Various (Platform Trial) OCTOBER 14, 2025

STAMPEDE: Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy

STAMPEDE Trial Management Group

Bottom Line

The STAMPEDE trial is a landmark multi-arm, multi-stage platform study that has fundamentally reshaped the standard of care for men with high-risk, locally advanced, or metastatic hormone-naive prostate cancer by evaluating various systemic and local therapies.

Key Findings

1. Docetaxel chemotherapy: Demonstrated a significant overall survival (OS) benefit when added to standard-of-care androgen deprivation therapy (ADT) in men with metastatic hormone-sensitive prostate cancer (HR 0.78; P=0.006).
2. Abiraterone acetate: The addition of abiraterone to ADT significantly improved OS (HR 0.63; P<0.001) and failure-free survival (FFS) compared to ADT alone in patients with high-risk locally advanced or metastatic disease.
3. Prostate Radiotherapy (RT) in metastatic disease: RT to the primary tumor significantly improved OS in patients with low metastatic burden (HR 0.68; P=0.007) but showed no survival benefit in patients with high metastatic burden (HR 1.07; P=0.420).
4. Metformin: Evaluation in non-diabetic patients indicated no significant improvement in OS (HR 0.91; P=0.15) or other prostate cancer-specific outcomes, though it demonstrated favorable impacts on metabolic parameters such as weight gain and lipid profiles.

Study Design

Design
Multi-Arm, Multi-Stage (MAMS) RCT
Open-Label
Sample
11,900
Patients
Duration
Variable (Long-term)
Median
Setting
Multicenter, UK and Switzerland
Population Men with newly diagnosed high-risk locally advanced, metastatic, or relapsing hormone-naive prostate cancer.
Intervention Various experimental systemic therapies (e.g., Docetaxel, Abiraterone, Metformin, Enzalutamide) or primary prostate radiotherapy.
Comparator Standard of care (long-term androgen deprivation therapy).
Outcome Overall survival (OS) and/or failure-free survival (FFS).

Study Limitations

The multi-arm, multi-stage (MAMS) design introduced complexity in trial interpretation and longitudinal comparison across heterogeneous treatment arms.
Most comparisons were open-label, which may introduce potential bias in reporting subjective outcomes and adverse events.
The diverse population (newly diagnosed vs. relapsing) and evolution of the standard of care over the long duration of the trial complicate the direct comparison of results between different arms.
The lack of survival benefit for some agents despite potential improvements in surrogate biomarkers (e.g., metabolic changes with metformin) highlights the necessity for hard clinical endpoints.

Clinical Significance

The STAMPEDE platform has provided the evidence base to incorporate early docetaxel, abiraterone, and targeted primary radiotherapy into the standard management of hormone-sensitive prostate cancer, leading to improved long-term survival outcomes for patients.

Historical Context

Initiated in 2005 at the MRC Clinical Trials Unit, STAMPEDE pioneered the use of a MAMS platform design to efficiently evaluate multiple novel therapeutic combinations against a single contemporaneous control arm in a rapidly evolving field of prostate cancer care.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the physiological rationale for using Androgen Deprivation Therapy (ADT) as the foundational 'control' arm in the STAMPEDE trial for all stages of advanced prostate cancer?

Key Response

Prostate cancer is heavily dependent on the androgen receptor (AR) signaling pathway for growth and survival. By suppressing testosterone production via medical or surgical castration, the primary driver of tumor proliferation is inhibited. STAMPEDE uses ADT as the backbone therapy because it has been the standard of care since the work of Huggins and Hodges in the 1940s, providing a baseline against which the additive benefits of newer systemic agents or radiotherapy can be measured.

Resident
Resident

In patients with newly diagnosed metastatic hormone-naive prostate cancer (mHSPC), how did the STAMPEDE trial findings regarding the addition of docetaxel compare to the CHAARTED trial, particularly concerning metastatic volume?

Key Response

While the CHAARTED trial initially suggested a survival benefit for docetaxel primarily in 'high-volume' metastatic disease, the STAMPEDE trial demonstrated an overall survival benefit for docetaxel in the metastatic cohort regardless of tumor burden. This led to a broad recommendation for docetaxel plus ADT as a management option for all fit patients with mHSPC, though subsequent data and the 'STAMPEDE low-volume' analysis have since refined the preference for certain agents based on volume and patient comorbidities.

Fellow
Fellow

Analyze the STAMPEDE data regarding the use of radiotherapy to the primary prostate tumor in the setting of metastatic disease; which specific subgroup derives an overall survival benefit, and why is this clinically significant?

Key Response

STAMPEDE showed that local radiotherapy to the prostate improved overall survival (OS) only in patients with a low metastatic burden (defined by the CHAARTED criteria as fewer than 4 bone metastases and no visceral metastases), with a 3-year OS of 81% vs 73%. In high-burden disease, there was no OS benefit. This was a landmark finding because it proved that treating the primary 'seed' can alter the trajectory of systemic 'soil' in oligometastatic states, leading to the inclusion of local RT in guidelines for low-volume M1 disease.

Attending
Attending

The STAMPEDE trial demonstrated that adding Abiraterone to ADT improved survival in metastatic disease, but the combination of Abiraterone and Enzalutamide did not further improve outcomes over Abiraterone alone. How does this impact your approach to treatment sequencing and 'intensification' in practice?

Key Response

The comparison of Arm J (Abiraterone + Enzalutamide) versus the contemporaneous control showed no survival advantage for the combination but significantly higher toxicity. This teaches that simultaneous dual AR-axis blockade does not overcome resistance more effectively than single-agent intensification. In practice, this reinforces the 'less is more' approach regarding toxicity—choosing one potent AR-targeted agent (Abiraterone or Enzalutamide) rather than both, and saving subsequent agents for sequential use at the time of castration resistance.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The STAMPEDE trial utilizes a Multi-Arm Multi-Stage (MAMS) platform design. Discuss how the use of an intermediate primary endpoint, such as Failure-Free Survival (FFS), affects the statistical power and efficiency of the trial when deciding which arms to graduate to the final Overall Survival (OS) analysis.

Key Response

The MAMS design uses FFS (a composite of PSA failure, progression, or death) as a 'futility' filter. If an arm does not show a pre-specified level of benefit in FFS during the internal pilot stages, it is discontinued. This allows for rapid testing of multiple agents simultaneously using a shared control arm, reducing the total sample size required compared to separate Phase III trials. However, it requires a high correlation between the intermediate endpoint and the final endpoint (OS) to ensure that potentially beneficial treatments are not prematurely discarded.

Journal Editor
Journal Editor

A major criticism of platform trials like STAMPEDE is the 'drifting control arm.' How should reviewers critically evaluate the internal validity of comparisons made between arms that were recruited during non-overlapping or only partially overlapping time periods?

Key Response

As the Standard of Care (SOC) evolves (e.g., the transition from ADT-alone to ADT+Docetaxel as SOC), the control group changes. Comparing an experimental arm from 2015 to a control group from 2005 is invalid due to changes in imaging (e.g., PSMA-PET), supportive care, and subsequent lines of therapy. Editors look for 'contemporaneous' control comparisons—ensuring that the patients in the control arm were randomized at the same time as those in the experimental arm to account for temporal biases in clinical practice.

Guideline Committee
Guideline Committee

How do the results of the STAMPEDE 'M0' (non-metastatic) analysis for Abiraterone plus ADT compare to current NCCN/EAU guidelines for high-risk locally advanced prostate cancer?

Key Response

The STAMPEDE M0 analysis showed that for very high-risk non-metastatic patients (node-positive or meeting specific high-risk criteria), the addition of 2 years of Abiraterone to ADT and RT significantly improved metastasis-free survival (MFS) and OS (HR 0.60 for death). Consequently, guidelines (e.g., EAU) were updated to include Abiraterone for 2 years as a Level 1 recommendation for men with high-risk/very high-risk M0 disease, shifting the paradigm from 'RT + ADT' to 'RT + ADT + Abiraterone'.

Clinical Landscape

Noteworthy Related Trials

2015

CHAARTED Trial

n = 790 · NEJM

Tested

Docetaxel plus androgen-deprivation therapy (ADT)

Population

Men with metastatic hormone-sensitive prostate cancer

Comparator

Androgen-deprivation therapy (ADT) alone

Endpoint

Overall survival

Key result: The addition of docetaxel to ADT significantly prolonged overall survival compared to ADT alone.
2017

LATITUDE Trial

n = 1,199 · NEJM

Tested

Abiraterone acetate plus prednisone and ADT

Population

Patients with high-risk metastatic hormone-sensitive prostate cancer

Comparator

ADT plus placebo

Endpoint

Overall survival and radiographic progression-free survival

Key result: Abiraterone plus prednisone with ADT significantly improved overall survival and radiographic progression-free survival compared to ADT alone.
2019

ARCHES Trial

n = 1,150 · JCO

Tested

Enzalutamide plus ADT

Population

Men with metastatic hormone-sensitive prostate cancer

Comparator

Placebo plus ADT

Endpoint

Radiographic progression-free survival

Key result: Enzalutamide plus ADT significantly reduced the risk of radiographic progression or death compared to ADT alone.

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