Trial Outcomes & Findings for Autologous Cell Therapy for Treatment of Fecal Incontinence (NCT NCT01600755)

NCT ID: NCT01600755

Last Updated: 2024-02-16

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 53 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2024-02-16

Participant Flow

Participant milestones

Measure	Iltamiocel AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Overall Study STARTED	53
Overall Study Iltamiocel Injection	48
Overall Study 3-Month Follow-Up	48
Overall Study 6-Month Follow-Up	48
Overall Study 12-Month Follow-Up	47
Overall Study COMPLETED	47
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Measure	Iltamiocel AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Overall Study Withdrawal by Subject	6

Baseline Characteristics

Autologous Cell Therapy for Treatment of Fecal Incontinence

Baseline characteristics by cohort

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=5 Participants
Age, Categorical >=65 years	24 Participants n=5 Participants
Age, Continuous	61.7 years STANDARD_DEVIATION 13.3 • n=5 Participants
Sex: Female, Male Female	45 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	47 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	44 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Canada	33 participants n=5 Participants
Region of Enrollment United Kingdom	15 participants n=5 Participants
28-Day Incontinence Diary (Number of Episodes)	16 Incontinence Episodes/28 Days n=5 Participants
28-Day Incontinence Diary (Days with Episodes)	12 Days with incontinence episodes n=5 Participants
Cleveland Clinic Incontinence Score (CCIS)	13.6 scores on scales STANDARD_DEVIATION 2.9 • n=5 Participants
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Lifestyle scale	2.5 scores on scales STANDARD_DEVIATION 0.9 • n=5 Participants
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Coping/behavior scale	1.7 scores on scales STANDARD_DEVIATION 0.6 • n=5 Participants
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Depression/perception scale	2.5 scores on scales STANDARD_DEVIATION 0.9 • n=5 Participants
Fecal Incontinence Quality of Life (FIQL) Questionnaire FIQL - Embarrassment scale	1.9 scores on scales STANDARD_DEVIATION 0.8 • n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Iltamiocel n=53 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Treatment-Related Adverse Events Study Product-related serious adverse events	0 events
Number of Treatment-Related Adverse Events Study Product-related adverse events	1 events
Number of Treatment-Related Adverse Events Injection procedure-related adverse events	5 events
Number of Treatment-Related Adverse Events Biopsy procedure-related adverse events	28 events

SECONDARY outcome

Timeframe: 3, 6, and 12 months post-treatment

Median number of fecal incontinence (FI) episodes as assessed by 28-day FI diary.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary) Fecal incontinence diary at 3 months	8 Incontinence Episodes/28 Days Interval 0.0 to 100.0
Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary) Fecal incontinence diary at 6 months	10 Incontinence Episodes/28 Days Interval 0.0 to 206.0
Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary) Fecal incontinence diary at 12 months	7 Incontinence Episodes/28 Days Interval 0.0 to 161.0

SECONDARY outcome

Timeframe: 3, 6, and 12 months post-treatment

Median number of days with fecal incontinence (FI) episodes, as assessed by 28-day FI diary.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary) FI diary at 3 months	7 days with incontinence episodes Interval 0.0 to 28.0
Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary) FI diary at 6 months	7 days with incontinence episodes Interval 0.0 to 28.0
Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary) FI diary at 12 months	5 days with incontinence episodes Interval 0.0 to 28.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 3 months	17 Participants
Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 6 months	19 Participants
Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 12 months	22 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 3 months	7 Participants
Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 6 months	12 Participants
Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 12 months	16 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 3 months	1 Participants
Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 6 months	4 Participants
Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes Fecal incontinence diary at 12 months	10 Participants

SECONDARY outcome

Timeframe: 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).

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Lifestyle scale at 12 months	0.6 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Lifestyle scale at 6 months	0.4 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Coping/Behavior scale at 3 months	0.4 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Depression/Self Perception scale at 3 months	0.3 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Depression/Self Perception scale at 6 months	0.3 scores on scales Standard Deviation 0.6
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Coping/Behavior scale at 6 months	0.5 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Coping/Behavior scale at 12 months	0.5 scores on scales Standard Deviation 0.7
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Depression/Self Perception scale at 12 months	0.4 scores on scales Standard Deviation 0.6
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Embarrassment scale at 3 months	0.5 scores on scales Standard Deviation 0.8
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Embarrassment scale at 6 months	0.5 scores on scales Standard Deviation 0.8
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Embarrassment scale at 12 months	0.6 scores on scales Standard Deviation 0.9
Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) Change in FIQL Lifestyle scale at 3 months	0.4 scores on scales Standard Deviation 0.7

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Iltamiocel n=48 Participants AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS)) Change in CCIS score at 3 months	-2.2 scores on scales Standard Deviation 3.0
Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS)) Change in CCIS score at 6 months	-2.0 scores on scales Standard Deviation 2.8
Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS)) Change in CCIS score at 12 months	-2.9 scores on scales Standard Deviation 2.8

Adverse Events

Iltamiocel

Serious events: 4 serious events

Other events: 31 other events

Deaths: 0 deaths

Serious adverse events

Measure	Iltamiocel n=53 participants at risk AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Infections and infestations Pneumonia	3.8% 2/53 • Number of events 3 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Urinary tract infection	1.9% 1/53 • Number of events 2 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Urosepsis	1.9% 1/53 • Number of events 2 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Renal and urinary disorders Nephrotic syndrome	1.9% 1/53 • Number of events 1 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Surgical and medical procedures Sinus operation	1.9% 1/53 • Number of events 1 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Gastrointestinal disorders Small intestine obstruction	1.9% 1/53 • Number of events 1 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment

Other adverse events

Measure	Iltamiocel n=53 participants at risk AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10\^6 cells.
Gastrointestinal disorders Diarrhea	15.1% 8/53 • Number of events 9 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Gastrointestinal disorders Hemorrhoids	5.7% 3/53 • Number of events 3 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Gastrointestinal disorders Proctalgia	5.7% 3/53 • Number of events 4 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Cystitis	5.7% 3/53 • Number of events 4 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Lower respiratory tract infection	11.3% 6/53 • Number of events 6 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Nasopharyngitis	7.5% 4/53 • Number of events 4 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Infections and infestations Urinary tract infection	9.4% 5/53 • Number of events 7 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Injury, poisoning and procedural complications Post procedural contusion	9.4% 5/53 • Number of events 5 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Injury, poisoning and procedural complications Procedural pain	26.4% 14/53 • Number of events 14 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Nervous system disorders Hypoesthesia	9.4% 5/53 • Number of events 5 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
Respiratory, thoracic and mediastinal disorders Cough	5.7% 3/53 • Number of events 3 • 12 months Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment

Additional Information

Ron Jankowski, PhD

Cook MyoSite Incorporated

Phone: 412-963-7380

Email: Ron.Jankowski@CookMyosite.com

Results disclosure agreements

• Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 30 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication unless confidential information is disclosed and cannot extend the embargo.
• Publication restrictions are in place

Restriction type: OTHER