A Pilot Study of 5-AZA and ATRA for Prostate Cancer With PSA-only Recurrence After Local Treatment

What Was Tested

In this phase 2 trial, researchers set out to test Leuprolide.

This is a prospective, open-label, randomized, cross-over, pilot study of reprogramming therapy in patients with recurrent PCa based on rising PSA only.

The primary objectives are to compare the disease progression-free rate at the end of 12 weeks of treatment between 5-AZA+ATRA and no therapy and to assess safety of the 5-AZA and ATRA combination.

All study enrollees will receive Lupron.

It had two arms (groups): 5-AZA + ATRA ('early' 5-AZA+ATRA) (experimental) and No therapy ('delayed' 5-AZA+ATRA) (active comparator).

Combination of 5-Azacitidine (5-AZA) + all trans retinoic acid (ATRA) group after one month of Lupron, group will receive treatment on a 28-day cycle, in the absence of prohibitive toxicities, for 3 cycles.

After one month of lupron, patients will receive no therapy for 3 cycles (12 weeks). After this observation period, patients will receive combination of 5-Azacitidine (5-AZA) + all trans retinoic acid (ATRA) group on a 28-day cycle, in the absence of prohibitive toxicities, for 3 cycles.

The registry classes the interventions as drug.

Who Took Part

Enrollment: 14 actual participants took part.

It was open to men only aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Histologically confirmed adenocarcinoma of the prostate. PSADT ≤ 10 months prior to initiation of ADT. No evidence of regional or active distant metastases, except for regional metastasis where salvage radiation therapy is not an option. Indication for ADT after receiving definitive local therapy. Males ≥ 18 years.

The full record lists 10 inclusion and 14 exclusion criteria; among the exclusions: patients who have received adt and/or other chemotherapy within 3 months prior to entering the study; patients may not be receiving any other investigational agents; history of allergic reactions attributed to compounds of similar chemical or biologic composition to 5-aza and atra.

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

Random assignment meant chance, not the researchers, decided who got Leuprolide and who got the comparison, which limits bias.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Being open-label, the Leuprolide study did not mask who received what.

It started in 2018-08-20 with completion listed for 2022-06-19.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Number of Participants With PSA Response (time frame: baseline and 24 weeks).

It also tracked 1 secondary outcome, for example Percentage of Patients With Prolongation of PSA Doubling Time (PSADT) Post-treatment.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Leuprolide in 14 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Leuprolide compound page, all human trials.