Rare TumourPhase 2SPRINGNCT06692491
Study of Precision Treatment for Rare Tumours in China Guided by PDO and NGS
A Phase 2 study testing Albumin-Bound Paclitaxel, Epirubicin and Gemcitabine in people with Rare Tumour, run by Peking University Shenzhen Hospital.
Trial 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Rare Tumour
- What is being tested?
- Albumin-Bound Paclitaxel, Epirubicin and Gemcitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2025to 31 Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, the age at the time of signing the informed consent is no less than 18 years old;
- Patients with advanced or metastatic rare solid tumor confirmed by histological confirmed;
- ECOG score is 0 or 1;
- Expected survival ≥12 weeks;
- 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;
- 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;
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- 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;
- 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;
- 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;
- 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);
- 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;
- 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;
- 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.
Who cannot join
- No patient lesions available for organoid model construction;
- History of interstitial lung disease or radiation pneumonitis of any type;
- 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);
- Current uncontrollable third cavity effusion, such as a large amount of pleural effusion，ascites , or pericardial effusion;
- 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;
- History of receiving other investigational drugs within 14 days or 5 half-lives (whichever is longer) prior to the first administration;
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- History of receiving live vaccine within 30 days prior to the first administration. Seasonal influenza vaccines that do not contain live viruses are allowed;
- Current active infection requiring systemic treatment (antibiotics); or any of the following:
- HIV positive or known history of acquired immunodeficiency syndrome;
- 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;
- Active tuberculosis (with exposure history or positive tuberculosis test; with clinical and / or imaging manifestations);
- Positive antibody of Treponema Pallidum
- 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]);
- History of myocardial infarction, coronary artery / peripheral artery bypass or cerebrovascular accident within 3 months;
- 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);
- History of receiving of any organ transplantation, including allogeneic stem cell transplantation. Transplantation without immunosuppression (corneal transplantation, hair transplantation) is excluded;
- Cardiovascular disease or symptom includes any of the following:
- History of Congestive Heart Failure requiring treatment and of New York Heart Association class III / IV CHF (see Appendix 3) ;
- Current ventricular arrhythmia requiring antiarrhythmic drugs treatment, or uncontrollable or unstable arrhythmia;
- Severe conduction disorder (such as grade II or III AV block);
- Angina requiring treatment;
- QT interval (QTC) of 12 lead ECG is ≥ 450 ms in male and ≥ 470 MS in female;
- History of congenital long QT syndrome, congenital short QT syndrome, torsade de pointe or pre-excitation syndrome;
- History of LVEF decline to below 50% determined by echocardiography or MUGA scan;
- History of myocardial infarction in the past 6 months.
- Inadequate bone marrow reserve or organ function;
- Pregnant or lactating women;
- 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).
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase II Clinical Study on Precision Treatment of Rare Tumours in China Guided by Patient-Derived Tumor Organoids (PDO) and Next-Generation Sequencing (NGS)
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).
- Conditions
- Rare Tumour
- Being tested
- Albumin-Bound Paclitaxel, Epirubicin, Gemcitabine, Vinorelbine, Cisplatin
- Keywords
- Rare Tumour, Patient-Derived Organoids (PDO), Next-Gneration Sequencing (NGS), Precision Treatment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Albumin-Bound PaclitaxelDrug
Also called Abraxane
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.
EpirubicinDrug
Also called Ellence
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.
GemcitabineDrug
Also called Gemzar
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.
VinorelbineDrug
Also called Navelbine, Vumon
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.
CisplatinDrug
Also called Platinol
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.
IrinotecanDrug
Also called Camptosar
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.
FluorouracilDrug
Also called Adrucil, 5-FU
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.
NivolumabDrug
Also called Opdivo
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.
PembrolizumabDrug
Also called Keytruda
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.
DurvalumabDrug
Also called Imfinzi
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.
AtezolizumabDrug
Also called Tecentriq
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.
SintilimabDrug
Also called Tyvyt
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.
TislelizumabDrug
Also called Baizean
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.
CamrelizumabDrug
Also called AiRuiKa
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.
ToripalimabDrug
Also called Tuoyi
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.
SerplulimabDrug
Also called Hansizhuang
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.
AdebrelimabDrug
Also called Abesive
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.
EnvafolimabDrug
Also called Epkinly
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.
OsimertinibDrug
Also called Tagrisso
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.
AlectinibDrug
Also called Alecensa
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.
VemurafenibDrug
Also called Zelboraf
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.
PamiparibDrug
Also called Balversa
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.
PyrotinibDrug
Also called Nerlynx
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.
ImatinibDrug
Also called Gleevec
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.
PalbociclibDrug
Also called Ibrance
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.
SavolitinibDrug
Also called Orpathys
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.
EntrectinibDrug
Also called Rozlytrek
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.
OtherDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Standard treatment Group | No intervention | 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. |
| Criteria-Fulfilled ICF Group | Experimental | 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. Receives: 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 |
| Compassionate-Use ICF Group | Experimental | 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. Receives: 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 |
| RWS ICF Group | Experimental | 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. Receives: Drug: Other |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From enrollment to the end of treatment at 8 weeks
Secondary outcomes (4)
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).
Measured: From enrollment to the end of treatment at 8 weeks
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.
Measured: From enrollment to the end of treatment at 8 weeks
Overall survival (OS)
Time from the first dose of treatment until the death of the patient from any cause.
Measured: From enrollment to the end of treatment at 8 weeks
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
Measured: From enrollment to the end of treatment at 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2024
- Study start1 Jan 2025 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rare TumourStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University Shenzhen Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, shubin wang, Peking University Shenzhen Hospital
- Investigators
- shunbin wang, Doctor, Study chair, Peking University Shenzhen Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06692491
- Sponsor's protocol ID
- PUShenzhenH RE [2024]No.(117)
- Acronym
- SPRING
- Status last verified
- Nov 2024
- First submitted
- 16 Sep 2024
- Last updated
- 18 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Clinical Study on Precision Treatment of Rare Tumours in China Guided by Patient-Derived Tumor Organoids (PDO) and Next-Generation Sequencing (NGS)
- ClinicalTrials.gov ID
- NCT06692491
- Acronym / sponsor ID
- SPRING / PUShenzhenH RE [2024]No.(117)
- Conditions
- Rare Tumour
- Keywords
- Rare Tumour, Patient-Derived Organoids (PDO), Next-Gneration Sequencing (NGS), Precision Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Peking University Shenzhen HospitalUniversity, hospital or other
- Study start
- 1 Jan 2025 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 18 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Standard treatment Group | No intervention | –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. |
| Criteria-Fulfilled ICF Group | Experimental | 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: EntrectinibIntervention 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. |
| Compassionate-Use ICF Group | Experimental | 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: EntrectinibPatients 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. |
| RWS ICF Group | Experimental | Drug: OtherPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Male or female, the age at the time of signing the informed consent is no less than 18 years old;
- Patients with advanced or metastatic rare solid tumor confirmed by histological confirmed;
- ECOG score is 0 or 1;
- Expected survival ≥12 weeks;
- 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;
Key exclusion criteria (28 listed)
- No patient lesions available for organoid model construction;
- History of interstitial lung disease or radiation pneumonitis of any type;
- 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);
- Current uncontrollable third cavity effusion, such as a large amount of pleural effusion，ascites , or pericardial effusion;
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Albumin-Bound PaclitaxelAlso: AbraxaneAlbumin-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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| EpirubicinAlso: EllenceEpirubicin 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| GemcitabineAlso: GemzarGemcitabine 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| VinorelbineAlso: Navelbine, VumonVinorelbine 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| CisplatinAlso: PlatinolCisplatin 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| IrinotecanAlso: CamptosarIrinotecan 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| FluorouracilAlso: Adrucil, 5-FUFluorouracil 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| NivolumabAlso: OpdivoNivolumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| PembrolizumabAlso: KeytrudaPembrolizumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| DurvalumabAlso: ImfinziDurvalumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| AtezolizumabAlso: TecentriqAtezolizumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| SintilimabAlso: TyvytSintilimab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| TislelizumabAlso: BaizeanTislelizumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| CamrelizumabAlso: AiRuiKaCamrelizumab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| ToripalimabAlso: TuoyiToripalimab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| SerplulimabAlso: HansizhuangSerplulimab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| AdebrelimabAlso: AbesiveAdebrelimab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| EnvafolimabAlso: EpkinlyEnvafolimab 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| OsimertinibAlso: TagrissoOsimertinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| AlectinibAlso: AlecensaAlectinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| VemurafenibAlso: ZelborafVemurafenib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| PamiparibAlso: BalversaPamiparib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| PyrotinibAlso: NerlynxPyrotinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| ImatinibAlso: GleevecImatinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| PalbociclibAlso: IbrancePalbociclib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| SavolitinibAlso: OrpathysSavolitinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| EntrectinibAlso: RozlytrekEntrectinib 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. | Drug | Compassionate-Use ICF Group, Criteria-Fulfilled ICF Group |
| OtherPatients 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. | Drug | RWS ICF Group |
Primary outcome
Objective Response Rate (ORR)
Time frame: From enrollment to the end of treatment at 8 weeks
The percentage of patients with a confirmed investigator-assessed complete or partial response according to Response Evaluation Criteria In Solid Tumours (RECIST) 1.1.
4 secondary outcomes
Disease Control Rate (DCR)
Time frame: From enrollment to the end of treatment at 8 weeks
Progression free survival (PFS)
Time frame: From enrollment to the end of treatment at 8 weeks
Overall survival (OS)
Time frame: From enrollment to the end of treatment at 8 weeks
Duration of response (DOR)
Time frame: From enrollment to the end of treatment at 8 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
shunbin wang, Doctor study chair
Peking University Shenzhen Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Albumin-Bound Paclitaxel, Epirubicin and Gemcitabine in people with Rare Tumour, run by Peking University Shenzhen Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact shubin wang, Doctor (+86 13823394076, wangshubin2013@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: From enrollment to the end of treatment at 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2024.