NCT06293716

CVL237 Tablets for APDS/PASLI

An Open, Dose-exploration Study, Followed by a Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Efficacy and Safety of CVL237 Tablets in Patients With APDS/PASLI

• Status: Unknown
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Convalife (Shanghai) Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This study was designed to evaluate the efficacy and safety of CVL237 tablets in patients with APDS/PASLI (activated phosphoinositol 3-kinase δ syndrome /p110 delta-activated mutation leading to senescent T cells, lymphadenopathy, and immune deficiency).

Detailed description

The study was divided into two parts. Part I is an open, dose-escalation study planned to enroll five patients with APDS/PASLI to determine the safety, tolerability, pharmacokinetics (PK), and in vivo pharmacodynamics (PD pAkt) of CVL237 tablets at two different dose levels. Patients were given CVL237 tablets, 100 mg/ day, QD, for 4 weeks (28 days), and safety assessment was performed on day 28 of the first cycle. If there was no safety risk, patients could take CVL237 tablets, 200 mg/ day, QD, for 4 weeks (28 days). Part II is a randomized, double-blind, placebo-controlled study of approximately 30 patients with APDS/PASLI. On day 1, patients were randomly assigned to the trial and placebo groups in a 2:1 ratio to take an oral CVL237 tablet or a placebo CVL237 simulant once daily. Efficacy and safety were evaluated on days 29, 57, and 85. The efficacy of CVL237 tablets in reducing lymphadyopathy will be investigated as measured by changes in the sum of diameter products (SPD) of target lesions selected from MRI or CT imaging according to the Lugano 2014 method, as well as changes in the percentage of naive B cells to total B cells, relative to baseline. The CVL237 tablets will also be evaluated for safety, PK.

Conditions

• PI3K and P110delta Hyperactivation Syndrome

Interventions

Type	Name	Description
DRUG	CVL237 tablets	CVL237 tablets, tablets, specification: 0.1g, Storage condition: not more than 30 ℃ storage.Validity: 72 months tentatively.
DRUG	CVL237 placebo tablets	CVL237 tablets, tablets, specification: 0g, Storage condition: not more than 30 ℃ storage.Validity: 72 months tentatively.

Timeline

• Start date: 2024-03-30
• Primary completion: 2025-12-29
• Completion: 2026-03-30
• First posted: 2024-03-05
• Last updated: 2024-03-05

Locations

1 site across 1 country: China