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Not yet recruitingNCT06692491SPRINGUpdated Nov 18, 2024

Study of Precision Treatment for Rare Tumours in China Guided by PDO and NGS

A Phase 2 interventional study of Albumin-Bound Paclitaxel and Epirubicin in Rare Tumour, sponsored by Peking University Shenzhen Hospital. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-11-18.

Sponsored by Peking University Shenzhen Hospital · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
200
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

The objective of this Phase II, open-label, multicenter, non-randomised controlled clinical trial is to guide precision treatment for patients with rare tumours based on Patient-Derived Organoids/Next-Generation Sequencing drug screening.

Read the detailed description

Rare tumours, defined as those with an annual incidence rate of less than 2.5/100,000 according to the data from the National Cancer Registry of the National Cancer Center of China, represent a significant unmet medical need due to the lack of high-level evidence-based clinical practice guidelines and standard regimens. Patient-Derived Organoids(PDOs), which preserves the histologic and genetic characteristics of patients; tumours, have demonstrated predictive value of clinical outcomes. Prior studies have shown that PDOs-based drug screens can predict treatment response with high sensitivity and specificity.The investigators thus propose the study: A Phase II Clinical Study on Precision Treatment of Rare Tumours in China Guided by Patient-Derived Organoids and Next-Generation Sequencing. Investigators aim to determine clinical efficacy of PDOs/NGS-guided precision treatment in patients with rare tumours. After tumour samples were collected, drug screening using PDOs and NGS will be conducted. Molecular Tumor board(MTB) will review the results and develop an individualised treatment regimens. Primary endpoint of this study is objective response rate (ORR). Secondary endpoints include disease control rate (DCR), progression-free survival (PFS), overall survival (OS) and duration of response (DoR).

02

Conditions studied

  • Rare Tumour

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Keywords

  • Rare Tumour
  • Patient-Derived Organoids (PDO)
  • Next-Gneration Sequencing (NGS)
  • Precision Treatment
03

In context

Neoplasms

9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.

This study's planned enrollment of 200 is above the median of 50 across 7,253 interventional studies indexed under Neoplasms.

Browse Neoplasms studies →

Lead sponsor

Peking University Shenzhen Hospital is the lead sponsor of 17 studies on the registry; 12 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female, the age at the time of signing the informed consent is no less than 18 years old;
  2. Patients with advanced or metastatic rare solid tumor confirmed by histological confirmed;
  3. ECOG score is 0 or 1;
  4. Expected survival ≥12 weeks;
  5. According to Response Evaluation Criteria in Solid Tumor (RECIST 1.1), there is at least one imaging measurable lesions, which has not previously undergone radiotherapy or with obvious disease progress after radiotherapy;
  6. Surgical specimens of paired tumor tissue and adjacent normal tissue, or fresh biopsy tissue samples or ≥500ml serous cavity effusion with positive pathological cytological examination must be provided. These specimens must be obtained without prior exposure to any other anti-tumor treatment, systemic anti-infection treatment, vaccination, et al. and are intended for organoid culture;
  7. Must have a primary or metastatic paraffin-embedded tissue (without radiotherapy) other than bone metastatic lesions before enrollment (within 2 years, 15-20 sheets, 4-6μm thick white slices, of which 5 need to be glued and baked ) and peripheral blood samples for NGS gene testing. If NGS results are already available but sufficient paraffin-embedded tissue cannot be provided, investigator are allowed the decision to enroll subjects according to the specific situation;
  8. In the condition that the primary lesions biopsy specimen has been provided, if the metastatic lesion is able to be biopsied(determined by investigator), it is suggested to keep the specimen for pathological testing and provide fresh tissue specimen;
  9. After the progression of the subject\'s disease, if conditions permit(determined by investigator), fresh tissue samples shall be obtained from the same biopsy lesions and the metastasis lesions of the previously obtained samples;
  10. Toxic and side effects caused by previous treatment need to be restored to ≤ Grade 1 or returned to the baseline value (NCI-CTCAE version 5.0, except for hair loss);
  11. Negative pregnancy test (only applicable for women with childbearing potential). No childbearing potential is defined as being postmenopausal for longer than one year or having undergone surgical sterilization or hysterectomy;
  12. All patients (male and female) agree to use an effective form of contraceptive measures during the treatment and within 8 weeks after the end of treatment;
  13. Signed, written informed consent of volunteers that join the group shall follow the trial treatment plan, follow-up plan and cooperate to observe the adverse events and efficacy.

Exclusion criteria

Exclusion Criteria:

  1. No patient lesions available for organoid model construction;
  2. History of interstitial lung disease or radiation pneumonitis of any type;
  3. Central Nervous System (CNS) metastases with brain metastases-related symptoms, which is not stable in neurology, or need to increase steroid dosage to control CNS disease. (Note: Patients with controlled CNS metastasis are eligible to participate in this study);
  4. Current uncontrollable third cavity effusion, such as a large amount of pleural effusion，ascites , or pericardial effusion;
  5. Major surgical operations or incomplete healing of injury within 4 weeks prior to study treatment\'s first administration and chest radiotherapy of > 30 Gy within 6 months;
  6. History of receiving other investigational drugs within 14 days or 5 half-lives (whichever is longer) prior to the first administration;
  7. History of receiving live vaccine within 30 days prior to the first administration. Seasonal influenza vaccines that do not contain live viruses are allowed;

  8. Current active infection requiring systemic treatment (antibiotics); or any of the following:

    1. HIV positive or known history of acquired immunodeficiency syndrome;
    2. Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection is defined as HBsAg positive and the number of HBV DNA copies exceeds the upper limit of normal value, or HCV AB positive;
    3. Active tuberculosis (with exposure history or positive tuberculosis test; with clinical and / or imaging manifestations);
    4. Positive antibody of Treponema Pallidum
  9. Current evidenced uncontrollable systemic diseases (such as severe mental, neurological, epilepsy or dementia, unstable or uncompensated respiratory, cardiovascular, liver or kidney diseases, uncontrolled hypertension [still greater than or equal to CTCAE Grade 3 hypertension after drug treatment]);
  10. History of myocardial infarction, coronary artery / peripheral artery bypass or cerebrovascular accident within 3 months;
  11. Diagnosed with a second type of malignant tumor within 5 years before the first diagnosis of a rare solid tumor (excluding completely resected basal cell carcinoma, bladder carcinoma in situ, cervical carcinoma in situ);
  12. History of receiving of any organ transplantation, including allogeneic stem cell transplantation. Transplantation without immunosuppression (corneal transplantation, hair transplantation) is excluded;

  13. Cardiovascular disease or symptom includes any of the following:

    1. History of Congestive Heart Failure requiring treatment and of New York Heart Association class III / IV CHF (see Appendix 3) ;
    2. Current ventricular arrhythmia requiring antiarrhythmic drugs treatment, or uncontrollable or unstable arrhythmia;
    3. Severe conduction disorder (such as grade II or III AV block);
    4. Angina requiring treatment;
    5. QT interval (QTC) of 12 lead ECG is ≥ 450 ms in male and ≥ 470 MS in female;
    6. History of congenital long QT syndrome, congenital short QT syndrome, torsade de pointe or pre-excitation syndrome;
    7. History of LVEF decline to below 50% determined by echocardiography or MUGA scan;
    8. History of myocardial infarction in the past 6 months.
  14. Inadequate bone marrow reserve or organ function;
  15. Pregnant or lactating women;
  16. History of swallowing dysfunction, active gastrointestinal disease or other diseases that significantly affect the absorption, distribution, metabolism and excretion of oral drugs. The patients with history of subtotal gastrectomy. (Note: this standard is not applicable to the sub schemes with the investigational drug as injection).
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
200 participants (estimated)

Study arms

  • No intervention
    Standard treatment Group

    Patients with rare tumours in this group will undergo standard treatment in accordance with the leading domestic and international guidelines (NCCN/ESMO/CSCO/CACA) and clinical trial.

  • Experimental
    Criteria-Fulfilled ICF Group

    Intervention in this group is to perform PDOs culture and drug screening of lesions from patients with rare tumours who failed to standard treatment and fully meet the inclusion and exclusion criteria. The MTB will review the results of the drug screening and NGS, and subsequently determine the personalised chemotherapy, targeted or immunological agents.

    Drug: Albumin-Bound Paclitaxel · Drug: Epirubicin · Drug: Gemcitabine · Drug: Vinorelbine · Drug: Cisplatin · Drug: Irinotecan · Drug: Fluorouracil · Drug: Nivolumab · Drug: Pembrolizumab · Drug: Durvalumab · Drug: Atezolizumab · Drug: Sintilimab · Drug: Tislelizumab · Drug: Camrelizumab · Drug: Toripalimab · Drug: Serplulimab · Drug: Adebrelimab · Drug: Envafolimab · Drug: Osimertinib · Drug: Alectinib · Drug: Vemurafenib · Drug: Pamiparib · Drug: Pyrotinib · Drug: Imatinib · Drug: Palbociclib · Drug: Savolitinib · Drug: Entrectinib

  • Experimental
    Compassionate-Use ICF Group

    Patients with rare tumours who failed to standard treatment and do not meet the requisite criteria (e.g. active hepatitis B, underlying disease in the exclusion criteria, symptomatic brain metastases) will provide informed consent and undergo PDOs culture and drug screening. The MTB will review the results of the drug screening and NGS, and subsequently determine the personalised chemotherapy, targeted or immunological agents.

    Drug: Albumin-Bound Paclitaxel · Drug: Epirubicin · Drug: Gemcitabine · Drug: Vinorelbine · Drug: Cisplatin · Drug: Irinotecan · Drug: Fluorouracil · Drug: Nivolumab · Drug: Pembrolizumab · Drug: Durvalumab · Drug: Atezolizumab · Drug: Sintilimab · Drug: Tislelizumab · Drug: Camrelizumab · Drug: Toripalimab · Drug: Serplulimab · Drug: Adebrelimab · Drug: Envafolimab · Drug: Osimertinib · Drug: Alectinib · Drug: Vemurafenib · Drug: Pamiparib · Drug: Pyrotinib · Drug: Imatinib · Drug: Palbociclib · Drug: Savolitinib · Drug: Entrectinib

  • Experimental
    RWS ICF Group

    Patients with rare tumours who failed to standard treatment will be included in a real-world study cohort and receive clinically conventional treatment if they do not undergo PDOs culture and NGS testing successfully or are unwilling to base their treatment on the results of PDOs and NGS for various reasons.

    Drug: Other

Interventions

  • DrugAlbumin-Bound Paclitaxel

    Albumin-Bound Paclitaxel 260mg/m2,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with albumin-bound paclitaxel if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Abraxane

  • DrugEpirubicin

    Epirubicin 90mg/m2,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with epirubicin if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Ellence

  • DrugGemcitabine

    Gemcitabine 1000mg/m2,D1,D8,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with gemcitabine if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Gemzar

  • DrugVinorelbine

    Vinorelbine 25-30mg/m2,D1,D8,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with vinorelbine if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Navelbine, Vumon

  • DrugCisplatin

    Cisplatin 90mg/m2,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with cisplatin if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Platinol

  • DrugIrinotecan

    Irinotecan 125mg/m2,D1,D8,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with irinotecan if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Camptosar

  • DrugFluorouracil

    Fluorouracil 600mg/m2,D1-5,q3w Patients with advanced rare tumours who failed to standard treatment will be administrated with fluorouracil if PDOs show the highest response to this drug under the guidance of MTB.

    Also known as: Adrucil, 5-FU

  • DrugNivolumab

    Nivolumab 3mg/kg,q2w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with nivolumab under the guidance of MTB.

    Also known as: Opdivo

  • DrugPembrolizumab

    Pembrolizumab 200mg,q2w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with pembrolizumab under the guidance of MTB.

    Also known as: Keytruda

  • DrugDurvalumab

    Durvalumab 10mg/kg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with durvalumab under the guidance of MTB.

    Also known as: Imfinzi

  • DrugAtezolizumab

    Atezolizumab 1200mg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with atezolizumab under the guidance of MTB.

    Also known as: Tecentriq

  • DrugSintilimab

    Sintilimab 200mg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with sintilimab under the guidance of MTB.

    Also known as: Tyvyt

  • DrugTislelizumab

    Tislelizumab 200mg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with tislelizumab under the guidance of MTB.

    Also known as: Baizean

  • DrugCamrelizumab

    Camrelizumab 200mg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with camrelizumab under the guidance of MTB.

    Also known as: AiRuiKa

  • DrugToripalimab

    Toripalimab 240kg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with toripalimab under the guidance of MTB.

    Also known as: Tuoyi

  • DrugSerplulimab

    Serplulimab 3mg/kg,q2w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with serplulimab under the guidance of MTB.

    Also known as: Hansizhuang

  • DrugAdebrelimab

    Adebrelimab 20mg/kg,q3w Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with adebrelimab under the guidance of MTB.

    Also known as: Abesive

  • DrugEnvafolimab

    Envafolimab 150mg,qw Patients with advanced rare tumours who failed to standard treatment carrying no targeted alterations will be administrated with envafolimab under the guidance of MTB.

    Also known as: Epkinly

  • DrugOsimertinib

    Osimertinib 80mg,qd Patients with advanced rare tumours who failed to standard treatment carrying EGFR mutations will be administrated with osimertinib under the guidance of MTB.

    Also known as: Tagrisso

  • DrugAlectinib

    Alectinib 600mg,bid Patients with advanced rare tumours who failed to standard treatment carrying ALK fusion will be administrated with alectinib under the guidance of MTB.

    Also known as: Alecensa

  • DrugVemurafenib

    Vemurafenib 960mg,bid Patients with advanced rare tumours who failed to standard treatment carrying BRAF V600E mutation will be administrated with vemurafenib under the guidance of MTB.

    Also known as: Zelboraf

  • DrugPamiparib

    Pamiparib 60mg,bid Patients with advanced rare tumours who failed to standard treatment carrying BRCA1/2 mutation will be administrated with pamiparib under the guidance of MTB.

    Also known as: Balversa

  • DrugPyrotinib

    Pyrotinib 400mg, qd Patients with advanced rare tumours who failed to standard treatment carrying HER-2 mutation or HER-2 over expression/amplification will be administrated with Pyrotinib under the guidance of MTB.

    Also known as: Nerlynx

  • DrugImatinib

    Imatinib 400mg, qd Patients with advanced rare tumours who failed to standard treatment carrying CKIT mutation will be administrated with imatinib under the guidance of MTB.

    Also known as: Gleevec

  • DrugPalbociclib

    Palbociclib 125mg, 21/7 dosing schedule Patients with advanced rare tumours who failed to standard treatment following HR(+)/HER-2(-) will be administrated with palbociclib under the guidance of MTB.

    Also known as: Ibrance

  • DrugSavolitinib

    Savolitinib 300mg, qd Patients with advanced rare tumours who failed to standard treatment carrying C-MET mutation will be administrated with savolitinib under the guidance of MTB.

    Also known as: Orpathys

  • DrugEntrectinib

    Entrectinib 600mg, qd Patients with advanced rare tumours who failed to standard treatment carrying ROS-1 fusion or NTRK fusion will be administrated with entrectinib under the guidance of MTB.

    Also known as: Rozlytrek

  • DrugOther

    Patients with advanced rare tumours who failed to standard treatment in RWS ICF Group will be administrated with empirical chemotherapy(e.g., "Albumin-Bound Paclitaxel", "Epirubicin", "Gemcitabine", "Vinorelbine", "Cisplatin", "Irinotecan", "Fluorouracil"), immunological(e.g., "Nivolumab", "Pembrolizumab", "Durvalumab", "Atezolizumab", "Sintilimab", "Tislelizumab", "Camrelizumab", "Toripalimab", "Serplulimab","Adebrelimab","Envafolimab") or targeted drugsl(e.g., "Osimertinib", "Alectinib", "Vemurafenib", "Vemurafenib", "Pyrotinib", "Imatinib", "Palbociclib", "Savolitinib", "Entrectinib")treatments.

06

What researchers measure

Primary outcomes

  1. Objective Response Rate (ORR)

    The percentage of patients with a confirmed investigator-assessed complete or partial response according to Response Evaluation Criteria In Solid Tumours (RECIST) 1.1.

    Time frame: From enrollment to the end of treatment at 8 weeks

Secondary outcomes

  1. Disease Control Rate (DCR)

    The percentage of evaluable patients who achieve confirmed Complete Response (CR), Partial Response (PR), or Stable Disease (SD) (lasting for ≥ 8 weeks).

    Time frame: From enrollment to the end of treatment at 8 weeks

  2. Progression free survival (PFS)

    Time from the first dose of treatment until the first documentation of disease progression (PD) or death from any cause, whichever occurs first.

    Time frame: From enrollment to the end of treatment at 8 weeks

  3. Overall survival (OS)

    Time from the first dose of treatment until the death of the patient from any cause.

    Time frame: From enrollment to the end of treatment at 8 weeks

  4. Duration of response (DOR)

    Time from the first tumor assessment showing a Complete Response (CR) or Partial Response (PR) to the first occurrence of disease progression (PD) or death before PD in subjects who achieved CR or PR, whichever occurs first

    Time frame: From enrollment to the end of treatment at 8 weeks

07

Study locations

No study locations are listed for this record.

08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 18, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06692491
Lead sponsor
Peking University Shenzhen Hospital
Responsible party
shubin wang (Director of Medical Oncology, Peking University Shenzhen Hospital) — Principal investigator
First posted
Nov 18, 2024
Start date
Jan 1, 2025 (estimated)
Primary completion
Dec 31, 2027 (estimated)
Completion
Dec 31, 2029 (estimated)
Last update
Nov 18, 2024

Study contacts

shubin wang, Doctor
Contact
wangshubin2013@163.com
+86 13823394076
shunbin wang, Doctor
study chair · Peking University Shenzhen Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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