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PolymyositisPhase 2NCT07122648

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

A Phase 2 study testing PN-101 in people with Polymyositis and Dermatomyositis, run by Paean Biotechnology Inc. at 5 sites in South Korea.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial 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… Read more about this study

Who is it for?
Ages 19 years and older
Conditions the study is for
Polymyositis, Dermatomyositis
What is being tested?
PN-101
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
36 participants
Where
5 sitesSouth Korea
Study dates
Dec 2025to Apr 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 19 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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
  • 1 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

Who cannot join

  • 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)
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  • 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
  • Findings of muscular inflammation or myopathy other than the indication (inclusion body myositis (IBM), drug-induced myopathy, amyloid myopathy, myotonic dystrophy, etc.)
  • 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
  • Glomerular filtration rate (GFR)* < 45 mL/min *eGFR (mL/min/1.73m^2) = 175 × (serum creatinine concentration (mg/dL))^-1.154 × (age)^-0.203 × (0.742 in female) [modification of diet in renal disease (MDRD) formula]
  • Hemoglobin < 10 g/dL
  • White blood cell (WBC) count < 3.0×10^9/L
  • Absolute neutrophil count (ANC) < 1.5×10^9/L (1500/mm^3)
  • Platelet count < 100×10^9/L
  • AST and ALT > 2.5 × ULN (except for the elevation due to muscle enzyme at the discretion of the investigator)
  • Alkaline phosphatase (ALP) > 2.5 × ULN
  • Total bilirubin > 1.5 × ULN (> 3 × ULN, in case of Gilbert's syndrome)
  • Thyroid stimulating hormone level exceeding the normal range (however, if the level exceeds the normal range due to the study indication at the discretion of the investigator, the subjects are allowed to enroll.)
  • A subject with a difficulty in the efficacy assessment including the muscular strength assessment during the trial
  • A subject who is determined to require prohibited concomitant treatment during the trial
  • Pregnant woman and lactating mother or woman of childbearing potential and man who is planning to have a child or not willing to practice acceptable contraception* for 36 weeks after the last dosing date *Hormonal contraception, intrauterine device or intrauterine system implant, surgical sterilization procedure/operation (vasectomy, tubal ligation, etc.)
  • Participation in other clinical trial and administration of an investigational product or application of an investigational device within 4 weeks or half-life x 5 (whichever is longer) prior to screening
  • A subject who is otherwise ineligible for this trial, at the discretion of the investigator

Trial locations and contacts

5 sites in 1 country. Contact the study team or a site near you to ask about taking part.

5 sites in 1 country

South Korea5 sites

  • Chung-Ang University Gwang Myeong Hospital

    Gwangmyeong, Gyeonggi-do, 14353

    Eun Hye Park, MD

  • Kyung Hee University Medical Center

    Seoul, 02447

    Yeonah Kim, MD

  • Seoul National University

    Seoul, 03080

    Eunyoung Lee, MD

  • Soon Chun Hyang University Hospital Seoul

    Seoul, 04401

    Hyunsook Kim, MD

  • Chung-Ang University Hospital

    Seoul, 06973

Other trials in South Korea

About this study

Official title: 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

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

Read the 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.
Being tested
placebo, PN-101
Keywords
mitochondria
Medical subject headings
Polymyositis, Dermatomyositis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • placeboBiological

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

  • PN-101Biological

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

  • PN-101Biological

    Participants will receive a single IV dose of 600 µg of PN-101.

Study groups in NCT07122648
Study groupRoleWhat they receive
Placebo GroupPlacebo comparatorNot described
Receives: Biological: placebo
300 ug of PN-101 groupExperimentalNot described
Receives: Biological: PN-101
600 ug of PN-101 groupExperimentalNot described
Receives: Biological: PN-101

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 12 weeks after the IP administration

Secondary outcomes (5)

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

    Assessment of IMACS-TIS at week 4, week 8, and week 16 after the IP administration. Total Improvement Score (TIS) based on absolute percentage change is assessed scale 0 to 100. Higher score indicates greater improvement.

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

  2. Response rate of IMACS-TIS

    Proportion of subjects with the IMACS-TIS ≥ 20 at week 4, week 8, week 12, and week 16 after IP administration

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

  3. Changes of Core Set Activity Measures(CSAM)

    Changes in CSAM for the IMACS assessed at week 4, week 8, week 12, and week16 after the IP administration from the baseline (Visit 2). The core set for the IMCS consists of six components: physician global disease activity, patient global disease activity, MMT-8, extramuscular activity, HAQ, and serum muscle enzyme levels.

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

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

    Changes in CDASI assessed at week 4, week 8, week 12 and week 16 after the IP administration from the baseline (Visit 2) for dermatomyositis only.

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

  5. Changes of Peak Pruritus Numeral Rating Scale(PPNRS)

    Changes in PPNRS assessed at week 4, week 8, week 12 and week 16 after the IP administration from the baseline (Visit 2) for dermatomyositis only. The intensity of pruritis is assessed on a patient reported scale 0 to 10.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2025
  2. Study startDec 2025 (estimated)
  3. Primary completionApr 2027 (estimated)
  4. Study completionApr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
36 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07122648
Sponsor's protocol ID
PN-101-201
Status last verified
Jul 2025
First submitted
7 Aug 2025
Last updated
14 Aug 2025
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PN-101 in people with Polymyositis and Dermatomyositis, run by Paean Biotechnology Inc. at 5 sites in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 30 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 14 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

International Myositis And Clinical Studies group-Total Improvement Score (IMACS-TIS) (time frame: 12 weeks after the IP administration). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07122648.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2025.

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