NCT04976140

Allogeneic Mitochondria (PN-101) Transplantation for Refractory Polymyositis or Dermatomyositis

A Prospective, Open, Dose-escalation, Multi-center, Phase 1/2a Trial to Evaluate the Safety, Tolerability and to Explore the Efficacy of PN-101 in Patients With Refractory Polymyositis or Dermatomyositis

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Paean Biotechnology Inc. · Industry
• Sex: All
• Age: 19 Years
• Healthy volunteers: Not accepted

Summary

To determine the maximum tolerated dose (MTD) based on the safety and tolerability after single-dose administration of PN-101 in patients with refractory polymyositis or dermatomyositis. To explore the efficacy after single-dose administration of PN-101 in patients with refractory polymyositis or dermatomyositis.

Detailed description

This Phase 1/2a clinical trial involves patients diagnosed with refractory polymyositis or dermatomyositis. The initial safety assessment is conducted, including development of DLT in subjects up to Week 2 after the investigational product administration. \[DLT assessment criteria and method\] * Low dose or intermediate dose level 1. No DLT developing in the initially enrolled 3 subjects (0/3): Increase the dose to the next dose group 2. DLT developing 1 of 3 subjects (1/3): Enroll 3 additional subjects at the same dose group and then assess DLT, and ① DLT developing in 1 out of total 6 subjects (1/3+0/3; 1/6): Increase the dose to the next dose group ② DLT developing in ≥ 2 out of total 6 subjects (≥ 2/6): In case of the low dose level, the trial is discontinued without MTD determination. In case of the intermediate dose level, MTD is assessed at the low dose which is a one lower dose level * High dose level 1. No DLT developing in the initially enrolled 3 subjects (0/3): Declare as the MTD 2. DLT developing 1 of 3 subjects (1/3): Enroll 3 additional subjects at the same dose group and then assess DLT, and ① DLT developing in 1 out of total 6 subjects (1/3+0/3; 1/6): Declare as the MTD ② DLT developing in ≥ 2 out of total 6 subjects (≥ 2/6): Assess MTD at the intermediate dose which is the one lower dose level

Conditions

• Polymyositis
• Dermatomyositis

Interventions

Type	Name	Description
BIOLOGICAL	PN-101	PN-101: Mitochondria isolated from Allogeneic Umbilical Cord-derived Mesenchymal Stem Cells. 3 or 6 subjects are enrolled in each dose group in line with the traditional 3+3 rule-based method, and the investigator intravenously administers a single-dose of the investigational product according to the planned dose.

Timeline

• Start date: 2021-12-15
• Primary completion: 2023-10-06
• Completion: 2023-10-06
• First posted: 2021-07-26
• Last updated: 2024-04-03

Locations

3 sites across 1 country: South Korea