Stem Cell Therapy for Inclusion Body Myositis

Recruiting at -1 trial locations

Overseen By Andrew J Heim

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: University of Kansas Medical Center

Must not be taking: Testosterone, Cannabis, Corticosteroids, others

Disqualifiers: Chronic infection, Cancer, Drug abuse, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

9 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that participants stop taking certain medications, such as corticosteroids, intravenous immunoglobulin (IVIg), and other immunosuppressants, within specific time frames before screening. Additionally, participants must not be on certain drugs like anticoagulants close to the procedure and should not be using other myositis treatments or cell/gene therapies.

What data supports the effectiveness of the treatment Adipose Derived Regenerative Cells for Inclusion Body Myositis?

Research on similar treatments, like mesenchymal stem cells, shows they can improve muscle repair and strength in conditions like Duchenne muscular dystrophy, suggesting potential benefits for muscle regeneration in Inclusion Body Myositis. ^{1 2 3 4 5}

Is stem cell therapy generally safe for humans?

Research on stem cell therapies, including those derived from adipose tissue, suggests they are generally safe in humans. For example, a study on a different type of stem cell therapy for Duchenne Muscular Dystrophy reported no adverse events up to 21 months after treatment, indicating a good safety profile. ^{4 5 6 7 8}

How does the treatment Adipose Derived Regenerative Cells differ from other treatments for inclusion body myositis?

Adipose Derived Regenerative Cells (stem cells from fat tissue) offer a novel approach by potentially enhancing muscle repair and regeneration, which is impaired in inclusion body myositis due to aging and cellular senescence. Unlike traditional treatments that may not address the underlying muscle degeneration, this therapy aims to replenish and rejuvenate the muscle's regenerative capacity. ^{1 2 9 10 11}

Research Team

Mazen Dimachkie

Principal Investigator

University of Kansas Medical Center

Eligibility Criteria

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.

Inclusion Criteria

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

Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM

Able to give informed consent

Exclusion Criteria

I haven't taken systemic steroids or immunosuppressants recently, except for local or specific minor uses.

I have taken blood thinners within 1 hour before liposuction.

My blood test shows high alkaline phosphatase, suggesting I might have Paget's disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part 1 - Standard of Care

Participants receive standard of care treatment and are monitored for safety

12 months

Regular visits as per standard of care

Part 2 - Stem Cell Injection

Participants receive stem cell injections in the forearm and thigh muscles

6 months

Staggered enrollment with safety assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• Adipose Derived Regenerative Cells

Trial Overview 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.

Participant Groups

Group I: Standard of Care Active Control 1 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 Injection Active Control 1 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.

Who Is Running the Clinical Trial?

University of Kansas Medical Center

Lead Sponsor

Trials

527

Recruited

181,000+

Findings from Research

The DT-DEC01 cell therapy demonstrated a strong safety profile with no adverse events reported up to 21 months after administration, indicating it is a safe treatment option for patients with Duchenne Muscular Dystrophy (DMD).

Functional improvements were observed in patients, including better performance in the 6-Minute Walk Test and other assessments, suggesting that DT-DEC01 may effectively enhance muscle function and overall health in DMD patients over a 12-month period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients: A 12 - Month Follow-Up Study After Systemic Intraosseous Administration. Siemionow, M., Biegański, G., Niezgoda, A., et al.[2023]

References

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

Increased aging in primary muscle cultures of sporadic inclusion-body myositis. [2017]

2. Germany pubmed.ncbi.nlm.nih.gov

Senescent fibro-adipogenic progenitors are potential drivers of pathology in inclusion body myositis. [2023]

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

Safety and efficacy of strength training in patients with sporadic inclusion body myositis. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Aggregate mesenchymal stem cell delivery ameliorates the regenerative niche for muscle repair. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Myostatin genetic inactivation inhibits myogenesis by muscle-derived stem cells in vitro but not when implanted in the mdx mouse muscle. [2021]

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

Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients: A 12 - Month Follow-Up Study After Systemic Intraosseous Administration. [2023]

7. United Arab Emirates pubmed.ncbi.nlm.nih.gov

The potential for treatment of skeletal muscle disorders with adipose-derived stem cells. [2022]

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

Transplantation of PSC-derived myogenic progenitors counteracts disease phenotypes in FSHD mice. [2023]

9. England pubmed.ncbi.nlm.nih.gov

Expression of myogenic regulatory factors and myo-endothelial remodeling in sporadic inclusion body myositis. [2021]

10. England pubmed.ncbi.nlm.nih.gov

Donor satellite cell engraftment is significantly augmented when the host niche is preserved and endogenous satellite cells are incapacitated. [2021]

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

Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles. [2021]