Clinical trial NCT01600755: Autologous Cell Therapy for Treatment of Fecal Incontinence
Official title: A Prospective Nonrandomized Study of Autologous Muscle Derived Cell (AMDC) Transplantation for Treatment of Fecal Incontinence
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Fecal Incontinence
Keywords: Cell Therapy
Autologous Cell Transplantation
Muscle-derived Cell
Anal Incontinence
Rectal Diseases
Intestinal Diseases
Gastrointestinal Diseases
Urinary Incontinence
Lead sponsor: Cook MyoSite
Sponsor class: INDUSTRY
First posted: 2012-05-17
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The aim of this clinical study is to investigate the safety and feasibility of Autologous Muscle Derived Cells (AMDC, generic name iltamiocel, preparation of a patient's own cells) injection into the anal sphincter for treatment of patients with fecal incontinence.

Detailed description: This study is designed to test the safety and feasibility of iltamiocel (Autologous Muscle Derived Cells, AMDC) as a treatment for fecal incontinence in men and women. Iltamiocel therapy seeks to allow remodeling of the external anal sphincter in patients with fecal incontinence from either defined structural defects or a generalized weakening of the external anal sphincter.

Eligibility criteria: Inclusion Criteria:

* Primary symptoms of fecal incontinence, as confirmed by patient medical history and physical examination
* Wexner (Cleveland Clinic Incontinence Severity (CCIS)) score ≥ 9
* Etiology of fecal incontinence is related, at least in part, to external anal sphincter dysfunction
* Failed conservative treatment

Exclusion Criteria:

* Gracilis sling repair or insertion of an artificial sphincter
* Inflammatory Bowel Disease
* Significant rectocele or rectal prolapse
* History of radiation treatment to the anal sphincter or adjacent structures
* Less than 18 years of age
* Pregnant, breastfeeding, or plans to become pregnant during the course of the study
* Neuromuscular disorder
* History of neoplasia within 5 years prior to enrollment, except for basal cell carcinoma, or is receiving or planning to receive anti-cancer medications
* Known bleeding diathesis or uncorrected coagulopathy
* Medical condition that would preclude treatment due to contraindications and/or warnings of concomitant medications or listed in the experimental product labeling
* Compromised immune system due to disease state, chronic corticosteroid use, or other immunosuppressive therapy
* Participating in another investigational drug or device study
* Unable or unwilling to provide informed consent
* Unable or unwilling to commit to the follow-up procedures

Primary outcomes:
measure: Number of Treatment-Related Adverse Events
timeFrame: 12 months
Secondary outcomes:
measure: Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary)
timeFrame: 3, 6, and 12 months post-treatment
measure: Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary)
timeFrame: 3, 6, and 12 months post-treatment
measure: Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes
timeFrame: 3, 6, and 12 months post-treatment
measure: Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes
timeFrame: 3, 6, and 12 months post-treatment
measure: Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes
timeFrame: 3, 6, and 12 months post-treatment
measure: Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire)
timeFrame: 3, 6, and 12 months post-treatment
measure: Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS))
timeFrame: 3, 6, and 12 months post-treatment
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: St. Paul's Hospital
state: British Columbia
city: London
country: United Kingdom
facility: Royal London Hospital
Study officials:
affiliation: St. Paul's Hospital
name: Manoj J Raval, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Knowles CH, Canestrari E, Jankowski RJ, Cardello K, Raval MJ. Safety and Efficacy of Iltamiocel Cellular Therapy for the Treatment of Fecal Incontinence. Results of a Phase 1/2 Study. Ann Surg. 2023 Dec 1;278(6):937-944. doi: 10.1097/SLA.0000000000005894. Epub 2023 May 5.