ABC008 for Inclusion Body Myositis

Not currently recruiting at 49 trial locations

Overseen By Colin Quinn, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Abcuro, Inc.

Disqualifiers: Other myositis, Autoimmune diseases, others

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ABC008 to determine its effectiveness for people with Inclusion Body Myositis (IBM), a condition that causes muscle weakness and difficulty moving. Researchers aim to assess the safety and efficacy of ABC008 by comparing it to a placebo (a substance with no active treatment). Participants will receive varying doses of ABC008 to identify the most effective one. Individuals diagnosed with IBM who can walk short distances with or without a device may be suitable for this study. As a Phase 2, Phase 3 trial, this study measures the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

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

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that ABC008 is likely to be safe for humans?

Research has shown that ABC008 has promising safety results from earlier studies. These studies found that ABC008 targets harmful T cells, which attack muscle tissue in people with Inclusion Body Myositis. Importantly, researchers reported no major safety issues, suggesting that ABC008 is generally well-tolerated by patients. While some minor side effects may occur, no serious problems have been noted so far. This information suggests that ABC008 could be a safe option for those considering joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Inclusion Body Myositis?

Researchers are excited about ABC008 for Inclusion Body Myositis because it targets a new mechanism that could potentially slow down the progression of this muscle-wasting disease. Unlike existing treatments, which primarily focus on managing symptoms, ABC008 is designed to directly address the underlying causes of muscle degeneration. By using innovative dosing options—0.5 mg/kg and 2.0 mg/kg—researchers are exploring its efficacy across different patient groups. This approach offers hope for more effective management of a condition that currently has limited treatment options.

What evidence suggests that this trial's treatments could be effective for Inclusion Body Myositis?

Research has shown that ABC008 may help treat Inclusion Body Myositis (IBM). An initial study found that ABC008 reduced certain immune cells, known as KLRG1+ CD8+ T cells and T-LGLs, which are believed to contribute to the disease. These cells are part of the immune system, and reducing them might slow IBM's progression. This early evidence suggests that ABC008 could manage IBM symptoms by addressing its root causes. Although more research is needed, these findings offer hope for people living with IBM. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults over 40 with Inclusion Body Myositis who can walk and rise from a chair unassisted. They must weigh between 40-150 kg and meet specific diagnostic criteria. Excluded are those with other myopathies, severe arthritis affecting assessments, or autoimmune diseases like lupus or rheumatoid arthritis.

Inclusion Criteria

I can walk 3 meters and sit down by myself, using aids if needed but without another person's help.

I have been diagnosed with IBM as per ENMC criteria and have histopathology results.

I am over 40 years old.

Exclusion Criteria

I have a condition that makes it hard to measure my muscle strength or do physical tests.

I have an autoimmune disease but it's not severe and under control.

I do not have any muscle conditions other than IBM.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Sentinel cohort receives first three doses of ABC008, safety data evaluated by DSMB

20 weeks

Multiple visits for dosing and monitoring

Treatment Part B

Double-blind safety and efficacy cohort receives ABC008 or placebo

76 weeks

Regular visits for assessments and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• ABC008

Trial Overview The study tests ABC008's effectiveness and safety in treating Inclusion Body Myositis. Participants will be randomly assigned to receive either ABC008 or a placebo without knowing which one they're getting (double-blind).

How Is the Trial Designed?

Group I: 2.0 mg/kg ABC008 Active Control 1 Intervention

Part A - ABC008 N=12 Part B - ABC008 N= 67

Group II: 0.5 mg/kg ABC008 Active Control 1 Intervention

Part A - ABC008 N=12 Part B - ABC008 N= 67

Group III: Placebo Placebo Group 1 Intervention

Part A - Placebo N= 6 Part B - Placebo N= 67

Who Is Running the Clinical Trial?

Abcuro, Inc.

Lead Sponsor

Trials

4

Recruited

540+

Syneos Health

Collaborator

Trials

181

Recruited

69,600+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05721573

A Study to Evaluate the Efficacy and Safety of ABC008 ... A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects ...

2. uclahealth.org uclahealth.org/clinical-trials/study-evaluate-efficacy-and-safety-abc008-inclusion-body

A Study to Evaluate the Efficacy and Safety of ABC008 ... A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis ... Documented histopathology results must be available prior to Baseline (Day 1) ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11879327/

Inclusion body myositis and immunosenescence Early data from a pilot study in three IBM patients demonstrated that ABC008 can selectively lower the proportion of circulating KLRG1+ CD8+ T cells and T-LGLs ...

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 ... Trial to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis.” After an initial flurry of ...

5. abcuro.com abcuro.com/wp-content/uploads/ACR-Late-Breaker-102521-1.pdf

Depletion of KLRG1+ T cells in a First-in-Human Clinical ... Rheumatol Int 2021. • Inclusion body myositis (IBM), a relentlessly progressive autoimmune skeletal muscle disease, has no effective available pharmacological.

6. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT05721573

ABC008 for Inclusion Body Myositis 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. abcuro.com abcuro.com/uncategorized/abcuro-presents-additional-data-from-ongoing-phase-1-clinical-trial-of-abc008-in-inclusion-body-myositis-ibm-at-gcom-2022/

Abcuro Presents Additional Data from Ongoing Phase 1 ... The additional data finds that ABC008 demonstrates proof-of-mechanism for depleting highly cytotoxic T cells, which attack and destroy muscle tissue in IBM.

8. hss.edu hss.edu/research/clinical-trials/randomized-double-blinid-placebo-multicenter-efficacy-safety-abc008-inclusion-body-myositis

A Phase II/III Randomized, Double-blind, Placebo- ... This is a his study soughts to find the safety and efficacy of subcutaneous injections of ABC008, a humanized a fucosylated monoclonal antibody directed ...