Crohn's Disease
Conditions
Brief summary
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.
Interventions
BIOLOGICALDarvadstrocel
Darvadstrocel injection
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
All patients with complex perianal fistulas in Crohn's Disease who received Darvadstrocel will be included.
Exclusion criteria
None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events | 36 Months | 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. |
| Percentage of Participants who Achieve Clinical Response | Baseline, Up to Months 36 | 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. |
| Percentage of Participants who Achieve Clinical Remission | Baseline, Up to Months 36 | 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. |
| Percentage of Participants who Achieve Combined Remission | Baseline, Up to Months 36 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Health-Related Quality of Life (HRQOL) Measured by EuroQoL Five Dimensions Five Levels (EQ-5D-5L) Scale | Baseline, Up to Months 36 | 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). |
| Change from Baseline in Harvey Bradshaw Index (HBI: Simple CDAI Score) | Baseline, Up to Months 36 | 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. |
| HRQOL Measured by Crohn's Anal Fistula Quality of Life (CAF-QoL) Scale | Baseline, Up to Months 36 | 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). |
| Percentage of Participants who Occurred Recurrence of Previously Treated Anal Fistulas in Participants who Achieved Clinical Remission at the Previous Visit | Baseline, Up to Months 36 | 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). |
| Percentage of Participants with New Perianal Abscess in a Previously Treated Anal Fistula | Baseline, Up to Months 36 | 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. |
| Change from Baseline in Perianal Disease Activity Index (PDAI) | Baseline, Up to Months 36 | 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. |
Countries
Japan
Contacts
Primary ContactTakeda Contact
Outcome results
None listed