Long-term Follow-up with Darvadstrocel for Perianal Fistula

No longer recruiting at 114 trial locations

Overseen By Takeda Study Registration Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Millennium Pharmaceuticals, Inc.

Disqualifiers: Time since ADMIRE-CD II, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to assess the long-term effects and symptom improvement of Darvadstrocel, a cell therapy, in people with complex perianal fistulas. Participants will not receive new treatment during this trial; instead, researchers will observe outcomes from previous treatments. The trial seeks individuals who completed the ADMIRE-CD II study within the last three months. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to the validation of a promising treatment.

Will I have to stop taking my current medications?

The trial does not specify whether you need to stop taking your current medications, but since participants will not receive any drug in this study, it seems unlikely that you would need to stop.

Is there any evidence suggesting that Darvadstrocel is likely to be safe for humans?

Research has shown that darvadstrocel is generally safe for treating complex perianal fistulas. Studies indicate that people tolerate it well. For instance, one study found that symptom relief lasted up to 104 weeks after treatment. Another study reported no major safety issues for up to 156 weeks. Additionally, a long-term follow-up study found no new safety concerns three years after treatment. Overall, these findings suggest that darvadstrocel is a promising and safe treatment option. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for perianal fistulas, which often involve antibiotics or surgery, Darvadstrocel offers a novel approach by using stem cells. Researchers are excited about Darvadstrocel because it utilizes expanded adipose-derived stem cells (eASCs) to potentially promote healing from within. This method targets the underlying issue by harnessing the regenerative power of stem cells, which is different from simply addressing symptoms or removing damaged tissue. This innovative approach could lead to more effective and less invasive treatment options for patients.

What evidence suggests that Darvadstrocel is effective for perianal fistula?

Research shows that darvadstrocel may help treat complex perianal fistulas in people with Crohn's disease. In earlier studies, about half of the patients who received darvadstrocel experienced significant symptom improvement or resolution. Predictions based on trial data and real-world evidence suggest these benefits could last up to 104 weeks. This trial will observe participants who previously received darvadstrocel for long-term efficacy and safety. Overall, darvadstrocel appears to be generally well-tolerated and may provide lasting symptom relief. ^{2 6 7 8 9}

Who Is on the Research Team?

Study Director

Principal Investigator

Takeda

Are You a Good Fit for This Trial?

This trial is for adults who have completed the ADMIRE-CD II study without dropping out, aimed at those with complex perianal fistulas often associated with Crohn's Disease. Participants must join within 3 months of completing the previous study.

Inclusion Criteria

Has participated in and completed the ADMIRE-CD II (NCT03279081) study (i.e., did not discontinue).

Exclusion Criteria

Has been more than 3 months since the participant completed the ADMIRE-CD II study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Long-term Follow-up

Participants are monitored for long-term safety and efficacy of darvadstrocel without receiving any drug

104 weeks

Multiple visits to the clinic and telephone follow-up every 3 months

Open-label Extension

Participants continue in the treatment group assigned in the ADMIRE-CD II study for further observation

104 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Darvadstrocel

Trial Overview The trial is not testing a new treatment but is focused on long-term follow-up to monitor side effects and symptom improvement in patients who were treated with Darvadstrocel for complex perianal fistula.

How Is the Trial Designed?

Group I: Darvadstrocel Experimental Treatment 1 Intervention

Participants who received a single dose of darvadstrocel, 120 million cells, intralesionally previously in the ADMIRE-CD II study were observed for efficacy and safety. No drug was administered in this study.

Group II: Placebo Placebo Group 1 Intervention

Participants who received darvadstrocel placebo-matching expanded adipose-derived stem cells (eASCs) intralesional injection previously in the ADMIRE-CD II study were observed for efficacy and safety. No drug was administered in this study.

Who Is Running the Clinical Trial?

Millennium Pharmaceuticals, Inc.

Lead Sponsor

Trials

406

Recruited

46,900+

Dr. Christophe Bianchi

Millennium Pharmaceuticals, Inc.

Chief Medical Officer since 2006

MD from University of Geneva

Dr. Deborah Dunsire

Millennium Pharmaceuticals, Inc.

Chief Executive Officer since 2005

MD from University of Witwatersrand

Takeda

Lead Sponsor

Trials

1,255

Recruited

4,219,000+

Dr. Naoyoshi Hirota

Takeda

Chief Medical Officer since 2020

MD from University of Tokyo

Christophe Weber

Takeda

Chief Executive Officer since 2015

PhD in Molecular Biology from Université de Montpellier

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39736547/

Predictive modeling to evaluate long-term treatment ... Predictive modeling to evaluate long-term treatment effectiveness of darvadstrocel in patients with complex perianal fistulas in Crohn's disease.

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2210740124001943

Real-life effectiveness of allogeneic expanded adipose ... Real life data about the long-term efficacy of darvadstrocel for treating perianal fistulas in Crohn's disease (CD) remain scarce. Aim: To report the ...

3. bmcgastroenterol.biomedcentral.com bmcgastroenterol.biomedcentral.com/articles/10.1186/s12876-024-03513-3

Predictive modeling to evaluate long-term treatment ... The aim of this study was to combine clinical trial data and real-world evidence using statistical methodologies to predict long-term effectiveness of DVS ...

4. journals.lww.com journals.lww.com/dcrjournal/fulltext/2022/05000/follow_up_study_to_evaluate_the_long_term_safety.16.aspx

Follow-up Study to Evaluate the Long-term Safety and... Darvadstrocel was well tolerated and clinical remission after treatment with darvadstrocel may be sustained for up to 104 weeks in patients with perianal ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39349101/

Real-life effectiveness of allogeneic expanded adipose- ... Conclusion: In this real-world setting, a successful response to darvadstrocel therapy based on clinical remission was reported in around half of the patients ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10785025/

Effectiveness and safety of darvadstrocel in patients with ... Darvadstrocel appears to be a new, potentially effective and safe treatment option for the management of complex perianal fistulas.

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35099555/

INSPECT: A Retrospective Study to Evaluate Long-term ... This retrospective chart review of patients treated with darvadstrocel indicates sustained remission and confirms a favorable safety profile up to 156 weeks.

8. gastrojournal.org gastrojournal.org/article/S0016-50851736726-4/fulltext

Long-term Efficacy and Safety of Stem Cell Therapy ... Therapies for perianal fistulas in patients with Crohn's disease are often ineffective in producing long-term healing. We performed a randomized ...

9. academic.oup.com academic.oup.com/ecco-jcc/article/18/Supplement_1/i1058/7586529

P541 Long-term follow-up results of darvadstrocel for complex ... This is the first prospectively observed clinical outcomes in CPF for 3 years after DVS administration. There were no new safety findings.