NCT06692491

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

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)

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Peking University Shenzhen Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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

Interventions

Type	Name	Description
DRUG	Albumin-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.
DRUG	Epirubicin	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.
DRUG	Gemcitabine	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.
DRUG	Vinorelbine	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.
DRUG	Cisplatin	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.
DRUG	Irinotecan	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.
DRUG	Fluorouracil	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.
DRUG	Nivolumab	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.
DRUG	Pembrolizumab	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.
DRUG	Durvalumab	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.
DRUG	Atezolizumab	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.
DRUG	Sintilimab	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.
DRUG	Tislelizumab	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.
DRUG	Camrelizumab	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.
DRUG	Toripalimab	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.
DRUG	Serplulimab	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.
DRUG	Adebrelimab	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.
DRUG	Envafolimab	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.
DRUG	Osimertinib	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.
DRUG	Alectinib	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.
DRUG	Vemurafenib	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.
DRUG	Pamiparib	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.
DRUG	Pyrotinib	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.
DRUG	Imatinib	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.
DRUG	Palbociclib	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.
DRUG	Savolitinib	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.
DRUG	Entrectinib	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.
DRUG	Other	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.

Timeline

• Start date: 2025-01-01
• Primary completion: 2027-12-31
• Completion: 2029-12-31
• First posted: 2024-11-18
• Last updated: 2024-11-18