's judgment and institutional practice. Treatment selection, dose, schedule, combination regimen, and duration are not assigned by the study protocol. Clinical response, recurrence, survival, adverse events, imaging findings, and longitudinal molecular biomarkers will be recorded prospectively.
Bruton tyrosine kinase inhibitor-based therapy
DrugStandard-of-care treatment containing a Bruton tyrosine kinase inhibitor as a principal therapeutic component, administered alone or in combination with other anticancer agents according to the treating physician's judgment and institutional practice. The specific BTK inhibitor, dose, schedule, combination regimen, and duration are not assigned by the study protocol. Clinical response, recurrence, survival, adverse events, imaging findings, and longitudinal molecular biomarkers will be recorded prospectively.
Treatment groups
400 Participants
are divided into 1 treatment groupGroup A: PCNSL Cohort2 interventions
Trial outcomes
Primary outcomes
1
Baseline data completeness rate
Percentage of enrolled participants with complete required baseline clinical, imaging, pathological, treatment, and biospecimen information in the electronic case report form. The numerator is the number of participants with all protocol-required baseline data; the denominator is the total number of enrolled participants.
Time frame
At baseline, from enrollment through completion of baseline assessments（up to 30 months）2
Paired tumor tissue and cerebrospinal fluid sample acquisition rate
Percentage of eligible participants from whom both evaluable tumor tissue and cerebrospinal fluid samples are successfully collected and stored according to the study standard operating procedures.
Time frame
within 1 month after biopsy or surgery (up to 30 months)3
Participant follow-up completion rate
Percentage of enrolled participants who complete the protocol-required clinical and imaging follow-up assessments at Month 12 and Month 24. Completion rates will be calculated separately for each time point.
Time frame
from enrollment to follow-up at Month 3, 6, 12, 24 and 36 (up to 36 months)4
Adherence to longitudinal cerebrospinal fluid sampling
Percentage of scheduled cerebrospinal fluid collection visits completed among participants enrolled in the longitudinal cerebrospinal fluid/peripheral blood subcohort.
Time frame
Postoperative Month 1 and every 3-6 months thereafter (up to 36 months)Secondary outcomes
1
Objective response rate
Percentage of evaluable participants achieving complete response or partial response on MRI, summarized according to prespecified baseline tumor multi-omics characteristics.
Time frame
From treatment initiation to follow-up at month 3,6,12,24 and 36 (up to 36 months)2
Best overall response
Best MRI response recorded during follow-up-complete response, partial response, stable disease, or progressive disease-summarized according to baseline tumor multi-omics characteristics.
Time frame
From treatment initiation to follow-up at month 3,6,12,24 and 36 (up to 36 months)3
Progression-free survival
Time from the prespecified index date to radiographic or clinical progression or death from any cause, whichever occurs first, analyzed according to baseline tumor multi-omics characteristics.
Time frame
From treatment initiation to follow-up at month 3,6,12,24 and 36 (up to 36 months)4
Overall survival
Time from the prespecified index date to death from any cause. Participants without a documented death will be censored at the date they were last known to be alive.
Time frame
From treatment initiation to follow-up at month 3,6,12,24 and 36 (up to 36 months)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Xiaohui Ren
Lead sponsor
Beijing Tiantan Hospital
Sponsor institution
Shandong Provincial Hospital
Collaborator
Tianjin Huanhu Hospital
Collaborator
Source ClinicalTrials.gov