A Phase 3 Study to Evaluate the Safety and Efficacy of APL-1202 as a Single-agent Oral Treatment Versus Intravesical Instillation of Epirubicin Hydrochloride in naïve Intermediate-risk NMIBC Patients (ASCERTAIN)

A Phase 3 Study to Evaluate the Safety and Efficacy of APL-1202 as a Single-agent Oral Treatment Versus Intravesical Instillation of Epirubicin Hydrochloride in naïve Intermediate-risk NMIBC Patients (ASCERTAIN)

Brief Summary:
A multi-center, randomized, open-label, parallel-controlled Phase Ⅲ clinical trial to evaluate the clinical safety and efficacy of APL-1202 as a single-agent oral treatment versus intravesical instillation of Epirubicin hydrochloride in naïve intermediate-risk non-muscle invasive bladder cancer (NMIBC) patients

Detailed Description:
A multi-center, randomized, open-label, placebo controlled Phase Ⅲ trial. The subjects must be histopathologically diagnosed, naïve intermediate-risk NMIBC patient. The screening period is 6 weeks prior to treatment.

The trial including 2 stages:
• First stages: The subjects are randomly assigned to receive APL-1202 single-agent oral treatment or intravesical instillation of Epirubicin hydrochloride at a ratio of 1:1. At this stage, an interim analysis will be performed after the first 100 subjects enrolled completing the 6-month visit (completing the second cystoscopy), and the Independent Data Monitoring Committee (IDMC) conducted a preliminary evaluation of the safety. If approved by the IDMC after the safety evaluation, the trial will proceed to the second stage. Before the result of the interim analysis are obtained, subjects receiving APL-1202 will continue to be given continuous administration if they have completed the 6-month visit (V4). After the interim analysis, if the safety of continuous administration is as expected, subjects receiving APL-1202 will continue to do the same, otherwise it will be adjusted to the administration of APL-1202 at every 3-month interval which is proven to be safe and tolerable.

• Second stage: After the interim analysis of the first stage, the subjects of the second stage will be enrolled, and the subjects were randomly assigned to receive APL-1202 single-agent oral treatment or intravesical instillation of Epirubicin hydrochloride at a ratio of 1:1. For treatment, according to the results of the interim analysis, if the safety of continuous administration reaches the expected level, subjects receiving APL-1202 will continue to do the same, otherwise it will be adjusted to the administration of APL-1202 at every 3-month interval which is proven to be safe and tolerable.

In this trial, the APL-1202 dosing regimen will continue until subjects exit the trial early for any reason, complete 24 months' treatment or the trial is terminated early.

Cystoscopy will be performed every 3 months in the enrolled subjects. If there is no recurrence in the enrolled subject during the treatment period (2 years after enrollment), cystoscopy will be performed every 6 months during the follow-up period until recurrence or the end of the clinical trial.

Safety assessment will be performed every 3 months during the treatment period.


Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 800 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-center, Randomized, Open-label, Parallel-controlled Phase 3 Clinical Trial to Evaluate the Clinical Safety and Efficacy of APL-1202 as a Single-agent Oral Treatment Versus Intravesical Instillation of Epirubicin Hydrochloride in naïve Intermediate-risk Non-muscle Invasive Bladder Cancer (NMIBC) Patients
Actual Study Start Date: September 29, 2021
Estimated Primary Completion Date: March 1, 2025
Estimated Study Completion Date: December 31, 2025

Arm:
- Active Comparator: Epirubicin hydrochloride group
- Experimental: APL-1202 group

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