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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Iltamiocel | Experimental | AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Iltamiocel | Biological | Single intrasphincteric injection of iltamiocel at a dose of 250 x 10^6 cells |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary) | Median number of fecal incontinence (FI) episodes as assessed by 28-day FI diary. | 3, 6, and 12 months post-treatment |
| Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Manoj J Raval, MD | St. Paul's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St. Paul's Hospital | Vancouver | British Columbia | V6Z 1Y6 | Canada | ||
| Royal London Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37144409 | Result | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Iltamiocel | AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Iltamiocel | AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Number | events | 12 months |
|
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12 months
Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Iltamiocel | AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ron Jankowski, PhD | Cook MyoSite Incorporated | 412-963-7380 | Ron.Jankowski@CookMyosite.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: Canada | Apr 14, 2016 | Jul 27, 2022 | Prot_SAP_000.pdf |
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: United Kingdom | Jan 24, 2017 | Jul 27, 2022 | Prot_SAP_001.pdf |
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| ID | Term |
|---|---|
| D005242 | Fecal Incontinence |
| D004688 | Encopresis |
| D012002 | Rectal Diseases |
| D007410 | Intestinal Diseases |
| D005767 | Gastrointestinal Diseases |
| D014549 | Urinary Incontinence |
| ID | Term |
|---|---|
| D004066 | Digestive System Diseases |
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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Median number of days with fecal incontinence (FI) episodes, as assessed by 28-day FI diary. |
| 3, 6, and 12 months post-treatment |
| Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with categorical ≥50% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | 3, 6, and 12 months post-treatment |
| Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with categorical ≥75% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | 3, 6, and 12 months post-treatment |
| Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with 100% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | 3, 6, and 12 months post-treatment |
| Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) | 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). | 3, 6, and 12 months post-treatment |
| 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. | 3, 6, and 12 months post-treatment |
| London |
| United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| 28-Day Incontinence Diary (Number of Episodes) | Median | Full Range | Incontinence Episodes/28 Days |
|
| 28-Day Incontinence Diary (Days with Episodes) | Median | Full Range | Days with incontinence episodes |
|
| Cleveland Clinic Incontinence Score (CCIS) | 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. | Mean | Standard Deviation | scores on scales |
|
| Fecal Incontinence Quality of Life (FIQL) Questionnaire | FIQL is a validated, 29-item tool assessing 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). | Mean | Standard Deviation | scores on scales |
|
|
| Secondary | Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary) | Median number of fecal incontinence (FI) episodes as assessed by 28-day FI diary. | Posted | Median | Full Range | Incontinence Episodes/28 Days | 3, 6, and 12 months post-treatment |
|
|
|
| Secondary | Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary) | Median number of days with fecal incontinence (FI) episodes, as assessed by 28-day FI diary. | Posted | Median | Full Range | days with incontinence episodes | 3, 6, and 12 months post-treatment |
|
|
|
| Secondary | Number of Participants With Categorical ≥50% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with categorical ≥50% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | Posted | Count of Participants | Participants | 3, 6, and 12 months post-treatment |
|
|
|
| Secondary | Number of Participants With Categorical ≥75% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with categorical ≥75% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | Posted | Count of Participants | Participants | 3, 6, and 12 months post-treatment |
|
|
|
| Secondary | Number of Participants With 100% Reduction in 28-day Fecal Incontinence Episodes | Number of participants with 100% reduction in fecal incontinence (FI) episodes as assessed by 28-day FI diary. | Posted | Count of Participants | Participants | 3, 6, and 12 months post-treatment |
|
|
|
| Secondary | Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire) | 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). | Posted | Mean | Standard Deviation | scores on scales | 3, 6, and 12 months post-treatment |
|
|
|
| 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. | Posted | Mean | Standard Deviation | scores on scales | 3, 6, and 12 months post-treatment |
|
|
|
| 0 |
| 53 |
| 4 |
| 53 |
| 31 |
| 53 |
| Urinary tract infection | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
|
| Urosepsis | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
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| Nephrotic syndrome | Renal and urinary disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Sinus operation | Surgical and medical procedures | MedDRA Version 18.1 | Systematic Assessment |
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| Small intestine obstruction | Gastrointestinal disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Hemorrhoids | Gastrointestinal disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Proctalgia | Gastrointestinal disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Cystitis | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA Version 18.1 | Systematic Assessment |
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| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA Version 18.1 | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA Version 18.1 | Systematic Assessment |
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| Hypoesthesia | Nervous system disorders | MedDRA Version 18.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA Version 18.1 | Systematic Assessment |
|
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.
| D001526 | Behavioral Symptoms |
| D001519 | Behavior |
| D019960 | Elimination Disorders |
| D001523 | Mental Disorders |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D059411 | Lower Urinary Tract Symptoms |
| D020924 | Urological Manifestations |
| Title | Measurements |
|---|---|
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| Title | Measurements |
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| Title | Measurements |
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| Title | Measurements |
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| Title | Measurements |
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| Change in FIQL Coping/Behavior scale at 3 months |
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| Change in FIQL Coping/Behavior scale at 6 months |
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| Change in FIQL Coping/Behavior scale at 12 months |
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| Change in FIQL Depression/Self Perception scale at 3 months |
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| Change in FIQL Depression/Self Perception scale at 6 months |
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| Change in FIQL Depression/Self Perception scale at 12 months |
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| Change in FIQL Embarrassment scale at 3 months |
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| Change in FIQL Embarrassment scale at 6 months |
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| Change in FIQL Embarrassment scale at 12 months |
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