Investigator choice of standard therapy versus sequential novel therapy arms in the treatment of relapsed follicular lymphoma (REFRACT): study protocol for a multi-centre, open-label, randomised, phase II platform trial

被引：1

作者：

Mcilroy, Graham ^{[1
]}

Lax, Sian ^{[1
]}

Gaskell, Charlotte ^{[1
]}

Jackson, Aimee ^{[1
]}

Rhodes, Malcolm ^{[2
]}

Seale, Tania ^{[3
]}

Fox, Sonia ^{[1
]}

Hopkins, Lousie ^{[1
]}

Okosun, Jessica ^{[4
]}

Barrington, Sally F. ^{[5
,6
]}

Ringshausen, Ingo ^{[7
]}

Ramsay, Alan G. ^{[8
]}

Calaminici, Maria ^{[9
,10
]}

Linton, Kim ^{[3
,11
]}

Bishton, Mark ^{[12
,13
]}

机构：

[1] Univ Birmingham, Canc Res UK Clin Trials Unit CRCTU, Birmingham, England

[2] Natl Canc Res Inst, London, England

[3] Univ Manchester, Div Canc Sci, Manchester, England

[4] Queen Mary Univ London, Barts Canc Inst, London, England

[5] Kings Coll London, London, England

[6] Kings Coll London, Guys & St ThomasPET Ctr, Sch Biomed Engn & Imaging Sci, Kings Hlth Partners, London, England

[7] UCL, UCL Canc Inst, London, England

[8] Kings Coll London, Fac Life Sci & Med, Sch Canc & Pharmaceut Sci, London, England

[9] Queen Mary Univ London, Dept Cellular Pathol Barts Hlth, London, England

[10] Queen Mary Univ London, Barts Canc Inst, Ctr Haematooncol, London, England

[11] Christie NHS Fdn Trust, Dept Med Oncol, Manchester, England

[12] Univ Nottingham, Translat Med Sci, Nottingham, England

[13] Nottingham Univ Hosp NHS Trust, Dept Haematol, Nottingham, England

来源：

BMC CANCER | 2024年 / 24卷 / 01期

基金：

英国工程与自然科学研究理事会;

关键词：

Clinical trial; Relapsed follicular lymphoma; Bayesian power prior methodology; Adaptive design; Epcoritamab; Lenalidomide; PROGRESSION-FREE SURVIVAL; METABOLIC TUMOR VOLUME; NON-HODGKIN-LYMPHOMA; POOLED ANALYSIS; PET-CT; PREDICTION; RISK;

D O I：

10.1186/s12885-024-12112-0

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Background Relapsed or refractory follicular lymphoma (rrFL) is an incurable disease associated with shorter remissions and survival after each line of standard therapy. Many promising novel, chemotherapy-free therapies are in development, but few are licensed as their role in current treatment pathways is poorly defined.Methods The REFRACT trial is an investigator-initiated, UK National Cancer Research Institute, open-label, multi-centre, randomised phase II platform trial aimed at accelerating clinical development of novel therapies by addressing evidence gaps. The first of the three sequential novel therapy arms is epcoritamab plus lenalidomide, to be compared with investigator choice standard therapy (ICT). Patients aged 18 years or older with biopsy proven relapsed or refractory CD20 positive, grade 1-3a follicular lymphoma and assessable disease by PET-CT are eligible. The primary outcome is complete metabolic response by PET-CT at 24 weeks using the Deauville 5-point scale and Lugano 2014 criteria. Secondary outcomes include overall metabolic response, progression-free survival, overall survival, duration of response, and quality of life assessed by EQ-5D-5 L and FACT-Lym. The trial employs an innovative Bayesian design with a target sample size of 284 patients: 95 in the ICT arm and 189 in the novel therapy arms.Discussion Whilst there are many promising novel drugs in early clinical development for rrFL, understanding the relative efficacy and safety of these agents, and their place in modern treatment pathways, is limited by a lack of randomised trials and dearth of published outcomes for standard regimens to act as historic controls. Therefore, the aim of REFRACT is to provide an efficient platform to evaluate novel agents against standard therapies for rrFL. The adaptive Bayesian power prior methodology design will minimise patient numbers and accelerate trial delivery.Trial registration ClinicalTrials.gov: NCT05848765; 08-May-2023.EudraCT 2022-000677-75; 10-Feb-2022.

引用

页数：13