Trial Outcomes & Findings for Phase 3 Study of Cx601 in Participants With Complex Perianal Fistulising Crohn's Disease (NCT NCT03706456)

NCT ID: NCT03706456

Last Updated: 2024-06-10

Results Overview

Combined remission of perianal fistulising CD is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression, and absence of collections \>2 cm in the treated fistulas, confirmed by central magnetic resonance imaging (MRI) assessment at Week 24 visit. In case of missing values, last observation carried forward (LOCF) method was applied. Percentages are rounded off to the nearest decimal point.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 22 participants
• Primary outcome timeframe: Week 24
• Results posted on: 2024-06-10

Participant Flow

Participants took part in the study at 9 investigative sites in Japan from 06 March 2019 to 10 February 2023.

Participants with a diagnosis of complex perianal fistulising Crohn's disease were enrolled to receive darvadstrocel once daily.

Participant milestones

Measure	Darvadstrocel 24 mL Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Overall Study STARTED	22
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Darvadstrocel 24 mL Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Overall Study Adverse Event	1
Overall Study Lack of Efficacy	2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of eASC as an intralesional injection, once on Day 1.
Age, Continuous	36.4 years STANDARD_DEVIATION 10.36 • n=22 Participants
Sex: Female, Male Female	8 Participants n=22 Participants
Sex: Female, Male Male	14 Participants n=22 Participants
Region of Enrollment Japan	22 Participants n=22 Participants
Height	166.0 centimeters (cm) STANDARD_DEVIATION 8.28 • n=22 Participants
Weight	68.33 kilograms (kg) STANDARD_DEVIATION 23.102 • n=22 Participants
Body Mass Index (BMI)	24.59 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 7.186 • n=22 Participants
Smoking Classification Participants has Never Smoked	16 Participants n=22 Participants
Smoking Classification Participants is a Current Smoker	2 Participants n=22 Participants
Smoking Classification Participants is an Ex-Smoker	4 Participants n=22 Participants
Duration of Crohn's Disease	11.3 years STANDARD_DEVIATION 6.64 • n=22 Participants
Concomitant Medications Biologics Only	9 Participants n=22 Participants
Concomitant Medications Immunosuppressants Only	2 Participants n=22 Participants
Concomitant Medications Biologics+Immunosuppressants	7 Participants n=22 Participants
Concomitant Medications None of 2 Types of Concomitant Drugs	4 Participants n=22 Participants

PRIMARY outcome

Timeframe: Week 24

Population: ITT Population included all participants enrolled in the study.

Combined remission of perianal fistulising CD is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression, and absence of collections >2 cm in the treated fistulas, confirmed by central magnetic resonance imaging (MRI) assessment at Week 24 visit. In case of missing values, last observation carried forward (LOCF) method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Combined Remission of Perianal Fistulising Crohn's Disease (CD) at Week 24	59.1 percentage of participants Interval 38.5 to 79.6

SECONDARY outcome

Timeframe: Week 24

Population: ITT Population included all participants enrolled in the study.

Clinical remission of perianal fistulising CD is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression at Week 24 Visit. In case of missing values, LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Clinical Remission of Perianal Fistulising CD at Week 24	59.1 percentage of participants Interval 38.5 to 79.6

SECONDARY outcome

Timeframe: Week 24

Population: ITT Population included all participants enrolled in the study.

Response of perianal fistulising CD is defined as the clinically confirmed closure of at least 50 percent of all treated external openings that were draining at Screening despite gentle finger compression at Week 24 visit. In case of missing values, LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Response of Perianal Fistulising CD at Week 24	81.8 percentage of participants Interval 65.7 to 97.9

SECONDARY outcome

Timeframe: Up to Week 24

Population: ITT Population included all participants enrolled in the study. Overall number of participants analyzed are the participants with clinical remission at least once during the 24-week period.

Time to clinical remission is defined as the time from the study product administration to the first visit by which clinical remission is observed. Clinical remission is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression.

Outcome measures

Measure	Darvadstrocel 24 mL n=16 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Clinical Remission of Perianal Fistulising CD by Week 24	30.0 days Interval 14.0 to 116.0

SECONDARY outcome

Timeframe: Up to Week 24

Population: ITT Population included all participants enrolled in the study. Overall number of participants analyzed are the participants with response at least once during the 24-week period.

Time to response is defined as the time from the study product administration to the first visit by which response is observed. Response is defined as the clinically confirmed closure of at least 50 percent of all treated external openings that were draining at Screening despite gentle finger compression.

Outcome measures

Measure	Darvadstrocel 24 mL n=19 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Response of Perianal Fistulising CD by Week 24	18.0 days Interval 12.0 to 30.0

SECONDARY outcome

Timeframe: Week 24

Population: ITT Population included all participants enrolled in the study. Overall number of participants analyzed are the participants who achieved clinical remission.

Relapse is defined as the clinically confirmed reopening of any of the treated external openings with active drainage, or the development of a collection >2 cm in the treated fistulas confirmed by central MRI assessment in participants who achieved clinical remission before Week 24. LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=16 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Relapse of Perianal Fistulising CD at Week 24 in Participants With Clinical Remission at a Previous Visit	25.0 percentage of participants Interval 3.8 to 46.2

SECONDARY outcome

Timeframe: Up to Week 24

Population: ITT Population included all participants enrolled in the study. Overall number of participants analyzed are the participants with clinical remission at least once during the 24-week period.

Time to Relapse is defined as the time from the first visit by which clinical remission is observed to the first visit by which relapse is observed. Relapse is defined as the clinically confirmed reopening of any of the treated external openings with active drainage, or the development of a collection >2 cm in the treated fistulas confirmed by central MRI assessment.

Outcome measures

Measure	Darvadstrocel 24 mL n=16 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Relapse of Perianal Fistulising CD by Week 24 in Participants With Clinical Remission at a Previous Visit	NA days Interval 85.0 to Median and upper limit of 95% confidence interval was not evaluated due to insufficient number of participants with relapse.

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: ITT Population included all participants enrolled in the study.

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. Each 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 20. Higher score indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in Perianal Disease Activity Index (PDAI): Total Score at Week 24 Baseline	4.8 score on a scale Standard Deviation 2.15
Change From Baseline in Perianal Disease Activity Index (PDAI): Total Score at Week 24 Change from Baseline at Week 24	-2.4 score on a scale Standard Deviation 2.22

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: ITT Population included all participants enrolled in the study.

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 indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in PDAI: Discharge Sub-Score at Week 24 Baseline	0.9 score on a scale Standard Deviation 0.64
Change From Baseline in PDAI: Discharge Sub-Score at Week 24 Change from Baseline at Week 24	-0.4 score on a scale Standard Deviation 0.79

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: ITT Population included all participants enrolled in the study.

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 pain 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 indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in PDAI: Pain Sub-Score at Week 24 Baseline	0.9 score on a scale Standard Deviation 0.92
Change From Baseline in PDAI: Pain Sub-Score at Week 24 Change from Baseline at Week 24	-0.6 score on a scale Standard Deviation 0.90

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: ITT Population included all participants enrolled in the study.

CDAI score is calculated from the following 8 items: (a) Number of liquid or very soft stools; (b) Abdominal pain; (c) General wellbeing; (d) Extraintestinal complications; (e) Antidiarrhoeal drugs; (f) Abdominal mass; (g) Hematocrit; and (h) Body weight. Scores of some items in CDAI are calculated based on patient diary. Total score ranges from 0 to 600. Higher score means more severe disease and especially severe disease was defined as a value of greater than 450. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in Crohn's Disease Activity Index (CDAI): Total Score at Week 24 Baseline	94.25 score on a scale Standard Deviation 60.048
Change From Baseline in Crohn's Disease Activity Index (CDAI): Total Score at Week 24 Change from Baseline at Week 24	-5.15 score on a scale Standard Deviation 47.500

SECONDARY outcome

Timeframe: Baseline and Week 24

Population: ITT Population included all participants enrolled in the study. Overall number of participants analyzed were participants with data available for analysis.

The Van Assche score represents the magnetic resonance imaging (MRI)- based severity of perianal lesion associated with Crohn's disease. Based on MRI data, the number, location and extension of fistula tracts, hyperintensity on T2-weighted images, presence or absence of collections (cavities >3 mm in diameter), and rectal wall involvement will be evaluated. The score range was from 0 to 22. Higher score means more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=18 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in Van Assche Score at Week 24 Baseline	14.5 score on a scale Standard Deviation 3.99
Change From Baseline in Van Assche Score at Week 24 Change from Baseline at Week 24	-1.7 score on a scale Standard Deviation 4.35

SECONDARY outcome

Timeframe: Week 52

Population: ITT Population included all participants enrolled in the study.

Combined remission is defined as the clinically confirmed closure of all treated external openings that were draining at the screening despite gentle finger compression, and absence of collections >2 cm in the treated fistulas which is confirmed by the central MRI assessment at Week 52 visit. In case of missing values, LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Combined Remission of Perianal Fistulising CD at Week 52	68.2 percentage of participants Interval 48.7 to 87.6

SECONDARY outcome

Timeframe: Week 52

Population: ITT Population included all participants enrolled in the study.

Clinical remission is defined as the clinically confirmed closure of all treated external openings that were draining at screening despite gentle finger compression at Week 52 visit. In case of missing values, LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Clinical Remission of Perianal Fistulising CD at Week 52	72.7 percentage of participants Interval 54.1 to 91.3

SECONDARY outcome

Timeframe: Week 52

Population: ITT Population included all participants enrolled in the study.

Response is defined as the clinically confirmed closure of at least 50 percent of all treated external openings that were draining at the screening despite gentle finger compression at Week 52 visit. In case of missing values, LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=22 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Response of Perianal Fistulising CD at Week 52	90.9 percentage of participants Interval 78.9 to 100.0

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants who achieved combined remission at least once during the 52-week period.

Time to combined remission is defined as the time from the study product administration to the first visit by which combined remission is observed. Combined remission is defined as the clinically confirmed closure of all treated external openings that were draining at the screening despite gentle finger compression, and absence of collections >2 cm in the treated fistulas which is confirmed by the central MRI assessment.

Outcome measures

Measure	Darvadstrocel 24 mL n=17 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Combined Remission of Perianal Fistulising CD by Week 52	175.5 days Interval 165.0 to 365.0

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants who achieved clinical remission at least once during the 52-week period.

Time to clinical remission is defined as the time from the study product administration to the first visit by which clinical remission is observed. Clinical remission is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression.

Outcome measures

Measure	Darvadstrocel 24 mL n=20 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Clinical Remission of Perianal Fistulising CD by Week 52	25.5 days Interval 14.0 to 109.0

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants who achieved response at least once during the 52-week period.

Time to response is defined as the time from the study product administration to the first visit by which response is observed. Response is defined as the clinically confirmed closure of at least 50 percent of all treated external openings that were draining at the Screening despite gentle finger compression.

Outcome measures

Measure	Darvadstrocel 24 mL n=21 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Response of Perianal Fistulising CD by Week 52	18.0 days Interval 12.0 to 30.0

SECONDARY outcome

Timeframe: Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with combined remission at Week 24.

Relapse is defined as the clinically confirmed reopening of any of the treated external openings with active drainage, or the development of a collection >2 cm in the treated fistulas confirmed by central MRI assessment in participants who achieved combined remission at Week 24. Combined remission is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression, and absence of collections >2 cm in the treated fistulas, confirmed by the central MRI assessment. LOCF method was applied. Percentages are rounded off to the nearest decimal point.

Outcome measures

Measure	Darvadstrocel 24 mL n=13 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Percentage of Participants With Relapse of Perianal Fistulising CD at Week 52 in Participants With Combined Remission at Week 24	23.1 percentage of participants Interval 0.2 to 46.0

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with combined remission at Week 24.

Time to relapse by Week 52 in participants who achieved combined remission at Week 24 is defined as the time from the combined remission at Week 24 to the first visit by which relapse is observed. Relapse is defined as the clinically confirmed reopening of any of the treated external openings with active drainage, or the development of a collection >2 cm in the treated fistulas confirmed by central MRI assessment in participants who achieved combined remission at Week 24. Combined remission is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression, and absence of collections >2 cm in the treated fistulas, confirmed by the central MRI assessment.

Outcome measures

Measure	Darvadstrocel 24 mL n=13 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Time to Relapse of Perianal Fistulising CD by Week 52 in Participants With Combined Remission at Week 24	NA days Median, lower and upper limits of confidence interval was not estimable due to low number of participants with relapse..

SECONDARY outcome

Timeframe: Baseline and Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with data available for analysis.

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. Each item is graded on a 5-point scale ranging from no symptoms (score of 0) to severe symptoms (score of 4) and total range of score is from 0 to 20. Higher score indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=20 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in PDAI: Total Score at Week 52	-2.8 score on a scale Standard Deviation 2.59

SECONDARY outcome

Timeframe: Baseline and Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with data available for analysis.

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 ranging from no symptoms (score of 0) to severe symptoms (score of 4) and total range of score is from 0 to 20. Higher score indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=20 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in PDAI: Discharge Sub-Score at Week 52	-0.5 score on a scale Standard Deviation 0.95

SECONDARY outcome

Timeframe: Baseline and Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with data available for analysis.

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 pain item is graded on a 5-point scale ranging from no symptoms (score of 0) to severe symptoms (score of 4) and total range of score is from 0 to 20. Higher score indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=20 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in PDAI: Pain Sub-Score at Week 52	-0.8 score on a scale Standard Deviation 1.06

SECONDARY outcome

Timeframe: Baseline and Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with data available for analysis.

CDAI score is calculated from the following 8 items: (a) Number of liquid or very soft stools; (b) Abdominal pain; (c) General wellbeing; (d) Extraintestinal complications; (e) Antidiarrhoeal drugs; (f) Abdominal mass; (g) Hematocrit; and (h) Body weight. Scores of some items in CDAI are calculated based on patient diary. Total score ranges from 0 to 600. Higher score indicates more severe disease and especially severe disease was defined as a value of greater than 450. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=21 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in CDAI: Total Score at Week 52	-20.86 score on a scale Standard Deviation 51.331

SECONDARY outcome

Timeframe: Baseline and Week 52

Population: ITT Population included all participants enrolled in the study. Overall number analyzed is the number of participants with data available for analysis.

The Van Assche score represents the MRI-based severity of perianal lesion associated with Crohn's disease. Based on MRI data, the number, location and extension of fistula tracts, hyperintensity on T2-weighted images, presence or absence of collections (cavities >3 mm in diameter), and rectal wall involvement will be evaluated. The total score ranges from 0 to 22. Higher score indicates more severe disease. Negative change from Baseline indicates improvement.

Outcome measures

Measure	Darvadstrocel 24 mL n=17 Participants Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Change From Baseline in Van Assche Total Score at Week 52	-2.2 score on a scale Standard Deviation 4.90

Adverse Events

Darvadstrocel 24 mL

Serious events: 11 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Measure	Darvadstrocel 24 mL n=22 participants at risk Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Gastrointestinal disorders Anal fistula	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Infections and infestations COVID-19	18.2% 4/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Renal and urinary disorders Calculus urinary	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Crohn's disease	13.6% 3/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Injury, poisoning and procedural complications Intestinal anastomosis complication	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Intestinal obstruction	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Pregnancy, puerperium and perinatal conditions Premature labour	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Small intestinal obstruction	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Renal and urinary disorders Tubulointerstitial nephritis	4.5% 1/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.

Other adverse events

Measure	Darvadstrocel 24 mL n=22 participants at risk Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acrochordon	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Anal fistula	27.3% 6/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Musculoskeletal and connective tissue disorders Back pain	13.6% 3/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Cheilitis	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Dental caries	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Musculoskeletal and connective tissue disorders Fistula	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Infections and infestations Nasopharyngitis	27.3% 6/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Nausea	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Infections and infestations Pharyngitis	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Gastrointestinal disorders Proctalgia	31.8% 7/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
General disorders Pyrexia	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
Infections and infestations Rhinitis	9.1% 2/22 • From first dose of study drug up to Week 156 At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: TrialDisclosures@takeda.com

Results disclosure agreements

• Principal investigator is a sponsor employee The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
• Publication restrictions are in place

Restriction type: OTHER