Autologous Cell Therapy for Treatment of Fecal Incontinence

Phase 1

Completed

Conditions: Fecal Incontinence

Interventions: Biological: Iltamiocel

Registration Number: NCT01600755

Lead Sponsor: Cook MyoSite

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 53

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Iltamiocel	Iltamiocel	AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.

Primary Outcome Measures

Name	Time	Method
Number of Treatment-Related Adverse Events	12 months	Assessment of frequency and severity of adverse events related to study product and study procedures through 12 months following treatment of FI in adult male and female subjects.

Secondary Outcome Measures

Name	Time	Method
Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary)	3, 6, and 12 months post-treatment	Median number of fecal incontinence (FI) episodes as assessed by 28-day FI diary.
Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes	3, 6, and 12 months post-treatment	Number of participants with categorical ≥50% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary.
Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes	3, 6, and 12 months post-treatment	Number of participants with categorical ≥75% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary.
Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes	3, 6, and 12 months post-treatment	Number of participants with 100% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary.
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire)	3, 6, and 12 months post-treatment	Mean change from baseline in patient-reported quality of life, as assessed by the Fecal Incontinence Quality of Life (FIQL) questionnaire. The FIQL is a validated, 29-item tool assessing the quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items, scored 0 to 4, with higher scores indicating a better QOL), Coping/Behavior (9 items, scored 0 to 4, with higher scores indicating a better QOL), Depression/Self-Perception (7 items, scored 0 to 4, with higher scores indicating a better QOL), and Embarrassment (3 items, scored 0 to 4, with higher scores indicating a better QOL).
Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary)	3, 6, and 12 months post-treatment	Median number of days with fecal incontinence (FI) episodes, as assessed by 28-day FI diary.
Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS))	3, 6, and 12 months post-treatment	Mean change from baseline in patient-reported fecal incontinence symptom severity, as assessed by the Cleveland Clinic Incontinence Score (CCIS) questionnaire. CCIS is a validated 5-questions tool assessing the frequency and the type of incontinence episodes (solid, liquid, gas), pad use, and lifestyle alteration. Scored from 0 to 20, with lower scores indicating less severe symptoms.