# Efficacy and Safety Study of TOOKAD® Soluble for Localised Prostate Cancer Compared to Active Surveillance

Source: https://onco.cc/trials/nct01310894/  
OnCo record `nct01310894` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A phase 3 trial of Padeliporfin in Prostate cancer, run by Steba Biotech S.A., completed.

## Summary

Efficacy and Safety Study of TOOKAD® Soluble for Localised Prostate Cancer Compared to Active Surveillance is a phase 3 interventional study registered as NCT01310894 by Steba Biotech S.A., with 413 participants recorded, started 2011-02 and reaching its primary completion in 2015-06. Interventions recorded: TOOKAD® Soluble. Conditions recorded: Prostate Cancer.

Linked to the drug Padeliporfin because the registry names it among its interventions and Prostate cancer among its conditions. Registry record only: no result has been read by an editor.

## Fields

- Kind: Trial
- Status: completed
- Last checked: 2026-09-22
- Also known as: PCM301; CLIN1001 PCM301; TOOKAD Soluble phase 3
- Tags: pipeline; ctgov-ingest
- Registry id: NCT01310894
- Phase: 3
- Setting: A European Randomised Phase 3 Study to Assess the Efficacy and Safety of TOOKAD® Soluble for Localised Prostate Cancer Compared to Active Surveillance
- Sponsor: Steba Biotech S.A.
- Enrolled: 413
- Result: Disease progression at 24 months in 28 percent after padeliporfin vascular-targeted photodynamic therapy against 58 percent under active surveillance (adjusted hazard ratio 0.34, 95 percent confidence interval 0.24 to 0.46); negative biopsy at 24 months in 49 against 14 percent. NICE TA546 does not recommend the treatment in England.
- Outcomes: Disease progression at 24 months: Padeliporfin vascular-targeted photodynamic therapy 28% vs Active surveillance 58%, HR 0.34; Negative prostate biopsy at 24 months: Padeliporfin vascular-targeted photodynamic therapy 49% vs Active surveillance 14%
- Replication: No independent randomised replication; it remains the only phase 3 comparison of a focal therapy against active surveillance in prostate cancer.

## Notes

- PCM301 randomised 413 men with low-risk, Gleason pattern 3 localised prostate cancer at 47 European university centres and community hospitals between 8 March 2011 and 30 April 2013. Treatment was 4 mg/kg padeliporfin intravenously over 10 minutes with optical fibres inserted into the prostate and activated by 753 nm laser light at a fixed 150 mW/cm for 22 minutes 15 seconds. Active surveillance followed best practice at the time of design: biopsy at 12-month intervals with PSA and digital rectal examination every 3 months. Median follow-up was 24 months.

The commonest grade 3 to 4 adverse events after treatment were prostatitis (three men, 2 percent, against one under surveillance), acute urinary retention (three, 2 percent, against one) and erectile dysfunction (two, 1 percent, against three). The commonest serious adverse event after treatment was urinary retention in 15 men, severe in three, resolving within 2 months in all (Lancet Oncology 2017).
- NICE TA546 does not recommend padeliporfin within its marketing authorisation for untreated, unilateral, low-risk prostate cancer. The committee's stated reasoning was that long-term studies show men with low-risk disease live as long under active surveillance, that radical therapies carry long-term severe side effects, and that better diagnostics now identify low-risk disease more accurately.

## Sources

- ClinicalTrials.gov NCT01310894: https://clinicaltrials.gov/study/NCT01310894
- PCM301 padeliporfin against active surveillance (Lancet Oncology 2017): https://doi.org/10.1016/S1470-2045(16)30661-1
- NICE TA546: padeliporfin for untreated localised prostate cancer, not recommended: https://www.nice.org.uk/guidance/ta546

## Connected records

- cancers: [Localised prostate cancer, very low and low risk](https://onco.cc/cancers/prostate-low-risk/), [Prostate cancer](https://onco.cc/cancers/prostate/)
- drugs: [Padeliporfin](https://onco.cc/drugs/padeliporfin/)
- companies: [Steba Biotech](https://onco.cc/companies/steba-biotech/)
- terms: [Focal and tissue-preserving treatment of prostate cancer](https://onco.cc/terms/prostate-focal-therapy/)
- trials: [ProtecT](https://onco.cc/trials/protect/)

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