What is the mechanism of action of this treatment?

Inclusion Body Myositis (IBM) treatments, such as Adipose-Derived Regenerative Cells (ADRCs), primarily aim to regenerate muscle tissue and reduce inflammation. ADRCs are believed to work by introducing regenerative cells that can differentiate into muscle cells, thereby potentially reversing muscle degeneration. Additionally, these cells may have anti-inflammatory properties that help mitigate the chronic inflammation characteristic of IBM. This dual action is crucial for IBM patients as it addresses both muscle weakness and inflammation, potentially slowing disease progression and improving muscle function.

What side effects are associated with this type of intervention?

Common side effects of treatments for Inclusion Body Myositis (IBM) that aim to regenerate muscle tissue and reduce inflammation, such as Adipose-Derived Regenerative Cells (ADRCs), include local reactions at the injection site, mild lymphadenopathy, and hypersensitivity reactions. These side effects are generally mild and resolve with treatment withdrawal. More severe complications, such as infections or immune reactions, are less common but possible. Larger trials are needed to confirm the long-term safety and efficacy of ADRCs.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for Inclusion Body Myositis (IBM) that focus on muscle regeneration and reducing inflammation similar to Adipose-Derived Regenerative Cells (ADRCs). Most treatments for IBM are supportive and symptomatic, including physical therapy and immunosuppressive drugs, but none have been definitively approved for muscle regeneration or significant inflammation reduction in IBM.

What other types of research exist?

Promising alternatives to Adipose-Derived Regenerative Cells (ADRCs) for Inclusion Body Myositis patients include Mesenchymal Stem Cells (MSCs), Muscle Precursor Cells (mpc), and Induced Pluripotent Stem Cells (iPSCs). These therapies have shown potential in regenerating muscle tissue and reducing inflammation in various studies.

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Regenerative Medicine

Stem Cell Therapy for Inclusion Body Myositis

N/A

Waitlist Available

Led By Mazen Dimachkie

Research Sponsored by University of Kansas Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Demonstrate being able to arise from a chair without support from another person or device. Subjects may use their arms to push up

Age at onset of weakness > 45 years

Must not have

History of chronic infection particularly HIV or Hepatitis B or C

History of cancer other than basal cell cancer less than five years prior, or other chronic serious medical illnesses

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3, 6, and 12 months post treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial tests the safety of injecting special cells from a patient's own fat into their muscles to treat Inclusion Body Myositis, a severe muscle disease. The goal is to see if these cells can help repair and strengthen the muscles.

Who is the study for?

This trial is for individuals with Inclusion Body Myositis who can get up from a chair unaided, walk at least 20 ft, meet specific diagnostic criteria, and have muscle strength scores of 6-9 in certain areas. Participants must be over 45 years old and able to consent. Exclusions include recent drug study participants, cannabis users, current smokers, those with certain medical conditions or infections, pregnant or breastfeeding women, and people not using effective birth control.

What is being tested?

The trial is testing the safety of injecting adipose derived regenerative cells into the forearm and thigh muscles of patients with Inclusion Body Myositis. It's an early-stage study involving nine subjects to see if this treatment might slow down or reverse muscle weakness.

What are the potential side effects?

While the primary focus is on safety rather than side effects, potential risks may include reactions at injection sites such as pain or infection; however detailed side effect profiles will be determined during this pilot study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can get up from a chair without help, but I can use my arms to assist.

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I started experiencing weakness after turning 45.

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I can walk at least 20 feet on my own with a walking aid but without someone's help.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of chronic infection, such as HIV or Hepatitis B/C.

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I have not had any cancer other than basal cell or serious illnesses in the last 5 years.

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I have followed all my previous treatment plans.

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I am a current smoker.

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I have taken blood thinners within 1 hour before my stem cell procedure.

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My IBM (a muscle disease) started before I was 45.

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I have an active skin infection where they plan to remove fat.

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My latest creatine kinase levels were very high due to IBM.

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My blood counts are low, and I have kidney, liver, or blood issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3, 6, and 12 months post treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3, 6, and 12 months post treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3, 6, and 12 months post treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety (Frequency and Severity of Adverse Events)

Secondary study objectives

Change in Grip Strength from Baseline

Familial benign pemphigus

Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from Baseline

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Active Control

Group I: Standard of CareActive Control1 Intervention

Standard of Care (SOC) Study: The 6 subjects in the late injection group will start on Part 1. - The Part 1 study subject participation is 12 months. Two subjects will be enrolled at each of Months 0, 3 and 6. This will include an initial assessment and SOC follow-up. Subjects will continue standard of care treatment. Part 1 study duration (with staggering included) will be 18 months. After Part 1, the late injection subjects may proceed to Part 2 depending on safety data from the early injection group (see 3. below).

Group II: Stem Cell InjectionActive Control1 Intervention

Stem Cell Injection: The three subjects randomized to early injections will proceed directly to Part 2 with staggered enrollment of 1 subject every 3 months. Once the safety data of the first subject at Month 3 is assessed, the second subject will be enrolled. Once the safety data of the first 2 subject (Subject 1 at Month 6 and Subject 2 at Month 3) are assessed, the third early injection subject will be enrolled in Part 2.

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Inclusion Body Myositis (IBM) treatments, such as Adipose-Derived Regenerative Cells (ADRCs), primarily aim to regenerate muscle tissue and reduce inflammation. ADRCs are believed to work by introducing regenerative cells that can differentiate into muscle cells, thereby potentially reversing muscle degeneration. Additionally, these cells may have anti-inflammatory properties that help mitigate the chronic inflammation characteristic of IBM. This dual action is crucial for IBM patients as it addresses both muscle weakness and inflammation, potentially slowing disease progression and improving muscle function.

Normal myogenic cells from newborn mice restore normal histology to degenerating muscles of the mdx mouse.Icing after skeletal muscle injury decreases M1 macrophage accumulation and TNF-α expression during the early phase of muscle regeneration in rats.Aligned nanofibers of decellularized muscle extracellular matrix for volumetric muscle loss.