Explore Clinical Trials

At Cure JM, we believe that research holds the key to better treatments. Clinical trials can help us get there. Participating in a clinical trial is one way you can contribute to advancing care for juvenile myositis and access new therapies before they’re widely available.

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Helpful Resources from the NIH
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Now Enrolling
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Medical professional works with a patient

Helpful Resources from the National Institutes of Health

These trusted resources from the National Institutes of Health (NIH) can help you better understand how clinical trials work, especially for children battling rare diseases:

These resources can help you feel informed, confident, and prepared to explore research options for you or your family.

Considering a clinical trial can feel overwhelming. Cure JM can connect families with parents and patients who have participated in clinical trials, offering the opportunity to ask questions and hear directly about their experience.

To learn more or request a connection, contact James Tealy, Family Education Director, at james.tealy@curejm.org.

Coming Soon

We’re excited to share that Children’s Hospital of Pittsburgh (CHP), under the leadership of Dr. Kathryn Torok, has been approved as a CAR‑T site for eligible JM patients. This is an important step forward for families, and more information on recruitment will be available soon.

Now Enrolling Clinical Trials

RESET-Myositis Trial: CAR-T Cell Therapy (CABA-201)

A phase 1/ 2 clinical trial, investigating a treatment that uses your own T cells to treat myositis.

Contact Info:

Resources:

Read more about this trial on clinicaltrials.gov here 

View More Details
Who Can Participate:
  • Children ages 6+ and adults with active myositis (JDM, JPM, or adult forms)
  • Must have active disease
  • Learn more here

A study physician will determine final eligibility.

How It Works:
  • T cells are extracted from the patient, reengineered into CAR T cells, and returned to the patient to target harmful immune cells that may be causing disease
  • All participants in the RESET-Myositis Trial will receive CAR-T cell therapy— there is no placebo group in this study
  • Learn more about the CAR-T process and how it works here

The trial is enrolling at multiple locations across the U.S., including:

Sites enrolling pediatric patients age 6-26*

  • Chicago, IL (Comer Children’s, University of Chicago)
  • San Francisco, CA (UCSF)
  • Philadelphia, PA (CHOP) 
  • Ann Arbor, MI (University of Michigan) 
  • New York, NY (Hospital for Special Surgery)
  • New York, NY (Memorial Sloan Kettering Cancer Center)
  • Chicago, IL (Lurie Children’s Hospital)
  • The Bronx, New York (Children’s Hospital at Montefiore
  • Seattle, WA (Seattle Children’s)
  • Aurora, Colorado (Children’s Hospital Colorado)
  • Not yet recruiting: Bethesda, Maryland (NIH)
  • Not yet recruiting: Pittsburgh, PA (UPMC Children’s Hospital of Pittsburgh)
  • More locations listed here

Sites enrolling age 18+

  • Orange County, CA (UC Irvine)
  • Jacksonville, FL (Mayo Clinic)
  • Nashville, TN (Vanderbilt)
  • Houston, TX (Houston Methodist & MD Anderson)
  • Kansas City, MO (UMKC)
  • Rochester, MN (Mayo Clinic)
  • Portland, OR (OHSU)
  • Atlanta, GA (Emory)
  • United Kingdom: London, Manchester, Salford
  • Not yet recruiting: Pittsburgh, PA (UPMC Arthritis and Autoimmunity Center)
  • More locations listed here

*Each children’s hospital may have slightly different maximum age guidelines, please contact clinicaltrials@cabalettabio.com to learn more

Travel:
  • Travel to one of the sites may be covered, please contact to learn more

Talk to your doctor or email the study team at clinicaltrials@cabalettabio.com

A Phase 1 Study of FT819 in B‑cell Mediated Autoimmune Diseases

This study is testing FT819, an investigational off‑the‑shelf CAR‑T cell therapy designed to target and eliminate B cells involved in autoimmune disease. FT819 is made from a single healthy donor and is available on demand, without the need for leukapheresis.

Contact Info: 

  • Sponsor: Fate Therapeutics
  • Email: clinicaltrials@fatetherapeutics.com
  • Phone: 858‑875‑1800

Resources:

Read more about this trial on clinicaltrials.gov here 

View More Details

Who Can Participate

  • Ages 12 to 70
  • Diagnosed with active B‑cell–mediated autoimmune disease (SLE, AAV, IIM, or SSc)
  • Have tried at least two prior treatments that were not effective
  • Adequate organ function
  • Able to provide consent/assent
  • Must weigh at least 50 kg (110 lbs)
  • Key Exclusions
  • Pregnancy or breastfeeding
  • Significant heart, lung, liver, or kidney impairment
  • Active or recent serious infections
  • Prior CAR‑T therapy or organ transplant
  • Recent cancer
  • Allergies to study medications

What the Study Involves

Participants may receive FT819:

  • With or without chemotherapy (fludarabine, cyclophosphamide, or bendamustine)
  • As a single dose or two doses, depending on study group
  • As an IV infusion Hospital stay is typically 3 days.

The study will evaluate:

  • Safety and side effects
  • How FT819 behaves in the body
  • Effects on B‑cell activity
  • Improvements in disease activity and quality of life (SF‑36)

Study Locations (Selected)

This study is recruiting at 18 sites across the U.S. and internationally, including:

  • University of California Irvine (CA)
  • University of Minnesota (MN)
  • Duke University (NC)
  • Montefiore Medical Center (NY)
  • University of Oklahoma (OK)
  • Jefferson Einstein Hospital (PA)
  • Paris, France
  • Uppsala University, Sweden
  • Cambridge & London, United Kingdom

Descartes-08 for Children, Adolescents, and Young Adults with JDM

A Phase 1/2 clinical trial evaluating the safety, tolerability, and potential effectiveness of Descartes-08, an investigational cell therapy for children, adolescents, and young adults living with childhood-onset systemic lupus erythematosus (SLE), ANCA-associated vasculitis (AAV), juvenile myasthenia gravis, and juvenile dermatomyositis (JDM).

Contact Info:

Resources:

Read more about this trial on clinicaltrials.gov here

View More Details
Who Can Participate:

  • Ages 12 and older

  • Confirmed diagnosis of childhood-onset juvenile dermatomyositis

  • Moderate disease activity and prior systemic treatment

  • Ability (patient or guardian) to provide written informed consent

A study physician will determine final eligibility.

How It Works:

Descartes-08 is a cell-based therapy designed to modulate the immune system and reduce autoimmune inflammation.
The study is interventional, meaning participants receive an investigational drug under close supervision to assess safety and response.

Locations:

  • St. Louis, Missouri, United States, 63110
  • Washington D.C., District of Columbia, United States, 20010

Participant Benefits:

  • Study-related care and monitoring at no cost

  • Access to an emerging therapy being investigated for several childhood autoimmune conditions

  • Contribution to research that may improve future treatments for JDM and related diseases

Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis

A Phase 2 clinical trial evaluating whether topical ruxolitinib 1.5% cream can safely and effectively treat refractory cutaneous dermatomyositis — skin disease that has not improved with standard topical and systemic treatments.

Contact Info:

Read more about this trial on clinicaltrials.gov here

View More Details

Who Can Participate

Adults 18–89 years old with:

  • Classic dermatomyositis (CD), juvenile‑onset dermatomyositis (JD), or amyopathic dermatomyositis (AD)

  • Active skin disease (CDASI activity score >6; BSA 1–20%)

  • Prior systemic treatment (e.g., steroids, methotrexate, MMF, IVIG, etc.)

  • Prior failure of at least one topical medication

  • Stable systemic regimen for ≥60 days

A study physician will determine final eligibility.

What the Study Involves

  • Open‑label, single‑arm pilot study

  • Participants apply ruxolitinib 1.5% cream to active skin lesions

  • Evaluations at 8 and 12 weeks using CDASI (Cutaneous Dermatomyositis Activity and Severity Index)

Why This Matters

Cutaneous dermatomyositis can be painful, persistent, and difficult to treat. This study explores whether a targeted topical JAK inhibitor can reduce inflammation and improve skin symptoms for patients who haven’t responded to other options.

FAST for DM (Fatty Acid Supplementation Trial for Dermatomyositis)

The FAST for DM Study is evaluating whether omega-3 fatty acids from fish oil, combined with a healthy diet based on the Dietary Guidelines for Americans, can help reduce inflammation and improve muscle strength, function, and quality of life for people living with Dermatomyositis (DM) and Juvenile Dermatomyositis (JDM). Participants will receive dietary coaching and take either fish-oil or placebo capsules for six months, with an optional open-label 12-week extension.

Contact Info:

  • Sponsor: National Institutes of Health (NIH)
  • Principal Investigator: Lisa G. Rider, M.D.
  • Recruitment Office: NIH Clinical Center Office of Patient Recruitment (OPR)
  • Phone: 800-411-1222 TTY: 711
  • Email: ccopr@nih.gov | fastdmsupport@nih.gov

Resources:

Read more about this trial on clinicaltrials.gov here

View More Details

Who Can Participate:

Adults 18–60 years old living in the United States who can read English and have mild to moderately active DM or JDM. Participants must be able to travel to Bethesda, Maryland for study visits, have access to the internet, and be willing to follow a healthy diet and take study capsules for 6 months. People with overlap connective-tissue diseases, severe myositis, meat or fish/shellfish allergies are not eligible.

How It Works:

Participants will be randomly assigned to receive either fish-oil or placebo capsules, taken twice daily.

All participants will follow a healthy diet with the support of a registered dietitian and attend five onsite visits over six months. These include physical exams, lab tests, imaging scans, and muscle strength assessments. Participants will also wear an activity-tracking watch for two 7-day periods.

After 24 weeks, participants may continue for an optional 12-week extension in which all receive fish-oil supplements.

Where:

National Institutes of Health Clinical Center Bethesda, Maryland 20892 View on Map

Participant Benefits:

Access to expert care and regular health assessments Personalized dietary coaching at no cost Opportunity to contribute to research on nutrition-based therapies for DM and JDM All study procedures, tests, and supplements provided free of charge.

JASMINE Study: Anifrolumab for PM, DM, and JDM

A phase 3 clinical trial, investigating a subcutaneous medication (injection) called anifrolumab, designed to reduce inflammation.

Study Leaders & Contacts:

Resources:

Read more about this trial here

View More Details

Where:

View all Sites in the U.S. and Abroad here

Who Can Join:

  • Adults 18+ with PM, DM, or JDM (polymyositis, dermatomyositis, or juvenile dermatomyositis)

  • JDM participants must have been diagnosed 10+ years ago

  • Moderate to severe disease activity

  • Learn more here

A study physician will determine final eligibility.

How It Works:

  • Anifrolumab blocks type I interferon pathways involved in immune overactivation

  • The JASMINE study is a randomized, double-blind, placebo-controlled Phase 3 trial, which means that neither the doctors or patients will know if you are receiving anifrolumab or a placebo

  • A study doctor will talk to you about what this means 

Participant Benefits:

  • Study-related care and treatment at no cost

  • Some travel support may be available

Active Clinical Trials (Not Currently Recruiting)​

ALKIVIA Trial: Efgartigimod (SC) for Myositis

The ALKIVIA study is a Phase 2/3 clinical trial evaluating an investigational treatment called efgartigimod. This medication is designed to target parts of the immune system that may be contributing to inflammation in myositis.

Researchers are studying whether this treatment is safe and effective in improving symptoms such as muscle weakness and overall disease activity.

Read more about this trial on clinicaltrials.gov here

View More Details

Who CanParticipate

(Not currently recruiting)

  • Adults ages 18 and older

  • Diagnosed with an inflammatory myopathy, including:

    • Dermatomyositis (DM)

    • Polymyositis (PM)

    • Immune-mediated necrotizing myopathy (IMNM)

    • Antisynthetase syndrome

  • Must have active disease (such as muscle weakness or signs of inflammation)

A study physician determined final eligibility.

How It Works

  • Participants were randomly assigned to receive either:

    • The investigational treatment (efgartigimod), or

    • A placebo (inactive treatment)

  • The medication is given as a subcutaneous (under the skin) injection

  • Researchers compare how participants respond to treatment over time

What Researchers Are Measuring

  • Overall improvement in disease activity

  • Muscle strength

  • Patient and physician assessments of disease activity

  • Time to meaningful improvement

Locations

This study is being conducted at multiple sites across the United States, including major academic and clinical centers. View more information here.

Important Notes

  • This study includes a placebo group

  • The trial is ongoing but not currently enrolling new participants

  • Findings from this study may help inform future treatment options for myositis

A Phase 2 Study of Empasiprubart in Adults With Dermatomyositis

This clinical trial is evaluating the safety and efficacy of empasiprubart, an investigational therapy, in adults with dermatomyositis. Participants will receive either empasiprubart or placebo for 25 weeks, followed by a long‑term safety follow‑up period.

Info:

Sponsor: argenx

Study Type

Interventional, Phase 2 Randomized, double‑blind, placebo‑controlled

View full study details on clinicaltrials.gov here

 

View More Details

Who Can Participate

Adults 18 years and older with:

  • A confirmed diagnosis of dermatomyositis or juvenile‑onset dermatomyositis

  • Active muscle disease and at least mild skin disease

  • Stable background DM treatment

  • Required vaccinations completed before starting the study

Key exclusions include: other inflammatory myopathies, significant interstitial lung disease, active infections, recent malignancy, or major uncontrolled medical conditions.

Study Duration

Approximately 92 weeks total

  • 25‑week treatment period

  • 65‑week safety follow‑up

Primary Outcomes

  • Incidence of adverse events

  • Discontinuation due to adverse events

Secondary Outcomes

  • Total Improvement Score (TIS) based on ACR/EULAR criteria

Locations

28 global sites, including:

  • United States: California, Florida, Ohio

  • Europe: Greece, Italy, Poland, Spain

  • Georgia (country)

  • Moldova

Frequently Asked Questions

Clinical trials involve potential risks and benefits. A study doctor will help you understand what’s involved and whether a trial may be right for you.

Who Can Join?

Eligibility varies by study. Some are open to children or adults; others focus on treatment-resistant (refractory) or long-standing disease. Contact the study team to learn more.

How to Get Started

  • Contact the Study Team – They can confirm eligibility and answer your questions
  • Talk to Your Doctor – Share trial info and discuss whether a study may be appropriate

  • Stay Informed – Trials evolve quickly; check back regularly for updates or new opportunities
James Tealy
  • Contact James Tealy, Cure JM’s Manager of Family & Clinician Education – James is a JM parent and can explain things in a simple, easy-to-understand way.
    Contact James at James.Tealy@curejm.org

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