Actively Recruiting
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)
Led by Restem, LLC. · Updated on 2026-04-27
40
Participants Needed
2
Research Sites
186 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 18 years or older
- Diagnosis of idiopathic inflammatory myositis based on 2017 EULAR/ACR criteria with a qualifying score
- Active disease shown by elevated muscle enzymes, MRI, EMG, or muscle biopsy within 12 weeks prior to screening
- Muscle weakness or skin symptoms meeting specific strength or activity scores
- Stable treatment with immunosuppressants or at least 5 mg prednisone daily for specified durations
- Positive for myositis-associated antibody or evaluated to exclude similar conditions
- Adequate lung function with oxygen saturation of 94% or higher on room air
- Left ventricular ejection fraction of 30% or higher within 8 weeks prior to screening
- Ability to comply with study requirements
- Use of effective contraception during and for three months after the study for those of reproductive age
- Ability to understand and provide informed consent
You will not qualify if you...
- Diagnosis of inclusion body myositis, juvenile dermatomyositis or polymyositis, or significant overlap autoimmune diseases except Sjogren's syndrome
- Use of Rituxan within 12 weeks before randomization or unstable dosing if already on it
- Use of other biologic or investigational drugs within 6 half-lives of the agent
- Myositis-associated interstitial lung disease or heart involvement limiting participation
- End-stage disease with irreversible muscle damage
- Predominant muscle wasting despite optimized treatment
- Non-immune muscle diseases
- Cancer related to myositis or active cancer within 2 years except certain skin or cervical cancers
- Hypersensitivity to study product components including DMSO
- Pregnant or lactating participants
- Severe cardiac or pulmonary disease, active infection, or other conditions preventing safe participation
- Expected need for surgery during the trial
- History of poor compliance with medical therapy
- Organ transplant recipient
- Low neutrophil count below specified thresholds
- Severe kidney impairment
- Recent or planned live virus vaccination
- Hepatitis B or C infection or HIV
- Conditions impairing muscle strength assessments such as Parkinson's disease or severe musculoskeletal disorders
- Any other condition judged by investigators to prevent safe enrollment, treatment, or follow-up
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Trial Site Locations
Total: 2 locations
1
Malcom Randall North Florida/South Georgia VA Medical Center
Gainesville, Florida, United States, 32608
Actively Recruiting
2
Bioresearch Partner
Miami, Florida, United States, 33143
Actively Recruiting
Research Team
C
Clinical Trial Contact
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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