A Survey of Darvadstrocel in People With Crohn's Disease

Study Overview
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Brief Summary
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This study is a survey in Japan of Darvadstrocel injection used to treat Crohn's disease people with complicated anal fistula. The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from Darvadstrocel injection and to check if Darvadstrocel injection improves symptoms of Crohn's disease. During the study, participants with Crohn's disease will take Darvadstrocel injection according to their clinic's standard practice. The study doctors will check for side effects from Darvadstrocel for 36 months.

Official Title
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Postmarketing Surveillance Registry to Evaluate Safety and Efficacy of Darvadstrocel for the Treatment of Complex Perianal Fistulas in Crohn's Disease Patients (J-INSPIRE) (All-case Surveillance)

Conditions
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Crohn's Disease

Intervention / Treatment
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* Biological: Darvadstrocel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria All patients with complex perianal fistulas in Crohn's Disease who received Darvadstrocel will be included. Exclusion Criteria None

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Darvadstrocel<br>Darvadstrocel, 24 mL suspension of 120 million cells as a perilesional injection, once. Participants received interventions as part of routine medical care. | Biological: Darvadstrocel<br>* Darvadstrocel injection<br>* Other names: Cx601;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events | An adverse event is any untoward medical occurrence in a participant or clinical investigation subject administered a pharmaceutical product. It does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a pharmaceutical product (including an investigational product for a new indication in Japan), whether or not related to the pharmaceutical product. | 36 Months | 
| Percentage of Participants who Achieve Clinical Response | Clinical Response is defined as the clinically confirmed closure of at least 50% of the fistulas with this product as compared to baseline that were not drained with gentle finger compression. | Baseline, Up to Months 36 | 
| Percentage of Participants who Achieve Clinical Remission | Clinical remission is defined as the clinically confirmed closure of all fistulas treated with this product as compared to baseline with no discharge despite gentle finger compression. | Baseline, Up to Months 36 | 
| Percentage of Participants who Achieve Combined Remission | Combined remission is defined as the clinically confirmed closure of all baseline external openings that were not drained by gentle finger compression with this product treatment, and absence of an abscess greater than or equal to 2 cm in size confirmed by pelvic MRI (if performed) performed within 6 months of this product treatment. | Baseline, Up to Months 36 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants who Occurred Recurrence of Previously Treated Anal Fistulas in Participants who Achieved Clinical Remission at the Previous Visit | Recurrence of previously treated anal fistulas in patients who achieved clinical remission at the previous visit is defined as clinically confirmed recurrence with expulsion in any of the external openings treated with this product or the presence of a 2-axis or larger abscess > 2 cm in a previously treated fistula confirmed by MRI scan (if performed). | Baseline, Up to Months 36 | 
| Percentage of Participants with New Perianal Abscess in a Previously Treated Anal Fistula | New perianal abscess in a previously treated anal fistula is defined as fluid collection with a margin enhancing effect such that a high signal on T2-weighted image and a high signal edge on Gadolinium-enhanced T1-weighted image of the pelvis at the site of administration of this product, if performed. A new perianal abscess will be reported as an adverse event and the investigator will determine the causality with this product treatment. | Baseline, Up to Months 36 | 
| Change from Baseline in Perianal Disease Activity Index (PDAI) | The PDAI is a scoring system to evaluate the severity of perianal lesion associated with Crohn's disease. It includes the following 5 items: (a) Discharge; (b) Pain; (c) Restriction of sexual activity; (d) Type of perianal disease; and (e) Degree of induration. The discharge item is graded on a 5-point scale where 0 indicates no symptoms and 4 indicates severe symptoms and total range of score is from 0 to 4. Higher score means more severe disease. Negative change from Baseline indicates improvement. | Baseline, Up to Months 36 | 
| Change from Baseline in Harvey Bradshaw Index (HBI: Simple CDAI Score) | The HBI is a physician-reported index consisting of five clinical manifestations of CD. HBI items include performance status, abdominal pain, number of watery stools per day, abdominal mass, and complications. The HBI is a cumulative scoring system with the following threshold for lesion activity: Remission (< 5), mild (5   7), moderate (8   16), severe (> 16). Higher score means more severe disease. Negative change from Baseline indicates improvement. | Baseline, Up to Months 36 | 
| Health-Related Quality of Life (HRQOL) Measured by EuroQoL Five Dimensions Five Levels (EQ-5D-5L) Scale | EQ-5D-5L is a self-administered preference-based measure of health status suitable for calculating quality-adjusted life-years (QALYs) to inform economic evaluations. EQ-5D-5L includes 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and 5 response levels for each domain (no problems, slight problems, moderate problems, severe problems, and extreme problems). Scores ranges from best (100) to worst (0). | Baseline, Up to Months 36 | 
| HRQOL Measured by Crohn's Anal Fistula Quality of Life (CAF-QoL) Scale | CAF-QoL scale is a measure to check QOL related to Crohn's anal fistula. CAF-QoL scale includes 28-item questionnaires in 3 categories (symptoms of anal fistula, influence of treatment for anal fistula, and quality of life), and 5 response levels for each domain and each score (Not at all: 0, Rarely applicable: 1, Neither: 2, Almost Applicable: 3, Very Much Applicable: 4) for 27-item questionnaires and text format for remaining 1 questionnaire. Scores ranges from best (0) to worst (104). | Baseline, Up to Months 36 |