NCT07122648

Phase 2 Trial to Evaluate the Efficacy, Safety of Allogeneic Mitochondria (PN-101) in Patients With Refractory Polymyositis or Dermatomyositis

A Prospective, Multi-center, Randomized, Double-blinded, Placebo-controlled, Parallel, Phase 2 Trial to Evaluate the Efficacy, Safety of PN-101(Mitochondria Isolated From Allogeneic Umbilical Cord-derived Mesenchymal Stem Cells) Single Dose in Patients With Refractory Polymyositis or Dermatomyositis

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Paean Biotechnology Inc. · Industry
• Sex: All
• Age: 19 Years
• Healthy volunteers: Not accepted

Summary

The efficacy of PN-101 in subjects with polymyositis or dermatomyositis will be evaluated at Week 12 using IMACS-TIS in comparison with the placebo control group. The safety and efficacy will be evaluated following administration of PN-101 to subjects with polymyositis or dermatomyositis, in comparison with the placebo group

Detailed description

This Phase II clinical trial involves patients diagnosed with polymyositis or dermatomyositis. * Only subjects who have voluntarily provided written informed consent and have been determined to meet the inclusion/exclusion criteria will participate in this clinical trial. * Subjects will be randomly assigned in a 1:1:1 ratio to the PN-101 300 ug group, 600 ug group, or placebo control group according to their randomization number, and stratified based on the diagnosis of either polymyositis or dermatomyositis. * The investigator will administer a single intravenous dose of either the placebo or the investigational product to subjects in the placebo control group and the treatment groups. * Each subject who receives the investigational product will be monitored for any acute adverse events, such as hypersensitivity, within 30 minutes after administration. * Safety and efficacy will be evaluated according to the visit schedule after administration of the investigational product.

Conditions

• Polymyositis
• Dermatomyositis

Interventions

Type	Name	Description
BIOLOGICAL	placebo	Participants will receive a single IV dose of 300 µg of placebo
BIOLOGICAL	PN-101	Participants will receive a single IV dose of 300 µg of PN-101
BIOLOGICAL	PN-101	Participants will receive a single IV dose of 600 µg of PN-101.

Timeline

• Start date: 2025-12-01
• Primary completion: 2027-04-01
• Completion: 2027-04-01
• First posted: 2025-08-14
• Last updated: 2025-08-14

Locations

5 sites across 1 country: South Korea