Autologous Cell Therapy for Treatment of Fecal Incontinence

A Prospective Nonrandomized Study of Autologous Muscle Derived Cell (AMDC) Transplantation for Treatment of Fecal Incontinence

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01600755

Enrollment

Registered

2012-05-17

Start date

2013-03-28

Completion date

2021-10-27

Last updated

2024-02-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fecal Incontinence

Keywords

Cell Therapy, Autologous Cell Transplantation, Muscle-derived Cell, Anal Incontinence, Rectal Diseases, Intestinal Diseases, Gastrointestinal Diseases, Urinary Incontinence

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.

Interventions

BIOLOGICALIltamiocel

Single intrasphincteric injection of iltamiocel at a dose of 250 x 10\^6 cells

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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 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.
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).
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.

Countries

Canada, United Kingdom

Participant flow

Participants by arm

Arm	Count
Iltamiocel AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.	48
Total	48

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Withdrawal by Subject	6

Baseline characteristics

Characteristic	Iltamiocel
28-Day Incontinence Diary (Days with Episodes)	12 Days with incontinence episodes
28-Day Incontinence Diary (Number of Episodes)	16 Incontinence Episodes/28 Days
Age, Categorical <=18 years	0 Participants
Age, Categorical >=65 years	24 Participants
Age, Categorical Between 18 and 65 years	24 Participants
Age, Continuous	61.7 years STANDARD_DEVIATION 13.3
Cleveland Clinic Incontinence Score (CCIS)	13.6 scores on scales STANDARD_DEVIATION 2.9
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	47 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Coping/behavior scale	1.7 scores on scales STANDARD_DEVIATION 0.6
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Depression/perception scale	2.5 scores on scales STANDARD_DEVIATION 0.9
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Embarrassment scale	1.9 scores on scales STANDARD_DEVIATION 0.8
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Lifestyle scale	2.5 scores on scales STANDARD_DEVIATION 0.9
Race (NIH/OMB) American Indian or Alaska Native	1 Participants
Race (NIH/OMB) Asian	2 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	1 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	44 Participants
Region of Enrollment Canada	33 participants
Region of Enrollment United Kingdom	15 participants
Sex: Female, Male Female	45 Participants
Sex: Female, Male Male	3 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 53
other Total, other adverse events	31 / 53
serious Total, serious adverse events	4 / 53

Outcome results

Primary

Number of Treatment-Related Adverse Events

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.

Time frame: 12 months

Arm	Measure	Group	Value (NUMBER)
Iltamiocel	Number of Treatment-Related Adverse Events	Study Product-related serious adverse events	0 events
Iltamiocel	Number of Treatment-Related Adverse Events	Study Product-related adverse events	1 events
Iltamiocel	Number of Treatment-Related Adverse Events	Injection procedure-related adverse events	5 events
Iltamiocel	Number of Treatment-Related Adverse Events	Biopsy procedure-related adverse events	28 events

Secondary

Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS))

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.