Safety and Efficacy of ULSC on Disease Severity and Steroid Tapering in Participants With Dermatomyositis/ Polymyositis (DM/PM), Also Known as Idiopathic Inflammatory Myopathy (IIM)

NCT07160205 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: RES001-DMPM-02
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell (ULSC) product performs when treating Dermatomyositis/Polymyositis (DM/PM), also known as idiopathic inflammatory myopathy (IIM) in adults. It will assess safety and efficacy in relieving symptoms of DM/PM with ULSC administered in three intravenous (IV) doses of 150 million cells per dose. The main questions that this study plans to answer are:

• Is ULSC as safe as placebo (a look-alike saline without cells) in repeated IV infusion?
• Does ULSC improve symptoms of DM/PM after three doses? Researchers will compare ULSC to placebo and evaluate changes from baseline (before first dose) to after each dose and after all three doses are completed per treatment study period.
• For participants undergoing steroid (e.g., prednisone) therapy for DM/PM, does ULSC allow their steroid dose to be reduced? Does ULSC reduce need for rescue therapy? Participants will have been diagnosed with either DM or PM:
• Diagnosed according to the EULAR/ACR 2017 Classification Criteria for idiopathic inflammatory myositis (IIM), which includes DM and PM.
• Positive for myositis-associated antibody or undergone evaluation to exclude mimics. Participants in this study will:
• Participate for total of 25 months with 15 in-person clinic visits and 8 virtual visits on phone or video call.
• Receive both ULSC and placebo for a total of 6 IV infusions (260 mL) 3 months apart.
• Receive 3 doses of ULSC and 3 doses placebo in either of two sequences, as assigned: ULSC first and placebo second, or placebo first and ULSC second.
• If undergoing steroid therapy, will have steroid dose taper prescribing lower doses starting two weeks after the second infusion.
• Return for follow-up visits after each dose and up to 12 months after final dose.
• Have follow-ups including self-reported questionnaires, physical exam, muscle strength and endurance tests, blood tests, pulmonary function tests, and other assessments.

Conditions

Idiopathic Inflammatory Myositis (IIM); DERMATOMYOSITIS OR POLYMYOSITIS

Outcome Measures

Primary Outcomes (2)

• Safety based on Adverse Events (AEs) and Serious Adverse Events (SAEs) that begin during or following treatment infusion.

  Cumulative listing of all AEs/SAEs per cohort with descriptive statistics for categorical variables and count variables to compare between ULSC and Placebo, with emphasis on All SAEs and AEs/SAEs suspected to be treatment infusion-related.

  Each visit from Day 0, 7 days, and 30 days after each infusion, and all follow-up visits up to 12 months after the final treatment infusion.

• Efficacy based on Total Improvement Score (TIS, expressed as continuous variable) in the 2016 ACR/EULAR Myositis Response Criteria

  Total Improvement Score (TIS) as continuous variable (scale of 0 to 100 points) will be assessed; TIS is a weighted average of the sub-scores in the 6 core set measures that comprise the validated outcome measure in the 2016 ACR/EULAR Myositis Response Criteria.

  From baseline (i.e., before first dose per treatment) to 7 months (i.e., one month after the third/final dose per treatment) for each treatment group and each study period.

Secondary Outcomes (23)

• Need for Rescue Therapy (Incidence)

  From baseline to 7 months for both study periods.

• Need for Rescue Therapy (Time)

  From baseline to 7 months for both study periods.

• Glucocorticoid Dose (prednisone equivalents per day) Tapering

  From baseline to 4 months and 7 months for both study periods.

• Total Improvement Score (TIS) improvement category (minimal, moderate, or major improvement) in the 2016 ACR/EULAR Myositis Response Criteria

  From baseline to 7 months for both study periods.

• Total Improvement Score (TIS, expressed as continuous variable) in the 2016 ACR/EULAR Myositis Response Criteria after each repeat dose

  From baseline to 1 month, 4 months, and 7 months for both study periods.

Other Outcomes (26)

• Erythrocyte Sedimentation Rate (ESR) - systemic inflammatory/immune marker

  From baseline to 7 months for both study periods and post-study follow-up at 25 months.

• C-Reactive Protein (CRP) - systemic inflammatory/immune marker

  From baseline to 7 months for both study periods and post-study follow-up at 25 months.

• TGF-β1 - systemic inflammatory/immune marker

  From baseline to 7 months for both study periods and post-study follow-up at 25 months.

Study Arms (2)

Cohort 1: ULSC first; Placebo second

EXPERIMENTAL

Cohort 1 will receive 1.5 x 10\^8 ULSC per dose through IV infusion on Day 0, Month 3, and Month 6 (total of three doses). One month after the third dose, Cohort 1 will cross-over to receive Placebo IV infusion on Month 7, Month 10, and Month 13.

Biological: ULSC (1.5 x 10^8 cells/dose); Biological: Placebo (no cells)

Cohort 2: Placebo first; ULSC second

EXPERIMENTAL

Cohort 2 will receive Placebo IV infusion on Day 0, Month 3, and Month 6 (total of three Placebo infusions). One month after the third dose, Cohort 2 will cross-over to receive 1.5 x 10\^8 ULSC per dose through IV infusion on Month 7, Month 10, and Month 13.

Biological: ULSC (1.5 x 10^8 cells/dose); Biological: Placebo (no cells)

Interventions

ULSC (1.5 x 10^8 cells/dose) BIOLOGICAL

Allogeneic umbilical-cord lining stem cells (ULSC) are cryopreserved and supplied in vials to be thawed and prepared for infusion at point of use. Each dose of 1.5 x 10\^8 ULSC will be added into 250 sterile saline IV bag for infusion (total volume of 260 mL volume).

Cohort 1: ULSC first; Placebo second; Cohort 2: Placebo first; ULSC second

Placebo (no cells) BIOLOGICAL

The Placebo will be 250 ml sterile saline with vehicle (total volume of 260 mL) IV bag for infusion.

Cohort 1: ULSC first; Placebo second; Cohort 2: Placebo first; ULSC second

Eligibility Criteria

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

You may qualify if:

• Participants will be ≥18 years old.
• Diagnosis of idiopathic inflammatory myositis (IIM) based on 2017 EULAR/ACR Classification Criteria for adult IIM, corresponding to a score of ≥ 5.5 (≥ 6.7 with muscle biopsy).
• Active disease as defined by any one of the following test results:
• Elevated Creatine Kinase (CK) or Aldolase (more than 1.5 x the upper limit of normal) at screening, OR
• MRI positive for active, muscle inflammation within 12 weeks prior to screening, OR
• EMG read as active myositis within 12 weeks prior to screening, OR
• muscle biopsy obtained within 12 weeks of the screening showing active inflammatory disease.
• Muscle weakness or active cutaneous manifestations of dermatomyositis assessed at Screening and documented with either of the following scores:
• Bilateral MMT-8 score of ≤142/150, OR
• CDASI Total Activity score of ≥ 7.
• Participants must be receiving standard of care treatment with one or more immunosuppressants or at least 5 mg prednisone (or corticosteroid equivalent).
• Immunosuppressive doses should be stable for at least 12 weeks prior to enrollment. Participants must remain on stable immunosuppressive therapy for the duration of the trial unless discontinuation is warranted due to toxicity or another clinical reason.
• Hydroxychloroquine (HCQ) doses should be stable for at least 12 weeks prior to enrollment.
• Steroid doses should be stable for at least 4 weeks prior to enrollment. At screening and enrollment, maximum dose allowed is 25 mg/day prednisone (or corticosteroid equivalent).
• Allowed immunosuppressants include: methotrexate, azathioprine, mycophenolate, cyclosporine, IVIG, and others to be evaluated at the discretion of the investigator.

You may not qualify if:

• Adequate pulmonary function, defined as saturated oxygen (SpO2 ≥ 94%) on room air.
• Left ventricular ejection fraction (LVEF) ≥ 30% as assessed by echocardiogram (ECHO) or multigated acquisition (MUGA) scan performed within 8 weeks prior to Screening.
• Participants must have the ability to comply with the requirements of the study.
• All participants of reproductive age/capacity will be required to use adequate contraception, defined as at least one form of a highly effective contraceptive (i.e., condoms, hormonal birth control, IUD), with any partners during the study period and for at least three months beyond the study period, for safety.
• Participant will have the ability to understand and provide written informed consent.
• The presence of any of the following criteria excludes a participant from study enrollment:
• Diagnosis of IIM as part of an overlap syndrome (except overlap with Sjogren's syndrome).
• Initiation of Rituxan (rituximab) treatment within 12 weeks of randomization. If participant is already on Rituxan, they must remain on a stable dose throughout the trial.
• Use of other biologic or investigational drug within 6 half-lives for the agent.
• Diagnosis of myositis-associated interstitial lung disease or cardiac involvement sufficient to limit participation in the trial in the discretion of the PI.
• End-stage IIM with irreversible muscle involvement seen on biopsy.
• Patients with predominant muscle atrophy secondary to uncontrolled or chronic DM or PM, based on clinical, biochemical, and/or radiologic assessment, despite previous optimized treatment.
• Non-immune myopathies.
• Cancer associated with myositis.
• Hypersensitivity to study product components including history of hypersensitivity to dimethyl sulfoxide (DMSO).

Sponsors & Collaborators

• Restem, LLC.: lead

Study Sites (2)

Malcom Randall North Florida/South Georgia VA Medical Center

Gainesville, Florida, 32608, United States

RECRUITING

Bioresearch Partner

Miami, Florida, 33143, United States

RECRUITING

MeSH Terms

Conditions

Myositis; Dermatomyositis

Condition Hierarchy (Ancestors)

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

Study Officials

• Michael Bubb, MD

  Malcom Randall North Florida/South Georgia VA Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Trial Contact

CONTACT

352-278-8838; clinicaltrials@restem.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2025
• First Posted: September 8, 2025
• Study Start: January 6, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): August 1, 2029
• Last Updated: April 27, 2026

Record last verified: 2026-04

Locations

US (2)