Stem Cell Therapy for Inclusion Body Myositis

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

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test the safety of a new stem cell therapy using Adipose Derived Regenerative Cells for individuals with Inclusion Body Myositis (IBM), a condition that causes muscle weakness. Participants will receive stem cell injections in their forearm and thigh to determine if the treatment can safely slow, stop, or reverse the muscle issues associated with IBM. The trial seeks participants who began experiencing muscle weakness after age 45 and can walk at least 20 feet, even with a walking aid like a cane. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could lead to new treatment options for IBM.

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 prior data suggests that adipose derived regenerative cells are safe for patients with Inclusion Body Myositis?

Research shows that cells taken from fat tissue, known as adipose-derived regenerative cells (ADRCs), have been studied for their safety in treating conditions like Inclusion Body Myositis (IBM). Past studies generally found these cells safe when injected into muscles. For instance, one study found no serious side effects directly linked to the treatment in IBM patients.

However, some risks exist. Another study found that administering these cells to the whole body could cause blood clotting problems. These risks are less concerning for this trial because the cells are injected directly into muscles, not administered systemically.

Overall, current research suggests that ADRCs are safe for muscle injections in IBM, though caution is advised due to potential risks observed in other contexts. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Most treatments for inclusion body myositis focus on alleviating symptoms through physical therapy and medications like corticosteroids, which aim to reduce inflammation but often with limited success. Unlike these traditional approaches, the new treatment uses adipose-derived regenerative cells, which are stem cells harvested from a patient's own fat tissue. This approach promises to repair muscle damage at a cellular level, potentially leading to improved muscle function and strength. Researchers are excited because this method could offer a more targeted and regenerative solution, addressing the root cause of muscle degeneration rather than just managing symptoms.

What evidence suggests that adipose derived regenerative cells might be an effective treatment for Inclusion Body Myositis?

Research has shown that cells taken from a person's own fat, known as adipose-derived regenerative cells (ADRCs), might help treat Inclusion Body Myositis (IBM). In early studies, these cells improved muscle function in conditions similar to IBM, which involves muscle inflammation. This trial will compare ADRCs, administered through stem cell injections, with standard care treatment. This approach uses a person's own fat cells to repair muscle damage. Initial findings suggest that ADRCs could slow down or even reverse muscle weakness in IBM. While more research is needed, these early results offer promise for those exploring new treatment options. ^{1 2 3 4 6}

Who Is on the Research Team?

Mazen Dimachkie

Principal Investigator

University of Kansas Medical Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12445652/

Preclinical efficacy of adipose-derived cell therapies for the ... The therapies evaluated in this study have exhibited promising outcomes in the treatment of a preclinical model of myositis. These therapies ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896623002146

P53 Inclusion body myositis treatment with Celution ... The primary objective of this study is to assess in IBM the safety of an autologous graft consisting of adipose-derived regenerative cells (ADRCs) derived from ...

3. withpower.com withpower.com/trial/phase-myositis-inclusion-body-8-2021-ef65d

Stem Cell Therapy for Inclusion Body Myositis 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.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05032131?term=AREA%5BConditionSearch%5D(%22Myositis,%20Inclusion%20Body%22)&rank=10

Cell Therapy for IBM by Muscle Injection of ADSVF The study population will be adult patients suffering of an Inclusion Body Myositis (IBM) fulfilling the Lloyd criteria treated by sirolimus since at least 6 ...

5. clinicaltrial.be clinicaltrial.be/nl/details/1513?per_page=100&only_recruiting=0&only_eligible=0&only_active=0

Inclusion Body Myositis Treatment With Celution Processed... This study, "Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells" (IBM-ADRC) evaluates the safety and efficacy of the ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5689766/

A Safety Assessment of Adipose‐Derived Cell Therapy in ... This study reviewed the safety of adipose‐derived cell therapy. Thromboembolic complications were noted following systemic administration of cells.