Trial Outcomes & Findings for A Study of Darvadstrocel for Treating Complex Perianal Fistulas in Children and Teenagers With Crohn's Disease (NCT NCT04701411)

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

7 participants

Primary outcome timeframe

Week 24

Results posted on

2025-11-13

Participant Flow

Participants took part in the study at various investigative sites globally from 30 June 2021 to 07 May 2025.

Participants with complex perianal fistula due to Crohn's Disease (CD) participated in this study to receive darvadstrocel (Cx601). The study was terminated early based on the sponsor's decision and did not meet the planned enrollment number. All enrolled participants completed all the study assessments.

Participant milestones

Participant milestones
Measure	Darvadstrocel Participants were administered darvadstrocel (Cx601), 24 milliliters (mL) suspension of 120 million cells as a perilesional injection, once on Day 0.
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Darvadstrocel for Treating Complex Perianal Fistulas in Children and Teenagers With Crohn's Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Age, Continuous	15.7 years STANDARD_DEVIATION 1.38 • n=10 Participants
Sex: Female, Male Female	2 Participants n=10 Participants
Sex: Female, Male Male	5 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=10 Participants
Race (NIH/OMB) Asian	0 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=10 Participants
Race (NIH/OMB) Black or African American	0 Participants n=10 Participants
Race (NIH/OMB) White	7 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=10 Participants

PRIMARY outcome

Timeframe: Week 24

Population: Intent-to-treat (ITT) analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not.

Combined remission was defined as the closure of all treated external openings that were draining at baseline despite gentle finger compression, and absence of abscess(es) \>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 measures
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Percentage of Participants Who Achieved Combined Remission	42.9 percentage of participants Interval 9.9 to 81.6

SECONDARY outcome

Timeframe: Weeks 24 and 52

Population: ITT analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not.

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

Outcome measures

Outcome measures
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Percentage of Participants Who Achieved Clinical Remission Week 52	28.6 percentage of participants Interval 3.7 to 71.0
Percentage of Participants Who Achieved Clinical Remission Week 24	42.9 percentage of participants Interval 9.9 to 81.6

SECONDARY outcome

Timeframe: Weeks 24 and 52

Population: ITT analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not.

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

Outcome measures

Outcome measures
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Percentage of Participants Who Achieved Clinical Response Week 24	71.4 percentage of participants Interval 29.0 to 96.3
Percentage of Participants Who Achieved Clinical Response Week 52	28.6 percentage of participants Interval 3.7 to 71.0

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not.

Time to Clinical Remission was defined as the time in weeks from treatment start to first visit at which clinical remission was observed before Week 52; where clinical remission is said to have occurred if a clinical assessment showed closure of all treated external openings that were draining at baseline despite gentle finger compression. Participants without documented time to clinical remission by the end of study (Week 52), were censored at the date of last assessment along with the participants who discontinued without clinical remission before Week 52 at the date of last visit.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Time to Clinical Remission	NA weeks Interval 6.1 to Median and upper limit of 95% confidence interval (CI) were not estimable due to insufficient number of participants with events.

SECONDARY outcome

Timeframe: Up to Week 52

Population: ITT analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not.

Time to clinical response defined as the time in weeks from treatment start to first visit at which clinical response was observed before Week 52; where clinical response is said to have occurred if a clinical assessment showed closure of at least 50% of all treated external openings that were draining at baseline despite gentle finger compression. Participants without documented time to clinical response by the end of study (Week 52), were censored at the date of last assessment along with the participants who discontinued without clinical response before Week 52 at the date of last visit.

Outcome measures

Outcome measures
Measure	Darvadstrocel n=7 Participants Participants were administered darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.
Time to Clinical Response	6.6 weeks Interval 6.1 to The upper limit of 95% CI was not estimable due to censoring.

SECONDARY outcome

Timeframe: From Week 24 to Week 52

Population: ITT analysis set included all participants who underwent the fistula preparation procedure regardless of being treated or not. Overall number analyzed is the number of participants with combined remission at Week 24.

Relapse was defined as reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed in participants who were in combined remission at Week 24.