Muscle Fiber Fragment Injections for Bowel Incontinence

Overseen By Mary-Clare Day, RN, BSN

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Wake Forest University Health Sciences

Must not be taking: Immunosuppressants

Disqualifiers: Anorectal disease, Inflammatory bowel, Neurologic disease, Unstable cardiac, Uncontrolled diabetes, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 4

Duration: 12 weeks

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for bowel incontinence using injections of muscle fiber fragments. The aim is to regenerate the anal sphincter muscle, aiding in bowel movement control. Known as Autologous Muscle Fiber Fragment Injections, the treatment uses the patient’s own muscle cells taken from their thigh. Suitable candidates experience at least four episodes of bowel incontinence every two weeks and have not found relief with standard treatments. As an unphased trial, this study offers a unique opportunity to contribute to groundbreaking research that could lead to new treatment options.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you require immunosuppression or have certain health conditions, you may not be eligible to participate.

What prior data suggests that Autologous Muscle Fiber Fragment Injections are safe for treating bowel incontinence?

Research shows that injecting muscle cells into the anal sphincter can improve bowel control in people with fecal incontinence. Similar treatments have focused on safety. For example, treatments using a person's own muscle cells have been tested for bowel incontinence, and these studies did not report any major safety issues.

Using the patient's own muscle cells reduces the risk of rejection by the body. This approach has generally been well-tolerated in the past. However, individual results can vary. Prospective participants should discuss any concerns with the trial team, especially if specific health conditions or allergies are present. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for bowel incontinence, which often involve medications, dietary changes, or even surgery, autologous Muscle Fiber Fragment (MFF) injections offer a novel approach by directly repairing muscle defects. These injections are unique because they use muscle fibers harvested from the patient's own quadriceps, which may enhance compatibility and reduce rejection risks. Researchers are particularly excited about this treatment because it targets the underlying muscle weakness that contributes to bowel incontinence, potentially offering a more effective and less invasive solution for patients who haven’t found relief with traditional methods.

What evidence suggests that Autologous Muscle Fiber Fragment Injections might be an effective treatment for bowel incontinence?

This trial will evaluate the effectiveness of Muscle Fiber Fragment (MFF) injections for treating bowel incontinence. Research has shown that injecting muscle cells into the anal sphincter can improve bowel control for people with fecal incontinence. These injections rebuild the muscle at the bottom of the anus, reducing stool leakage. Similar treatments using small pieces of muscle have succeeded for those with anal sphincter issues who haven't found relief with other treatments. This method uses the patient's own muscle cells, which may help the body accept and respond better to the treatment. These findings offer hope for those dealing with bowel incontinence. ^{1 2 3 4 5}

Who Is on the Research Team?

Catherine Matthews, MD

Principal Investigator

Wake Forest University Health Sciences

Are You a Good Fit for This Trial?

This trial is for adults with bowel incontinence lasting over a year, who've tried other treatments without success. They must have an anal sphincter defect and frequent incontinence episodes. Women should use birth control during the study. Excluded are those with bleeding disorders, rectal pain or diseases, recent cancer, certain infections like HIV/Hepatitis B/C, severe heart/lung/kidney conditions, uncontrolled diabetes, or recent childbirth.

Inclusion Criteria

My anal sphincter has a defect of at least 30 degrees confirmed by ultrasound.

Women of childbearing potential must use acceptable contraceptives during this study

Participants must score >10 on Cleveland Clinic Fecal Incontinence Severity Scoring System (CCIS)

Exclusion Criteria

I have anemia, or my liver enzymes or bilirubin levels are higher than normal.

I do not have severe heart, lung, kidney problems, or uncontrolled diabetes.

I have had injections to bulk up my internal anal sphincter.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive autologous muscle fiber fragment injections for the treatment of fecal incontinence

12 weeks

1 visit (in-person) for injection

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

3 visits (in-person) at months 3, 6, and 12

What Are the Treatments Tested in This Trial?

Interventions

• Autologous Muscle Fiber Fragment Injections

Trial Overview The trial tests injections of Muscle Fiber Fragments (MFF) containing muscle precursor cells to repair the anal sphincter muscle and improve bowel control. Participants will receive these injections directly into their anal sphincter to see if it can regenerate functional muscle tissue.

How Is the Trial Designed?

Group I: Muscle Fiber Fragment (MFF) injections Experimental Treatment 1 Intervention

autologous muscle fiber fragment injections, harvested in an autologous fashion from the quadriceps muscle, for the treatment of Fecal Incontinence (FI) symptoms in men and women with a demonstrated anal sphincter defect and who have failed conservative treatments

Autologous Muscle Fiber Fragment Injections is already approved in United States, European Union for the following indications:

Approved in United States as Autologous Muscle Fiber Fragments for:

• Urinary incontinence due to sphincter insufficiency
• Anal sphincter muscle regeneration

Approved in European Union as Autologous Muscle Fiber Fragments for:

• Urinary incontinence due to sphincter insufficiency

Who Is Running the Clinical Trial?

Wake Forest University Health Sciences

Lead Sponsor

Trials

1,432

Recruited

2,506,000+

Published Research Related to This Trial

Injecting rat myoblasts into the intact anal sphincter is feasible, showing that these cells can survive and integrate into the host tissue, which could be a potential treatment for anal incontinence.

In this pilot study involving nonpregnant female Sprague Dawley rats, GFP-labeled myoblasts were successfully transplanted and confirmed to be present in the anal sphincter 10 days post-injection, indicating successful cell integration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Allogenic myoblast transplantation in the rat anal sphincter. Craig, JB., Lane, FL., Nistor, G., et al.[2012]

In a phase 2 randomized controlled trial involving 24 patients, intrasphincteric injections of autologous myoblasts (AMs) demonstrated significant clinical benefits for fecal incontinence (FI) at 12 months, with a median Cleveland Clinic Incontinence (CCI) score improving from 15 to 6.5 (P = 0.006).

While both the AM and placebo groups showed improvement at 6 months, the AM group maintained significant benefits at 12 months, with a response rate of 58% compared to only 8% in the placebo group, indicating the potential long-term efficacy of AM therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous Myoblasts for the Treatment of Fecal Incontinence: Results of a Phase 2 Randomized Placebo-controlled Study (MIAS). Boyer, O., Bridoux, V., Giverne, C., et al.[2019]

Intraurethral injection of minced autologous skeletal muscle tissue is a safe and moderately effective treatment for stress urinary incontinence, with significant reductions in leakage and improved ICIQ-SF scores observed in both uncomplicated (25% cure rate) and complicated (7% cure rate) cases after 1 year.

This simpler procedure shows comparable efficacy to more complex regenerative therapies using expanded muscle-derived cells, with no development of voiding dysfunction and only minor adverse events reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intraurethral injection of autologous minced skeletal muscle: a simple surgical treatment for stress urinary incontinence. Gräs, S., Klarskov, N., Lose, G.[2014]

A multicenter, placebo-controlled study showed that injecting high doses of autologous skeletal muscle-derived cells significantly improved fecal incontinence (FI) symptoms, particularly in patients with a shorter duration of FI and higher baseline incontinence episode frequency.

The treatment was safe, with no unexpected adverse events reported, and the benefits were sustained or increased over 12 months in the high cell count group, suggesting potential for this therapy in managing FI.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Skeletal Muscle-Derived Cell Implantation for the Treatment of Fecal Incontinence: A Randomized, Placebo-Controlled Study. Frudinger, A., Gauruder-Burmester, A., Graf, W., et al.[2023]

In a study involving 42 patients with urinary stress incontinence, transurethral ultrasound-guided injections of autologous myoblasts and fibroblasts led to complete cure in 35 patients, demonstrating the efficacy of this new treatment approach.

No postoperative side effects or complications were reported, indicating a high safety profile for this innovative therapy using autologous stem cells to treat urinary incontinence.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Stem cell therapy for urinary incontinence]. Strasser, H., Marksteiner, R., Margreiter, E., et al.[2018]

In a study involving 80 women with stress urinary incontinence, autologous muscle-derived cells for urinary sphincter repair showed no adverse events, indicating a strong safety profile over 12 months.

The treatment demonstrated potential efficacy, with higher doses leading to greater improvements in urinary leakage and quality of life scores, suggesting a dose-response relationship.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous muscle derived cells for treatment of stress urinary incontinence in women. Peters, KM., Dmochowski, RR., Carr, LK., et al.[2017]

In a clinical trial involving 39 patients (average age 60.6 years), ultrasound-guided injection of autologous skeletal muscle-derived cells (SMDC) into the external anal sphincter significantly reduced weekly incontinence episodes by at least 50% in 80.6% of males and 78.4% of females after 12 months.

The procedure was well tolerated with no serious adverse events reported, suggesting it is a safe and effective treatment for fecal incontinence due to external anal sphincter damage or atrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Skeletal muscle-derived cell implantation for the treatment of sphincter-related faecal incontinence. Frudinger, A., Marksteiner, R., Pfeifer, J., et al.[2019]

In a phase I clinical trial involving 10 patients with intrinsic sphincter deficiency (ISD), periurethral implantation of myofibres containing muscle precursor cells (MPCs) significantly increased intraurethral pressure and improved continence, with four out of five women experiencing notable improvements over 12 months.

The procedure was found to be safe, with no serious side effects reported, indicating that myofibre implantation could be a promising one-step treatment for urinary incontinence due to ISD, although further research is needed to enhance its effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Periurethral skeletal myofibre implantation in patients with urinary incontinence and intrinsic sphincter deficiency: a phase I clinical trial. Yiou, R., Hogrel, JY., Loche, CM., et al.[2015]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05396456?term=AREA%5BConditionSearch%5D(Muscle%20fibers)&rank=4

Study Details | Autologous Muscle Fiber Fragment Injections ... for the treatment of Fecal Incontinence (FI) symptoms in men and women with a demonstrated anal sphincter defect and who have failed conservative treatments.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5805121/

Autologous Myoblasts for the Treatment of Fecal ... ... effectiveness of neuromodulation in the treatment of faecal incontinence ... Autologous skeletal-muscle-derived cell injection for anal incontinence due ...

3. withpower.com withpower.com/trial/phase-fecal-incontinence-8-2022-49388

Muscle Fiber Fragment Injections for Bowel Incontinence Research shows that injecting muscle-derived cells into the anal sphincter has improved bowel control in patients with fecal incontinence. Similar treatments ...

4. trialx.com trialx.com/clinical-trials/listings/280285/autologous-muscle-fiber-fragment-injections/?&geo_lat=36.1024&geo_lng=-80.2228&radius=10&place=Winston%20Salem&user_country=United%20States

Autologous Muscle Fiber Fragment Injections Clinical trial for Fecal Incontinence , Autologous Muscle Fiber Fragment Injections. ... To regenerate functional anal sphincter muscle using ...

5. beinvolved.atriumhealth.org beinvolved.atriumhealth.org/studies/5/irb00085606

Using Muscle Fiber Fragment Injections to Treat Fecal ... Using Muscle Fiber Fragment Injections to Treat Fecal Incontinence. Study Leader ... muscle at the bottom of the anus can help with leaking stool. Need ...