Clinical trial NCT05396456: Autologous Muscle Fiber Fragment Injections
Official title: Autologous Muscle Fiber Fragment Injections for the Treatment of Fecal Incontinence
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Fecal Incontinence
Keywords: low anal pressures
passive incontinence
bowel movements
sphincter muscle
Lead sponsor: Wake Forest University Health Sciences
Sponsor class: OTHER
First posted: 2022-05-31
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Patients with refractory Fecal Incontinence (FI) will be identified from the Atrium Wake Forest Baptist Health outpatient Gastroenterology, Urogynecology and Colorectal Surgery clinics.
Primary purpose: TREATMENT

Brief summary: To regenerate functional anal sphincter muscle using muscle fiber fragments that contain muscle precursor cells (MPCs)

Detailed description: The Study hypothesizes that efficient re-assembly of injected fragments within host muscle tissue along the fiber direction of native muscle will be induced. It is anticipated that these fiber fragments would integrate into the host vascular and neural network.

Eligibility criteria: Inclusion Criteria:

* 18 years of age
* participants must experience 4 or more Fecal Incontinence (FI) episodes per 2 week period
* participants must score \>10 on Cleveland Clinic Fecal Incontinence Severity Scoring System (CCIS)
* participants must have had symptoms for at least 12 months
* participants who have failed standard medical and surgical treatments for FI
* participant will undergo anorectal manometry (ARM) testing
* participants will undergo endoscopic anal ultrasound and demonstrate an anatomic defect of the anal sphincter complex of at least 30 degrees
* women of childbearing potential must use acceptable contraceptives during this study

Exclusion Criteria:

* participants with symptomatic anorectal disease including symptomatic hemorrhoid disease, anal fissure or fistula causing symptoms such as bleeding, swelling, pain, or drainage
* participants with pre-existing ano-rectal pain of any cause
* participants with incontinence of flatus only
* chronic watery diarrhea which is the primary cause for fecal incontinence
* acute or chronic anorectal infections (including proctitis, recurrent abscesses, fistulae)
* presence of anorectal tumors
* active proctitis or inflammatory bowel disease
* previous injection of internal anal sphincter (IAS) with bulking agents
* participants requiring immunosuppression or who have any malignant disease within 3 years of enrollment
* participants with a defined bleeding disorder diagnosed and treated by a hematologist
* other exclusions include history of pelvic radiation, rectal prolapse, anorectal malformations, anorectal surgery within the previous 12 months, or treatments using injection or infrared coagulation for treatment of hemorrhoids
* participants with neurologic disease characterized by significant peripheral neuropathy or spinal cord dysfunction
* women who are pregnant, breastfeeding, or have had a child within the last year
* participants with a history of unstable cardiac function (New York Heart Association Functional Classification III or IV) or unstable pulmonary function requiring home oxygen, or abnormal kidney function (Cr \>1.5 mg/dl or on dialysis) or uncontrolled diabetes (Hemoglobin A1C \> 8 mg/dl)
* participants with anemia (hemoglobin \<10g, increased alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \>2 times normal) or increased bilirubin \>1.5mg/dl
* participants with Hepatitis B or C, or human immunodeficiency virus (HIV)-1 or 2
* rectal prolapse
* vaginal prolapse beyond the hymen
* unable to understand informed consent information even with provision of a medical translator

Primary outcomes:
measure: Cleveland Clinic Fecal Incontinence Severity Scoring System (CCIS) scores (also termed the Wexner score)
timeFrame: Month 3
measure: Cleveland Clinic Fecal Incontinence Severity Scoring System (CCIS) scores (also termed the Wexner score)
timeFrame: Month 6
measure: Cleveland Clinic Fecal Incontinence Severity Scoring System (CCIS) scores (also termed the Wexner score)
timeFrame: Month 12
Secondary outcomes:
measure: Change in Fecal Incontinence Quality of Life (FI-QOL) scores
timeFrame: baseline, Months 3 and 12
measure: Anorectal Manometry (ARM) Pressure scores
timeFrame: baseline, Months 3 and 12
measure: Internal Anal sphincter (IAS) Pressure scores
timeFrame: baseline, Months 3 and 12
measure: Rectal Anal Inhibitory Reflex (RAIR) Pressure scores
timeFrame: baseline, Months 3 and 12
measure: Fecal Incontinence Severity Index (FISI) scores
timeFrame: baseline, Months 3 and 12
Other outcomes:
measure: Anorectal endoscopic ultrasound (EUS)
timeFrame: Months 3 and 12
Locations:
city: Winston-Salem
country: United States
facility: Wake Forest University Health Sciences
state: North Carolina
Study officials:
affiliation: Wake Forest University Health Sciences
name: Catherine Matthews, MD
role: PRINCIPAL_INVESTIGATOR
References: