Muscle Fiber Fragment Injections for Bowel Incontinence

Recruiting in Palo Alto (17 mi)

Overseen by Catherine Matthews, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

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

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial aims to regenerate damaged anal sphincter muscles using small pieces of muscle that contain special cells. These pieces are injected into the damaged area to grow new muscle and restore function by connecting with the body's blood vessels and nerves.

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 data supports the effectiveness of the treatment Autologous 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 using muscle cells have also shown promise in improving urinary incontinence by increasing muscle function. ^{1 2 3 4 5}

Is Muscle Fiber Fragment Injection safe for humans?

Studies on similar treatments, like autologous muscle-derived cell injections for urinary and fecal incontinence, show no serious side effects, suggesting it is generally safe for humans. ^{1 2 3 6 7}

How is the treatment of Autologous Muscle Fiber Fragment Injections for bowel incontinence different from other treatments?

This treatment is unique because it involves injecting a patient's own muscle fibers into the anal sphincter to help repair and strengthen it, which is different from traditional treatments that may not use the body's own cells. This approach aims to improve muscle function and continence by promoting muscle regeneration directly at the site of damage. ^{1 2 4 5 8}

Research Team

Catherine Matthews, MD

Principal Investigator

Wake Forest University Health Sciences

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

• Autologous Muscle Fiber Fragment Injections (Muscle Regeneration)

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.

Participant Groups

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

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Wake Forest University Health Sciences Winston-Salem, NC

Who Is Running the Clinical Trial?

Wake Forest University Health Sciences

Lead Sponsor

Trials

1432

Recruited

2,506,000+

Dr. Julie Ann Freischlag

Wake Forest University Health Sciences

Chief Executive Officer since 2020

BS from University of Illinois, MD from Rush University

Dr. L. Ebony Boulware

Wake Forest University Health Sciences

Chief Medical Officer since 2022

MD from Duke University School of Medicine, MPH from Johns Hopkins Bloomberg School of Public Health

Findings from Research

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]

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]

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 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]

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]

References

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

Skeletal Muscle-Derived Cell Implantation for the Treatment of Fecal Incontinence: A Randomized, Placebo-Controlled Study. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Periurethral skeletal myofibre implantation in patients with urinary incontinence and intrinsic sphincter deficiency: a phase I clinical trial. [2015]

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

Autologous Myoblasts for the Treatment of Fecal Incontinence: Results of a Phase 2 Randomized Placebo-controlled Study (MIAS). [2019]

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

Allogenic myoblast transplantation in the rat anal sphincter. [2012]

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

Skeletal muscle-derived cell implantation for the treatment of sphincter-related faecal incontinence. [2019]

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

Autologous muscle derived cells for treatment of stress urinary incontinence in women. [2017]

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

Intraurethral injection of autologous minced skeletal muscle: a simple surgical treatment for stress urinary incontinence. [2014]

8. Germany pubmed.ncbi.nlm.nih.gov

[Stem cell therapy for urinary incontinence]. [2018]