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)

Conditions

• DERMATOMYOSITIS OR POLYMYOSITIS
• Idiopathic Inflammatory Myositis (IIM)

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

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).
• Placebo (no cells) — BIOLOGICAL
  The Placebo will be 250 ml sterile saline with vehicle (total volume of 260 mL) IV bag for infusion.

Study Details

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.

Key Dates

Start date

Jan 6, 2026

Status verified

Apr 2026

Primary completion

Sep 30, 2028

Completion

Aug 31, 2029

Study Design

Enrollment

40 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1: ULSC first; Placebo second
  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.
• Experimental: Cohort 2: Placebo first; ULSC second
  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.

Primary Outcome Measure

Safety based on Adverse Events (AEs) and Serious Adverse Events (SAEs) that begin during or following treatment infusion. [ Time Frame: 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. ]

Central Contacts

• Clinical Trial Contact
  352-278-8838
  [email protected]

Locations (2)

Find similar trials in Gainesville, FL

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