Trial Outcomes & Findings for A Study of Darvadstrocel in Adults With Crohn's Disease and Complex Perianal Fistula (NCT NCT04118088)

Last Updated: 2026-01-22

Results Overview

An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an AE with an onset that occurs after receiving study drug.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

53 participants

Primary outcome timeframe

From signing of informed consent form (ICF) up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks

Results posted on

2026-01-22

Participant Flow

Participants took part in the study at various investigative sites globally from 22 December 2020 to 14 February 2025.

Participants with a diagnosis of Crohn's Disease (CD) and complex perianal fistula were enrolled in this study to receive a single repeated administration of darvadstrocel (Alofisel). The study was terminated early based on the sponsor's decision.

Participant milestones

Participant milestones
Measure	Darvadstrocel Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mililiters (mL) suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Overall Study STARTED	53
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	42

Reasons for withdrawal

Reasons for withdrawal
Measure	Darvadstrocel Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mililiters (mL) suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Overall Study Study Terminated by Sponsor	38
Overall Study Lost to Follow-up	2
Overall Study Withdrawal by Subject	1
Overall Study Reason Not Specified	1

Baseline Characteristics

A Study of Darvadstrocel in Adults With Crohn's Disease and Complex Perianal Fistula

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Age, Continuous	41.7 years STANDARD_DEVIATION 11.00 • n=270 Participants
Sex: Female, Male Female	30 Participants n=270 Participants
Sex: Female, Male Male	23 Participants n=270 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=270 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=270 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=270 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=270 Participants
Race (NIH/OMB) Asian	1 Participants n=270 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=270 Participants
Race (NIH/OMB) Black or African American	0 Participants n=270 Participants
Race (NIH/OMB) White	52 Participants n=270 Participants
Race (NIH/OMB) More than one race	0 Participants n=270 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=270 Participants

PRIMARY outcome

Timeframe: From signing of informed consent form (ICF) up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)	66 percentage of participants

PRIMARY outcome

Timeframe: From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

A serious adverse event (SAE) is defined as an untoward medical occurrence, significant hazard, contraindication, side effect or precaution that at any dose: results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant. A treatment-emergent SAE is an SAE which occurs after exposure to study treatment. Percentages were rounded off to the nearest single decimal place.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants With at Least One Treatment Emergent Serious Adverse Event (TESAE)	5.7 percentage of participants

PRIMARY outcome

Timeframe: From administration of repeat dose up to 156 weeks post-repeat administration

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

Female participants and/or female partners of male participants who become pregnant following treatment with the study product and reported the pregnancy on a paper pregnancy report form immediately or within 24 hours of awareness were reported.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Number of Reported Pregnancies During Study	1 pregnancies

PRIMARY outcome

Timeframe: From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

Adverse event of special interests (AESIs) include immunogenicity/alloimmune reactions, hypersensitivity reactions, ectopic tissue formation, medication errors, tumorigenicity, and transmission of infectious agents. A treatment-emergent AESI is an AESI which occurs after exposure to study treatment.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants With Treatment Emergent Adverse Event of Special Interest (TEAESI)	17 percentage of participants

SECONDARY outcome

Timeframe: At Weeks 24 and 156 post-repeat darvadstrocel administration

Population: As the sponsor decided to terminate the study before the MRI could be done, the data for this outcome measure which was planned to be collected through MRI was not collected.

Combined remission was defined as the closure of all treated external openings that were draining at baseline, despite gentle finger compression and absence of collection(s) \>2 centimeters (cm) (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by central magnetic resonance imaging (MRI) assessment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: At Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel. Number analyzed is the number of participants available for analysis at the specified time-point.

Clinical remission was defined as closure of all treated external fistula openings that were draining at baseline despite gentle finger compression.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants Who Achieved Clinical Remission Week 6	52.8 percentage of participants Interval 38.6 to 66.7
Percentage of Participants Who Achieved Clinical Remission Week 24	66.0 percentage of participants Interval 51.7 to 78.5
Percentage of Participants Who Achieved Clinical Remission Week 52	69.2 percentage of participants Interval 54.9 to 81.3
Percentage of Participants Who Achieved Clinical Remission Week 104	74.1 percentage of participants Interval 53.7 to 88.9
Percentage of Participants Who Achieved Clinical Remission Week 156	72.7 percentage of participants Interval 39.0 to 94.0

SECONDARY outcome

Timeframe: At Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration

Clinical response was defined as closure of at least 50% of all treated external fistula openings that were draining at baseline despite gentle finger compression.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants Who Achieved Clinical Response Week 6	61.5 percentage of participants Interval 47.0 to 74.7
Percentage of Participants Who Achieved Clinical Response Week 24	71.2 percentage of participants Interval 56.9 to 82.9
Percentage of Participants Who Achieved Clinical Response Week 52	73.1 percentage of participants Interval 59.0 to 84.4
Percentage of Participants Who Achieved Clinical Response Week 104	77.8 percentage of participants Interval 57.7 to 91.4
Percentage of Participants Who Achieved Clinical Response Week 156	72.7 percentage of participants Interval 39.0 to 94.0

SECONDARY outcome

Timeframe: From Week 24 to Week 156 post-repeat darvadstrocel administration

Population: As the sponsor decided to terminate the study before the MRI could be done, the data for this outcome measure which was planned to be collected through MRI was not collected.

Relapse was defined as reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed that were in the combined remission at Week 24 or the development of a collection \>2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by centrally read MRI assessment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Week 24 to the day of relapse post-repeat darvadstrocel administration

Population: SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel. Time to relapse is presented in the subset of participants who were in clinical remission at Week 24.

Time to Relapse was defined as the time in days to reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed, relative to Week 24.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=35 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Time to Relapse	932.0 days Interval 748.0 to The upper limit of 95% confidence interval (CI) was not estimable due to censoring.

SECONDARY outcome

Timeframe: At Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Percentage of Participants With New Perianal Abscess in Treated Fistula Week 6	3.8 percentage of participants
Percentage of Participants With New Perianal Abscess in Treated Fistula Week 24	5.7 percentage of participants
Percentage of Participants With New Perianal Abscess in Treated Fistula Week 52	0 percentage of participants
Percentage of Participants With New Perianal Abscess in Treated Fistula Week 104	0 percentage of participants

SECONDARY outcome

Timeframe: Baseline to Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration

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). Higher score means more severe disease.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=53 Participants Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Change at Week 24	-0.5 score on a scale Standard Deviation 0.93
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Change at Week 52	-0.6 score on a scale Standard Deviation 1.00
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Baseline	1.1 score on a scale Standard Deviation 1.43
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Baseline	1.1 score on a scale Standard Deviation 0.81
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Baseline	1.4 score on a scale Standard Deviation 0.95
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Change at Week 6	-0.3 score on a scale Standard Deviation 1.17
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Change at Week 24	-0.6 score on a scale Standard Deviation 1.16
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Change at Week 52	-0.6 score on a scale Standard Deviation 1.32
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Change at Week 104	-0.6 score on a scale Standard Deviation 1.39
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Discharge: Change at Week 156	-0.4 score on a scale Standard Deviation 1.36
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Baseline	1.1 score on a scale Standard Deviation 0.79
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Change at Week 6	-0.5 score on a scale Standard Deviation 0.95
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Change at Week104	-0.6 score on a scale Standard Deviation 0.80
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Pain Restriction of Activities: Change at Week156	-0.8 score on a scale Standard Deviation 0.87
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Change at Week 6	-0.4 score on a scale Standard Deviation 0.99
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Change at Week 24	-0.4 score on a scale Standard Deviation 1.10
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Change at Week 52	-0.5 score on a scale Standard Deviation 1.23
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Change at Week104	-0.4 score on a scale Standard Deviation 1.31
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Restriction of Sexual Activity: Change at Week156	-0.8 score on a scale Standard Deviation 1.25
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Baseline	2.0 score on a scale Standard Deviation 0.48
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Change at Week 6	-0.4 score on a scale Standard Deviation 0.88
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Change at Week 24	-0.8 score on a scale Standard Deviation 1.11
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Change at Week 52	-0.8 score on a scale Standard Deviation 1.16
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Change at Week 104	-0.8 score on a scale Standard Deviation 1.24
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Type of Perianal Disease: Change at Week 156	-0.6 score on a scale Standard Deviation 0.92
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Change at Week 6	-0.4 score on a scale Standard Deviation 1.03
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Change at Week 24	-0.5 score on a scale Standard Deviation 1.03
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Change at Week 52	-0.7 score on a scale Standard Deviation 0.90
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Change at Week 104	-0.6 score on a scale Standard Deviation 0.85
Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score Degree of Induration: Change at Week 156	-0.5 score on a scale Standard Deviation 0.82

Adverse Events

Darvadstrocel

Serious events: 3 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Darvadstrocel n=53 participants at risk Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Infections and infestations Anal abscess	1.9% 1/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Nervous system disorders Cerebrovascular accident	1.9% 1/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Gastrointestinal disorders Proctalgia	1.9% 1/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

Other adverse events

Other adverse events
Measure	Darvadstrocel n=53 participants at risk Participants who had previously received darvadstrocel were administered a single repeat dose of darvadstrocel, 24 mL suspension of 120 million cells (5 million cells/mL), as a perilesional injection into the fistula.
Infections and infestations COVID-19	13.2% 7/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Gastrointestinal disorders Diarrhoea	5.7% 3/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Musculoskeletal and connective tissue disorders Fistula discharge	5.7% 3/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Infections and infestations Influenza	5.7% 3/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.
Gastrointestinal disorders Proctalgia	15.1% 8/53 • From signing of ICF up to 156 weeks post-repeat administration (the only administration in this study), up to approximately 164 weeks SAF consisted of all participants who were enrolled in the study and received treatment with darvadstrocel.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place