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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 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2026-04-27

No results posted yet for this study

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

Interventions

BIOLOGICAL

ULSC (1.5 x 10^8 cells/dose)

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).

BIOLOGICAL

Placebo (no cells)

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

Sponsors & Collaborators

  • Restem, LLC.

    lead INDUSTRY

Principal Investigators

  • Michael Bubb, MD · Malcom Randall North Florida/South Georgia VA Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-01-06
Primary Completion
2028-09-30
Completion
2029-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07160205 on ClinicalTrials.gov