A Study of Darvadstrocel in Adults With Crohn's Disease and Complex Perianal Fistula
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|ClinicalTrials.gov Identifier: NCT04118088|
Recruitment Status : Recruiting
First Posted : October 8, 2019
Last Update Posted : October 27, 2021
|Condition or disease||Intervention/treatment||Phase|
|Crohn's Disease Complex Perianal Fistula||Biological: Darvadstrocel||Phase 4|
The biological being tested in this study is called darvadstrocel (Alofisel). This study is examining the long-term safety and efficacy of a repeat dose of darvadstrocel in people who have Crohn's disease and complex perianal fistula.
The study will enroll approximately 50 patients. Participants will be assigned to one treatment group to receive:
• Darvadstrocel 120 million cells
All participants would be allowed to receive one repeat dose of darvadstrocel in the whole study. This multi-center trial will be conducted worldwide. The overall time to participate in this study is approximately 3 years. Participants will make multiple visits to the clinic.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Postauthorization Safety Study of the Long-Term Safety and Efficacy of Repeat Administration of Darvadstrocel in Patients With Crohn's Disease and Complex Perianal Fistula|
|Actual Study Start Date :||December 22, 2020|
|Estimated Primary Completion Date :||March 31, 2026|
|Estimated Study Completion Date :||March 31, 2026|
Participants who have previously received darvadstrocel would receive a single repeat dose of darvadstrocel 120 million cells (5 million cells/mL), by local injection into the fistula.
Darvadstrocel suspension of human expanded adipose stem cells.
- Percentage of Participants with at Least 1 Treatment-Emergent Adverse Event (TEAE) [ Time Frame: From administration of repeat dose up to 156 weeks post-repeat administration ]An 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.
- Percentage of Participants with at Least 1 Treatment Emergent Serious Adverse Event (TESAE) [ Time Frame: From administration of repeat dose up to 156 weeks post-repeat administration ]An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
- Percentage of Participants with Special Situation Reports (SSRs) [ Time Frame: From administration of repeat dose up to 156 weeks post-repeat administration ]An SSR includes pregnancy, any case in which a pregnant participant is exposed to a study product or in which a female participant or female partner of a male participant becomes pregnant following treatment with a study product. Exposure is considered either through maternal exposure or via semen following paternal exposure or infant exposure from breast milk.
- Percentage of Participants with Adverse Event of Special Interest (AESI) [ Time Frame: From administration of repeat dose up to 156 weeks post-repeat administration ]AESI includes immunogenicity/alloimmune reactions, hypersensitivity, transmission of infectious agents, tumorgenicity (applying to malignant tumors only), ectopic tissue formation, medication errors.
- Percentage of Participants who Achieve Combined Remission of Perianal Fistula(s) [ Time Frame: At Week 24 and at Week 156 post-repeat darvadstrocel administration ]Combined remission is defined as the closure of all treated external openings that were draining at baseline, despite gentle finger compression and absence of collection(s) >2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by central magnetic resonance imaging (MRI) assessment.
- Percentage of Participants who Achieve Clinical Remission [ Time Frame: At Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration ]Clinical remission is defined as closure of all treated external fistula openings that were draining at baseline despite gentle finger compression.
- Percentage of Participants who Achieve Clinical Response [ Time Frame: At Weeks 6, 24, 52, 104, and 156 post-repeat darvadstrocel administration ]Clinical response is defined as closure of at least 50% of all treated external fistula openings that were draining at baseline despite gentle finger compression.
- Percentage of Participants with Relapse From Week 24 Combined Remission [ Time Frame: From Week 24 to Week 156 post-repeat darvadstrocel administration ]Relapse is 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.
- Time to Relapse [ Time Frame: From Week 24 to the Day of relapse post-repeat darvadstrocel administration ]Time to Relapse is 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.
- Percentage of Participants with New Perianal Abscess in Treated Fistula [ Time Frame: Up to Week 156 post-repeat darvadstrocel administration ]
- Change From Baseline in Score of Discharge and Pain Items of Perianal Disease Activity Index (PDAI) Score [ Time Frame: 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) and total range of score is from 0 to 20. Higher score means more severe disease.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04118088
|Contact: Takeda Contact||+1-877-825-3327||medinfoUS@takeda.com|
|Study Director:||Study Director||Takeda|