ClinicalTrials.gov record 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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2 randomized, double-blind trial compares single intravenous doses of PN-101 at two dose levels with placebo. It targets adults with polymyositis or dermatomyositis who have had an inadequate response to standard treatments. Participants receive one IV infusion and are followed with disease activity and safety assessments through 16 weeks, with the primary efficacy assessment at 12 weeks.
What it is studying. The study tests PN-101—mitochondria isolated from allogeneic umbilical cord-derived mesenchymal stem cells—given as a single IV dose (300 µg or 600 µg) and compares disease activity and safety against placebo in adults with refractory polymyositis or dermatomyositis, using the IMACS Total Improvement Score and other measures.
What the researchers hope to learn. To evaluate the efficacy and safety of single IV doses of PN-101 in refractory polymyositis and dermatomyositis. Primary scientific goal: measure change in the International Myositis and Clinical Studies Group Total Improvement Score (IMACS-TIS) at 12 weeks. Secondary goals: IMACS-TIS at other timepoints, response rates, changes in core IMACS measures, cutaneous disease severity (CDASI) and itch (PPNRS) for dermatomyositis.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- placebo
- PN-101
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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
- 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
Exclusion criteria
- 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
- 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
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomly assigned (1:1:1) and blinded to receive a single intravenous dose of PN‑101 (300 µg or 600 µg) or placebo. You will be observed for acute reactions (about 30 minutes) immediately after the infusion, then attend scheduled visits with safety checks and efficacy assessments at about weeks 4, 8, 12 and 16. Assessments include muscle-strength testing (MMT‑8), blood tests, patient questionnaires, and for dermatomyositis skin scores; steroid/immunosuppressant doses may be kept stable or adjusted within 20% as allowed. The study team will monitor safety throughout and perform the planned evaluations according to the visit schedule.
How the study is structured. Phase 2, randomized, double-blind, placebo-controlled, parallel-group trial. Participants are randomly assigned 1:1:1 to receive a single IV dose of PN-101 300 µg, PN-101 600 µg, or placebo. Safety monitoring includes observation for acute adverse events (about 30 minutes) after infusion and scheduled follow-up visits with assessments at weeks 4, 8, 12 (primary), and 16.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in South Korea. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on dermatomyositis
This trial is also listed on this site under polymyositis.
Important dates
- Study start
- December 2025 (estimated)
- Primary completion
- April 2027 (estimated)
- Study completion
- April 2027 (estimated)
- Target enrollment
- 36 participants (estimated)
- Last updated on registry
- Aug 14, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Paean Biotechnology Inc.
Participating sites (5)
- Chung-Ang University Gwang Myeong Hospital
Gwangmyeong, Gyeonggi-do, 14353 · South Korea
- Eun Hye Park, MD
- Kyung Hee University Medical Center
Seoul, 02447 · South Korea
- Yeonah Kim, MD
- Seoul National University
Seoul, 03080 · South Korea
- Eunyoung Lee, MD
- Soon Chun Hyang University Hospital Seoul
Seoul, 04401 · South Korea
- Hyunsook Kim, MD
- Chung-Ang University Hospital
Seoul, 06973 · South Korea
Terms used on this page · explanations by MedicalUpdates.ai
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- control group
- The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Glomerular filtration rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- prohibited concomitant
- A medicine that participants may not use during the study, usually because it could interfere with the study treatment or its measurement.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- half-life
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- randomly assigned
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- 1:1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- stratified
- Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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