Stem Cell Therapy for Dermatomyositis or Polymyositis

(IIMPACT Trial)

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Restem, LLC.

Must be taking: Immunosuppressants, Prednisone

Must not be taking: Rituxan, Biologics

Disqualifiers: Inclusion body myositis, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 31

Duration: 18 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how a stem cell treatment derived from the umbilical cord lining, known as ULSC, can aid adults with Dermatomyositis or Polymyositis, conditions that cause muscle inflammation and weakness. Researchers aim to determine if this treatment is safe and effective in reducing symptoms and whether it can help reduce steroid doses. Participants must have a diagnosis of one of these muscle inflammation conditions and currently experience symptoms like muscle weakness.

As a Phase 2, Phase 3 trial, this study measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering a chance to contribute to groundbreaking research.

Do I have to stop taking my current medications for the trial?

The trial does not require you to stop your current medications. You must stay on stable doses of your immunosuppressants and steroids unless there's a clinical reason to change them. If you're on steroids, the trial includes a plan to gradually reduce your dose after the second infusion.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that stem cells from the lining of the umbilical cord (ULSC) have been safe in past studies. These studies reported no harmful effects directly linked to the treatment, suggesting that ULSC is generally safe for people. Participants received ULSC through an IV, and researchers closely monitored the treatment for any negative effects.

Moreover, the FDA has fast-tracked ULSC therapy for idiopathic inflammatory myopathy, which includes dermatomyositis and polymyositis. This fast-tracking indicates that the treatment is under quicker review due to its promise and safety.

Overall, current evidence supports the safety of ULSC for humans, with no major safety concerns reported in previous trials. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for DM/PM?

Unlike the standard care for dermatomyositis or polymyositis, which often includes corticosteroids and immunosuppressants, ULSC (Umbilical cord-derived stem cells) offers a novel approach by using stem cells to potentially reduce inflammation and repair damaged tissues. Researchers are excited about ULSC because it harnesses the regenerative properties of stem cells, which might lead to longer-lasting improvements and fewer side effects compared to traditional medications. This treatment is distinct in its delivery method, as it's administered through intravenous infusions, potentially offering a more direct and systemic impact on the condition.

What evidence suggests that ULSC might be an effective treatment for DM/PM?

Research has shown that stem cells from the lining of the umbilical cord might help treat dermatomyositis (DM) and polymyositis (PM). In earlier studies, this treatment was found to be safe, with no harmful effects observed in patients. Recipients of the treatment experienced stronger muscles and reduced inflammation, which are main symptoms of DM and PM. In this trial, participants in Cohort 1 will receive the stem cell treatment first, followed by a placebo, while those in Cohort 2 will receive a placebo first, followed by the stem cell treatment. Researchers are also investigating whether this treatment can reduce the need for steroids, potentially avoiding the long-term side effects of steroid use. Early results suggest that this stem cell treatment could be a beneficial option for people with these conditions. ^{1 3 4 5 6}

Who Is on the Research Team?

Michael Bubb, MD

Principal Investigator

Malcom Randall North Florida/South Georgia VA Medical Center

Are You a Good Fit for This Trial?

This trial is for adults with Dermatomyositis/Polymyositis (DM/PM), meeting the EULAR/ACR 2017 criteria and having myositis-associated antibodies or other evaluations. Participants must commit to a 25-month study period, including in-person and virtual visits, and be willing to receive both ULSC and placebo treatments.

Inclusion Criteria

Participants must have the ability to comply with the requirements of the study

Participant must have the ability to understand and provide written informed consent

Participants must either be positive for a myositis-associated antibody or have undergone evaluation to exclude mimics as deemed appropriate by the Investigator

Exclusion Criteria

Hypersensitivity to study product components including history of hypersensitivity to dimethyl sulfoxide (DMSO)

Pregnant or lactating participants

History of prevalent noncompliance with medical therapy

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 3 doses of ULSC and 3 doses of placebo in a cross-over design, with infusions 3 months apart

18 months

19 in-person visits, 4 virtual visits

Steroid Tapering

For participants on steroid therapy, tapering begins two weeks after the second infusion

4 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including self-reported questionnaires, physical exams, and various tests

12 months

Follow-up visits after each dose and up to 12 months after final dose

What Are the Treatments Tested in This Trial?

Interventions

• ULSC

Trial Overview The trial tests the safety and effectiveness of umbilical cord lining-derived stem cells (ULSC) versus placebo in treating DM/PM symptoms. It involves three IV doses of ULSC (150 million cells each) compared against saline placebo, assessing symptom improvement and potential steroid dose reduction.

How Is the Trial Designed?

Group I: Cohort 2: Placebo first; ULSC second Experimental Treatment 2 Interventions

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.

Group II: Cohort 1: ULSC first; Placebo second Experimental Treatment 2 Interventions

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.

Who Is Running the Clinical Trial?

Restem, LLC.

Lead Sponsor

Trials

2

Recruited

30+

Citations

1. acrabstracts.org acrabstracts.org/abstract/safety-and-efficacy-data-from-a-phase-i-trial-of-umbilical-lining-derived-stem-cells-ulsc-in-adult-dermatomyositis-polymyositis/

Safety and Efficacy Data from a Phase I Trial of Umbilical ... Here we have used ULSC in a phase I trial of DM/PM to evaluate the safety and tolerability of USLC in DM/PM, as well as a number of exploratory efficacy ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07160205

NCT07160205 | Safety and Efficacy of ULSC on Disease ... 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 ...

3. firstwordpharma.com firstwordpharma.com/story/5913670

RESTEM Presents Phase 1 Data of its ULSC Program in ... Oral presentation at ACR highlights safety and efficacy data from the Phase 1 trial of umbilical cord outer lining-derived stem cells (ULSCs) in

4. cgtlive.com cgtlive.com/view/restem-ulsc-therapy-restem-l-fast-tracked-fda-idiopathic-inflammatory-myopathy

RESTEM's ULSC Therapy Restem-L Fast Tracked by FDA ... Taken together, the overall human experience with the intravenous infusion of ULSC has demonstrated an absence of adverse outcomes attributable ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9746473/

Therapeutic potential of mesenchymal stem cells for refractory ... There are currently two clinical trials based on human umbilical cord-derived MSC therapy for dermatomyositis by intravenous infusion (Table 3).

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07160205

NCT07160205 | Safety and Efficacy of ULSC on Disease ... The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell (ULSC) product performs when treating ...