Ulviprubart for Inclusion Body Myositis

Not currently recruiting at 20 trial locations

Overseen By Colin Quinn, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Abcuro, Inc.

Disqualifiers: Unresolved AE, Pregnancy, Other studies, 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: 10

Duration: 156 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ulviprubart, a treatment for people with Inclusion Body Myositis (IBM), a condition that causes muscle weakness and difficulty with everyday activities. The goal is to assess the long-term safety and effectiveness of this treatment. Participants who have completed specific earlier studies with ulviprubart might be suitable candidates. Those who finished either Study ABC008-IBM-101 or Study ABC008-IBM-201 may qualify for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential approval of a new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it mentions restrictions on the use of prohibited medications during the study. It's best to discuss your current medications with the study team to see if any adjustments are needed.

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

Research shows that ulviprubart is being tested for safety in patients with inclusion body myositis. Previous studies have shown promising results. In one study, ulviprubart reduced certain immune cells without causing major side effects, suggesting it may be well-tolerated.

In another study, patients received different doses of ulviprubart, including the 2.0 mg/kg dose used in the current trial. The safety results were positive in these trials.

These findings are encouraging for those considering joining the trial. While more information will help confirm safety, earlier research indicates the treatment has been well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for inclusion body myositis (IBM) focus on managing symptoms with anti-inflammatory medications or physical therapy, but they don't address the underlying causes of the disease. Ulviprubart (ABC008) is unique because it targets a specific immune system pathway, potentially slowing down the muscle damage that characterizes IBM. Delivered through a convenient subcutaneous injection every eight weeks, Ulviprubart offers a promising new approach by directly modulating immune responses, which could lead to better outcomes for patients. Researchers are excited because this treatment could mean less frequent dosing and a more targeted therapy compared to existing options.

What evidence suggests that ulviprubart might be an effective treatment for inclusion body myositis?

Research shows that ulviprubart may help treat inclusion body myositis (IBM). Studies have found that patients taking ulviprubart experienced improved muscle strength and function, addressing the muscle weakness caused by IBM. Previous trials also found that most people tolerated ulviprubart well. These findings suggest it could be an effective option for people with IBM. Although more research is needed, the early results offer promise for those considering this treatment. ^{4 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for people with Inclusion Body Myositis who finished a prior ulviprubart study (ABC008-IBM-101 or ABC008-IBM-201). Participants must understand the study, agree to follow its rules including site visits and self-injections, use effective contraception if applicable, and have shown good compliance in previous studies.

Inclusion Criteria

I am a man and will not donate sperm during the study and for 180 days after it ends.

I can read and understand the consent form and have signed it.

I completed a specific part of either Study ABC008-IBM-101 or Study ABC008-IBM-201.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Within visit window defined in Appendix 1 of the protocol

1 visit (in-person)

Baseline

Baseline assessments and initiation of long-term extension treatment

Day 1

1 visit (in-person)

Treatment

Participants receive ulviprubart 2.0 mg/kg via subcutaneous injection every eight weeks

156 weeks

Visits every 8 weeks (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ulviprubart (ABC008)

Trial Overview The trial tests long-term safety and effectiveness of ulviprubart (ABC008) in those with IBM. It's an open-label, multicenter study where all participants receive the drug after completing earlier phases of ulviprubart research.

How Is the Trial Designed?

Group I: Ulviprubart (ABC008) 2.0 mg/kg SC Experimental Treatment 1 Intervention

All eligible subjects, regardless of treatment assignment or dose level in their initial study, will be administered ulviprubart at a dose of 2.0 mg/kg via subcutaneous (SC) injection Q8W.

Who Is Running the Clinical Trial?

Abcuro, Inc.

Lead Sponsor

Trials

4

Recruited

540+

Published Research Related to This Trial

In a large study involving 251 participants with inclusion body myositis, bimagrumab demonstrated a good safety profile, with no significant adverse cardiac effects and a similar rate of adverse events compared to placebo.

However, bimagrumab did not show any improvement in the primary outcome measure, the 6-minute walking distance, indicating that while it is safe, it may not be effective in enhancing mobility in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial. Hanna, MG., Badrising, UA., Benveniste, O., et al.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06450886

Long-term Extension Study of Ulviprubart (ABC008) in ... ABC008-IBM-202 is an open-label, multicenter study to evaluate the safety and efficacy of long-term administration of ulviprubart (ABC008) in subjects with ...

2. abcuro.com abcuro.com/clinical-trials/

Clinical Trials Abcuro has evaluated ulviprubart (ABC008) in a Phase 1 study in patients with inclusion body myositis, or IBM, at several clinics in Australia.

3. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT05721573

ABC008 for Inclusion Body Myositis - UCSF Clinical Trials A Phase II/III randomized, double-blind, placebo-controlled, multicenter study to determine the efficacy and safety of ABC008 in the treatment of subjects with ...

4. myositis.org myositis.org/blog/seeing-the-other-side-on-being-a-clinical-trial-participant/

Seeing the other side: On being a clinical trial participant As a person living with inclusion body myositis (IBM), I had been looking for opportunities to participate in a clinical trial, eager to “see it from the other ...

5. clinicaltrials.eu clinicaltrials.eu/trial/study-on-long-term-safety-of-ulviprubart-for-patients-with-inclusion-body-myositis/

Long-term safety and effectiveness study of Ulviprubart in ... This study focuses on evaluating a treatment for people with inclusion body myositis, a rare muscle disorder that causes progressive muscle ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05721573

NCT05721573 | A Study to Evaluate the Efficacy and ... A Phase II/III randomized, double-blind, placebo-controlled, multicenter study to determine the efficacy and safety of ABC008 in the treatment of subjects with ...

7. neurology.org neurology.org/doi/10.1212/WNL.0000000000211798

Results from a 48-week, Open-label, Phase 1 Study in ... Ulviprubart led to deep and selective depletion of peripheral blood KLRG1+ T cells in patients with IBM. Together with the favorable safety ...

8. abcuro.com abcuro.com/wp-content/uploads/25ABC005_AAN-Ph1-PK-PD-Safety-PO.pdf

Pharmacokinetics, pharmacodynamics, and safety profile ... Data are from n=3, n=3, and n=5 patients who received a single dose of 0.1 mg/kg, 0.5 mg/kg, or 2.0 mg/kg ulviprubart, respectively. Baseline is ...