Ublituximab for Necrotizing Myositis

(SUMMIT Trial)

Not yet recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Must be taking: Immunosuppressants, Glucocorticoids

Must not be taking: Biologics, JAK inhibitors

Disqualifiers: End-stage myositis, Severe liver disease, Malignancy, Chronic infections, 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: 7

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ublituximab, a monoclonal antibody therapy, as a combination treatment for individuals with early, active necrotizing myopathy. This muscle disease occurs when the immune system mistakenly attacks healthy muscle tissue. Researchers aim to determine the effectiveness of ublituximab when added to existing treatments compared to a placebo. The trial includes two groups: one receives ublituximab first, while the other starts with a placebo before switching to the medication. Suitable participants have been diagnosed with this muscle disease, experience muscle weakness, and possess specific antibodies linked to the condition. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of participants.

Do I need to stop my current medications to join the trial?

You can continue taking one background immunosuppressant medication, like methotrexate or mycophenolate, as long as it has been stable for at least 12 weeks before joining the trial. If you're on glucocorticoids, the dose must be stable for at least 4 weeks before joining. Other medications not listed may need to be stopped, but the protocol doesn't specify all details.

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

Research has shown that ublituximab has been tested in at least one human trial, providing some safety information. Although this specific study does not offer detailed safety results, reaching this stage is a positive sign for the treatment.

In other studies, similar treatments like rituximab have been used for immune-related conditions. These treatments are generally considered safe, especially for patients who have not responded well to other therapies. While this does not guarantee the same for ublituximab, it offers some reassurance about its potential safety.

Overall, the current stage of the trial suggests that ublituximab is likely well-tolerated so far. However, staying updated on any new findings as the trial progresses is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for necrotizing myositis?

Unlike the standard treatments for necrotizing myositis, which typically involve high doses of corticosteroids and immunosuppressants, ublituximab offers a promising new approach. Ublituximab is a monoclonal antibody that targets a specific protein on B-cells, which are part of the immune system. This targeted action could potentially reduce inflammation and muscle damage more effectively and with fewer side effects than current therapies. Researchers are excited about ublituximab because of its potential to provide a more precise and efficient way to manage this debilitating condition.

What evidence suggests that ublituximab might be an effective treatment for necrotizing myositis?

This trial will evaluate the effectiveness of ublituximab for necrotizing myositis. Studies have shown that ublituximab may benefit people with immune-mediated necrotizing myopathy, a condition where the immune system mistakenly attacks the muscles. Research has indicated that similar treatments, like rituximab, have been effective for related muscle conditions, especially when other treatments fail. Ublituximab targets specific immune cells, potentially reducing harmful inflammation. Previous patients with related conditions experienced improved muscle strength and fewer symptoms. While specific data on ublituximab's effectiveness in necrotizing myositis is still emerging, its mechanism suggests promise. ^{1 3 4 6 7}

Who Is on the Research Team?

Julie Paik, M.D., M.H.S.

Principal Investigator

Johns Hopkins Hospital: Division of Rheumatology

Are You a Good Fit for This Trial?

This trial is for people with early, active immune-mediated necrotizing myopathy who have specific autoantibodies. It's not clear what the exclusion criteria are, but typically these might include other health conditions or treatments that could interfere with the study.

Inclusion Criteria

I have been diagnosed with IMNM and test positive for Anti-SRP or Anti-HMGCR antibodies.

My muscle weakness or high CK levels due to IMNM started less than a year ago.

Definite or probable IIM per the 2017 EULAR/ACR classification criteria

Exclusion Criteria

I have been diagnosed with a specific muscle disease other than PM or DM.

I have severe muscle damage or atrophy that affects my mobility.

I haven't taken any immune system altering drugs in the last 12 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Ublituximab or placebo as first-line combination therapy, with doses at Day 0, Week 2, Week 24, and Week 48

48 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

3 visits (in-person)

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Ublituximab

Trial Overview The trial is testing Ublituximab as an additional first-line treatment compared to a placebo. The goal is to see how well it works over 24 weeks using a specific scoring system developed by rheumatology and myositis experts.

How Is the Trial Designed?

Group I: Initial Ublituximab Active Group Experimental Treatment 2 Interventions

Receives 150 mg Ublituximab at Day 0 and 450 mg Ublituximab at Week 2, followed by 450 mgs of Ublituximab at Weeks 24 and 48. At Week 26, will receive a Placebo dose of 450 mg to maintain the blind.

Group II: Initial Placebo of ublituximab Group Experimental Treatment 2 Interventions

Receives 150 mg Placebo at Day 0 and 450 mg Placebo at Week 2. Receives 150 mg Ublituximab at Week 24 followed by 450 mg Ublituximab at Weeks 26 and 48.

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+

Rho Federal Systems Division, Inc.

Industry Sponsor

Trials

44

Recruited

15,000+

TG Therapeutics

Collaborator

Trials

1

Recruited

20+

Autoimmunity Centers of Excellence

Collaborator

Trials

23

Recruited

1,400+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07103746

NCT07103746 | Ublituximab in Autoantibody Positive ... TIS is a composite endpoint based on improvement in the 6 Disease Activity Core Set Measure (CSM) scores and ranges from 0 to 100 (2016 American College of ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41161910/

Updates in the Management of Immune-Mediated ... Glucocorticoids remain first-line, but steroid-sparing agents have improved long-term outcomes. IVIG and rituximab are effective in refractory cases, and ...

3. withpower.com withpower.com/trial/phase-2-myositis-10-2025-8dd32

Ublituximab for Necrotizing Myositis (SUMMIT Trial) This trial is for people with early, active immune-mediated necrotizing myopathy who have specific autoantibodies. It's not clear what the exclusion criteria ...

4. frontiersin.org frontiersin.org/journals/immunology/articles/10.3389/fimmu.2022.1051609/full

Efficacy and safety of rituximab treatment in patients with ... RTX may be an effective and relatively safe treatment choice in patients with IIMs, especially for refractory cases.

5. sciencedirect.com sciencedirect.com/science/article/pii/S1878747923009455

Immunotherapies for Immune-Mediated Myopathies They reported that 83% of randomized patients met the definition of improvement by around 20 weeks and that the treatment was well tolerated and had a ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7958167/

Rituximab in the treatment of immune-mediated necrotizing ... Our data may support the use of RTX as an effective treatment strategy against IMNM resistant to steroids and multiple immunotherapies.

7. researchgate.net researchgate.net/publication/397060499_Updates_in_the_Management_of_Immune-Mediated_Necrotizing_Myopathy

Updates in the Management of Immune-Mediated ... Safety data were also collected. Results. Twenty-six patients with IIM treated with hf-SCIg for remission induction (n = 5) or maintenance ...