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

NCT07122648 | Not Yet Recruiting Phase 2

• 1 other identifier: PN-101-201
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 5

Brief 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

Conditions

Polymyositis; Dermatomyositis

Keywords

mitochondria

Outcome Measures

Primary Outcomes (1)

• International Myositis And Clinical Studies group-Total Improvement Score (IMACS-TIS)

  Assessment of IMACS-TIS at Week 12 (Visit 6) after the IP administration. Total Improvement Score (TIS) based on absolute percentage change is assessed scale 0 to 100. Higher score indicates greater improvement.

  12 weeks after the IP administration

Secondary Outcomes (5)

• International Myositis And Clinical Studies group-Total Improvement Score (IMACS-TIS)

  4 weeks, 8 weeks , and 16 weeks after the IP administration

• Response rate of IMACS-TIS

  4 weeks, 8 weeks , 12 weeks, and 16 weeks after the IP administration

• Changes of Core Set Activity Measures(CSAM)

  Visit 2(Day 1), Visit 4(4 weeks), Visit 5(8 weeks), Visit 6(12 weeks), Visit 7(16 weeks)

• Changes of Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

  Visit 2(Day 1), Visit 4(4 weeks), Visit 5(8 weeks), Visit 6(12 weeks), Visit 7(16 weeks)

• Changes of Peak Pruritus Numeral Rating Scale(PPNRS)

  Visit 2(Day 1), Visit 4(4 weeks), Visit 5(8 weeks), Visit 6(12 weeks), Visit 7(16 weeks)

Study Arms (3)

Placebo Group

PLACEBO COMPARATOR

Biological: placebo

300 ug of PN-101 group

EXPERIMENTAL

Biological: PN-101

600 ug of PN-101 group

EXPERIMENTAL

Biological: PN-101

Interventions

placebo BIOLOGICAL

Participants will receive a single IV dose of 300 µg of placebo

Placebo Group

PN-101 BIOLOGICAL

Participants will receive a single IV dose of 300 µg of PN-101

300 ug of PN-101 group

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult aged 19 years or more
• A subject who is diagnosed with polymyositis or dermatomyositis and satisfies all of the followings:
• Clinical profile: Slowly progressing clinical profile with symmetrical and apparent muscular weakness confirmed at the proximal muscle (in case of dermatomyositis, clinical findings related with characteristic skin symptoms\*)
• Gottron's papules or sign, erythema purpura, poikiloderma, calcinosis cutis, etc.
• Serum test: Serum creatine kinase (CK) elevated (CK ≥ 1.3 × upper limit of normal (ULN)) or serum myositis-specific antibodies (MSA) positive
• Electromyography (EMG): Presence of a finding that indicates myopathy
• Baseline (prior to the investigational product administration) manual muscle testing-8 (MMT-8) result \< 125/150 (bilaterally), and at least 2 of the following International Myositis and Clinical Studies Group (IMACS) core set results
• Physician global disease activity \[visual analogue scale (VAS)\] ≥ 2 cm
• Patient global disease activity \[VAS\] ≥ 2 cm
• Health assessment questionnaire (HAQ) disability assessment ≥ 0.25
• or more items with the serum muscle enzyme \> 1.3 × ULN
• Global extramuscular disease activity \[VAS\] \> 1 cm
• Individuals who are currently receiving glucocorticosteroids and/or steroid-sparing drugs such as immunosuppressants or immunomodulators for the treatment of polymyositis or dermatomyositis but are deemed to have an inadequate response to treatment, or who are unable to continue existing treatment due to drug-related adverse events or side effects (however, during the clinical trial, the dosage of steroids and immunosuppressants may be adjusted within 20% of the dose prior to the study participation)
• Individuals who are receiving exercise or physical therapy and have agreed to maintain the same intensity and frequency of their current therapy
• A subject who fully understands the trial and provided voluntary written consent to take part in the trial

You may not qualify if:

• Subjects who meet any of the following criteria will not be eligible to participate in this clinical trial:
• A subject with clear muscular damage, with the VAS-based myositis damage index (MDI) of ≥ 5 at screening
• A subject with the following medical history or surgical history
• A surgical operation history within 12 weeks of screening
• Malignant tumor within 5 years of screening (excluding a subject who passed 3 years or more from complete recovery of cervical cancer or skin squamous cell carcinoma)
• Patients diagnosed with polymyositis or dermatomyositis before the age of 10 (Juvenile PM or Juvenile DM)
• A patient with severe respiratory muscular weakening or interstitial pulmonary disease (a patient who has no moderate or severe dyspnea and has stable interstitial pneumonia may participate)
• A patient with the following comorbidity at screening
• Acute viral infection or severe infection
• Active hepatitis B (e.g.: HBsAg positive and HBV DNA detected) or hepatitis C (e.g.: Anti-HCV positive and HCV RNA \[qualitatively\] detective)
• Human Immunodeficiency virus (HIV) positive
• Autoimmune disease such as rheumatoid arthritis (RA), systemic lupus erythematosus, psoriatic arthritis, etc. (however, in case of the overlap syndrome, a subject may participate if diseases other than inflammatory myositis are stable and myositis is thought to be due to inflammatory myositis.)
• Findings of cardiac disorder such as moderate or severe heart failure (New York Heart Association Class III/IV) or QT corrected interval prolonged
• Serious disease that may affect this study, at the discretion of the investigator (neurological disorder, cardiovascular disorder, uncontrolled blood pressure or diabetes, etc.)
• Hematological, renal and hepatic dysfunction based on the following laboratory findings at screening

Sponsors & Collaborators

• Paean Biotechnology Inc.: lead

Study Sites (5)

Chung-Ang University Gwang Myeong Hospital

Gwangmyeong, Gyeonggi-do, 14353, South Korea

Location

Kyung Hee University Medical Center

Seoul, 02447, South Korea

Location

Seoul National University

Seoul, 03080, South Korea

Location

Soon Chun Hyang University Hospital Seoul

Seoul, 04401, South Korea

Location

Chung-Ang University Hospital

Seoul, 06973, South Korea

Location

MeSH Terms

Conditions

Polymyositis; Dermatomyositis

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double-blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: August 14, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): April 1, 2027
• Last Updated: August 14, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

KR (5)