ABC008 for Inclusion Body Myositis

Recruiting at 44 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

231 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called ABC008 to help people with Inclusion Body Myositis, a muscle disease with few treatment options. The drug may work by affecting certain immune cells. Researchers will check if it improves muscle strength and quality of life.

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 ABC008 safe for humans?

The treatment, also known as Abacavir (ABC), has been linked to severe allergic reactions in some people with a specific genetic marker (HLA-B*57:01). However, not everyone with this marker experiences these reactions, and other factors may play a role. In some studies, the drug was tolerated without adverse reactions in certain conditions. ^{1 2 3 4 5}

How is the drug ABC008 different from other treatments for inclusion body myositis?

ABC008 may offer a novel approach by specifically targeting CD8+ T cells, which are involved in the muscle inflammation seen in inclusion body myositis, unlike other treatments that are generally resistant to immunosuppressive therapy. ^{6 7 8 9 10}

Eligibility Criteria

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

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

Treatment Details

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).

Participant Groups

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+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Unravelling the Proteomics of HLA-B*57:01+ Antigen Presenting Cells during Abacavir Medication. [2022]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Proteomic Profiling and T Cell Receptor Usage of Abacavir Susceptible Subjects. [2022]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Abacavir alters the transcription of inflammatory cytokines in virologically suppressed, HIV-infected women. [2021]

4. Switzerland pubmed.ncbi.nlm.nih.gov

A Screening Test for HLA-B∗15:02 in a Large United States Patient Cohort Identifies Broader Risk of Carbamazepine-Induced Adverse Events. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

A transgenic mouse model for HLA-B*57:01-linked abacavir drug tolerance and reactivity. [2019]

6. England pubmed.ncbi.nlm.nih.gov

Diagnosis, pathogenesis and treatment of inclusion body myositis. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Isolated inclusion body myopathy caused by a multisystem proteinopathy-linked hnRNPA1 mutation. [2020]

8. Italy pubmed.ncbi.nlm.nih.gov

Treatment of inclusion body myositis with cyclosporin-A or tacrolimus: successful long-term management in patients with earlier active disease and concomitant autoimmune features. [2017]

9. United States pubmed.ncbi.nlm.nih.gov

HLA-DRB1 allele and autoantibody profiles in Japanese patients with inclusion body myositis. [2022]

10. India pubmed.ncbi.nlm.nih.gov

Sporadic Inclusion Body Myositis: A Clinicopathological Study. [2022]