Muscle Fiber Fragment Treatment for Urinary Incontinence

Overseen By Gopal Badlani, MD

Age: 18+

Sex: Female

Trial Phase: Phase 1 & 2

Sponsor: Wake Forest University

Must not be taking: Urination affecting drugs

Disqualifiers: Hypercontractile bladder, HIV, diabetes, others

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

Compensation: Varies

Number of Visits: 6

Duration: 1 day

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for women with urinary incontinence, specifically targeting those with bladder outlet issues, such as stress urinary incontinence. The treatment, called Autologous Muscle Fiber Fragments, uses small pieces of the participant's own muscle, injected into the area that controls urination, to assess safety and potential effectiveness. Women diagnosed with urinary incontinence due to weak sphincters and a bladder capacity over 100 ml may be suitable for this study. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

Yes, if you are taking any medications that affect urination, including prescription drugs, over-the-counter drugs, or dietary supplements, you will need to stop taking them to participate in this trial.

Is there any evidence suggesting that autologous muscle fiber fragments are likely to be safe for humans?

Research has shown that using muscle fibers from the body is generally safe for treating urinary incontinence. One study found that injecting these muscle cells into the bladder area was safe and well-tolerated by adult women with stress urinary incontinence. Another study, which used varying amounts of muscle cells, reported no major safety issues. These findings suggest that using one's own muscle cells could be a safe method to address urinary problems. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike standard treatments for urinary incontinence, which often involve medications, pelvic floor exercises, or surgical interventions, the autologous muscle fiber fragment treatment is unique because it uses a patient's own muscle fibers. Researchers are excited about this approach as it involves directly injecting these muscle fragments into the bladder neck sphincter region, potentially providing a more natural and long-lasting solution by strengthening weakened muscles. This method could offer a less invasive alternative to surgery and might reduce the risk of side effects associated with other treatments, leading to improved quality of life for patients.

What evidence suggests that autologous muscle fiber fragments might be an effective treatment for urinary incontinence?

Research shows that using a person's own muscle cells might help treat urinary incontinence. In this trial, participants will receive Autologous Muscle Fiber Fragments, injected into the bladder neck sphincter region. Studies have found that these injections can improve symptoms in adults. Because these muscle cells come from the patient's own body, rejection is less likely. Trials found this treatment effective and safe for reducing symptoms of stress urinary incontinence, with benefits lasting at least 48 months. Overall, early results are promising for those dealing with urinary incontinence. ^{1 2 3 6 7}

Who Is on the Research Team?

Gopal Badlani, MD

Principal Investigator

Wake Forest School of Medicine, Dept. of Urology

Are You a Good Fit for This Trial?

This trial is for adult women aged 18-75 with urinary incontinence due to bladder neck or urethral issues. They must not be pregnant, breastfeeding, and should use birth control if sexually active. Participants need a bladder capacity over 100 ml and normal kidney function. Excluded are those with neurological disorders, muscle diseases, coagulation problems, significant pelvic organ prolapse, recent cellular therapy or investigational drug use.

Inclusion Criteria

My bladder can hold more than 100 ml of urine.

My kidney function is normal.

I am not pregnant, breastfeeding, and follow strict birth control or am not sexually active.

Exclusion Criteria

My bladder can hold less than 100 cc of urine.

I have a diagnosed bladder or kidney condition.

I am taking medication that affects how often I urinate.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo a biopsy and receive a single injection of autologous muscle fiber fragments into the bladder neck sphincter region

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

5 visits (in-person) at 1 week, 6 weeks, 3 months, 6 months, and 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Autologous Muscle Fiber Fragments

Trial Overview

The study tests the safety of autologous muscle fiber fragments as a treatment for urinary incontinence caused by sphincter insufficiency. It's aimed at helping those who have this condition due to either acquired (like stress incontinence) or congenital reasons.

How Is the Trial Designed?

Group I: Autologous Muscle Fiber Fragments Experimental Treatment 1 Intervention

Autologous Muscle Fiber Fragments administered via a single,direct injection into the bladder neck sphincter region

Who Is Running the Clinical Trial?

Wake Forest University

Lead Sponsor

Trials

193

Recruited

151,000+

Published Research Related to This Trial

The study involving 38 women showed that injecting autologous muscle-derived cells for stress urinary incontinence was safe, with only minor side effects like pain or bruising at the injection sites.

Patients receiving higher doses of these cells experienced significantly better outcomes, with a greater percentage achieving a 50% or more reduction in incontinence symptoms compared to those receiving lower doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous muscle derived cell therapy for stress urinary incontinence: a prospective, dose ranging study. Carr, LK., Robert, M., Kultgen, PL., et al.[2022]

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]

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]

Injecting autologous muscle-derived cells into the bladder and urethra of female rats is feasible and shows significant persistence of these cells over time, with a notable presence at 30 days post-injection.

Compared to bovine collagen, which showed minimal persistence after 30 days, the muscle-derived cells maintained approximately 88% of their initial expression, suggesting they may be a more effective treatment option for stress urinary incontinence.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Persistence and survival of autologous muscle derived cells versus bovine collagen as potential treatment of stress urinary incontinence. Yokoyama, T., Yoshimura, N., Dhir, R., et al.[2007]

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 study involving 8 children with bladder exstrophy, autologous myoblast injections significantly improved urinary continence, with 88% of patients achieving daytime dryness after an average follow-up of 15.3 months.

Urodynamic studies showed marked improvements in bladder capacity and function, indicating that this treatment could be a promising option for managing urinary incontinence in children, especially when combined with pelvic floor exercises.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transurethral autologous myoblast injection for treatment of urinary incontinence in children with classic bladder exstrophy. Kajbafzadeh, AM., Elmi, A., Payabvash, S., et al.[2008]

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]

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]

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