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

NCT ID: NCT04701411

Last Updated: 2025-11-13

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-30
• Study Completion Date: 2025-05-07

Brief Summary

A perianal fistula is an abnormal passageway that develops between the rectum and the skin near the anus. The fistula is considered complex if it branches into several openings or an abscess is also present.

The main aim of this study is to learn if complex perianal fistulas in children and teenagers close after treatment with darvadstrocel.

2 to 3 weeks before treatment with darvadstrocel, each participant will have surgery to clean the fistula and to drain any abscesses. On the day of treatment, each participant will have the fistula cleaned and will receive an injection of darvadstrocel near the fistula, under anesthetic.

For up to 1 year after treatment, participants will regularly visit the clinic for follow-up. The fistula will be examined and any side effects from the treatment will be recorded. Participants will have an MRI at one clinic visit (about 24 weeks after treatment).

Detailed Description

The drug being tested in this study is called darvadstrocel (Cx601, cell suspension containing 120 million cells of allogeneic expanded adipose-derived mesenchymal stem cells [eASCs]). Darvadstrocel is being tested to treat complex perianal fistula in pediatric participants who have Crohn's disease (CD). This study will look at the safety and efficacy of darvadstrocel in the treatment of complex perianal fistula in CD.

The study will enroll at least 20 patients who will receive a single dose of darvadstrocel.

This multi-center trial will be conducted worldwide. The overall time to participate in this study is 52 weeks.

Participants will make multiple visits to the clinic. In unavoidable circumstances, such as the coronavirus disease 2019 pandemic, exceptions may be granted for alternative methods for conducting participant visits with approval by the medical monitor and/or sponsor.

Conditions

Crohn's Disease; Complex Perianal Fistula

Keywords

Drug therapy.

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Darvadstrocel

Participants were administered darvadstrocel (Cx601), 24 milliliters (mL) suspension of 120 million cells as a perilesional injection, once on Day 0.

Group Type: EXPERIMENTAL

Darvadstrocel

Intervention Type: BIOLOGICAL

Darvadstrocel perilesional injection.

Interventions

Darvadstrocel

Darvadstrocel perilesional injection.

Intervention Type: BIOLOGICAL

Other Intervention Names

Cx601

Eligibility Criteria

Inclusion Criteria

1. Has a CD diagnosis based on accepted clinical, endoscopic, histological and/or radiologic criteria at least 6 months before the screening visit.

2. Has complex perianal fistula refractory to at least one of the following treatments: immunosuppressants or biologics (anti-TNFs, anti-integrin, anti-interleukin [IL] 12/23). Fistula(s) refractory to therapy is defined in this study as follows: Immunosuppressants: Inadequate response after 3 months, based on clinical assessment, or more treatment with azathioprine, 6-mercaptopurine or methotrexate. Biologics: Inadequate response after 14 weeks (16 weeks for anti-IL 12/23), based on clinical assessment, or more standard treatment for induction and maintenance.

3. A complex perianal fistula(s) that meets one or more of the following criteria, modified from the American Gastroenterological Association (AGA) technical review: High intersphincteric, transsphincteric, extrasphincteric, or suprasphincteric as assessed by MRI. Presence of 2 or 3 external openings (tracts) as assessed by clinical examination. Associated fluid (abscess) collections as determined by MRI.

This study requires that the participant has complex perianal fistulas with a maximum of 2 internal openings and a maximum of 3 external openings, based on clinical assessment. Darvadstrocel treatment is targeted for fistulas that connect between internal and external openings. A central reading of a locally performed pelvic MRI will be performed to confirm the location of the fistula and potential associated perianal abscess(es). Fistulas must have been draining for at least 6 weeks before the screening visit. Participants with actively draining simple subcutaneous fistulas, at the time of the screening visit, are not allowed in this study.

4. Has inactive or mildly active luminal CD defined by meeting all of the following criteria:

1. Colonoscopy, flexible sigmoidoscopy or rectoscopy performed either at screening or within the 6 months before screening, demonstrating no rectal ulcers larger than 0.5 cm. A participant who has documented rectal ulcers larger than 0.5 cm within the 6 months before screening but has undergone subsequent treatment may be eligible if there are no rectal ulcers larger than 0.5 cm on a sigmoidoscopy or rectoscopy performed after treatment or at the time of screening.

Exclusion Criteria

1. Has received any investigational compound within 12 weeks/84 days before screening.

2. Has received darvadstrocel/eASC in a previous clinical study or as a therapeutic agent.

3. The participant weighs <10 kg at screening.

4. Has concomitant perianal fistula(s) with only internal or external opening(s).

5. Has concomitant internal fistula(s) such as ileo-vesical, rectovaginal or ileo-colonic fistula(s).

6. Has an abscess >2 cm, unless resolved in the preparation procedure.

7. Has rectal and/or anal stenosis, and/or active proctitis, which would restrict the surgical procedure.

8. The participant underwent surgery for the fistula other than drainage or seton placement.

9. Has diverting stomas.

10. Has ongoing systemic corticosteroid treatment or has been treated with systemic corticosteroids within 4 weeks before screening.

11. The participant requires new treatment with immunosuppressants/anti-TNF agents during the screening period.

12. The participant has known or suspected COVID-19 by the investigator within the past 2 months (additional testing may be performed at the discretion of the investigator). Positive antibody testing for COVID without other evidence of current or recent active infection does not exclude participation. Participants who were in screening at the time that COVID-19-related factors resulted in discontinuation may also be rescreened with approval of the sponsor or designee.

13. The participant requires surgery in the perianal region for reasons other than fistulas at the time of screening or foreseen either during the study and/or during the 24 weeks after treatment administration.

14. Has malignant tumor or a prior history of any malignant tumor, including any type of fistula carcinoma.

15. Has current or recent (within 3 months before the screening) history of abnormal, severe, progressive, uncontrolled hepatic, hematologic, gastrointestinal (except CD), endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral disease.

16. Has either congenital or acquired immunodeficiencies, including participants known to be HIV carriers or participants with, in the judgment of the investigator, are suspected to have monogenic inflammatory bowel disease.

17. Has previously received a bone marrow transplant.

18. Has a contraindication to MRI scan or other planned study procedures.

19. Has a contraindication to the anesthetic procedure.

20. Had major surgery or severe trauma within 6 months before the screening visit.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda Development Center Americas, Inc.

INDUSTRY

Sponsor Role: collaborator

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Takeda

Locations

Shamir Medical Center (Assaf Harofeh)

Be’er Ya‘aqov, , Israel

Rambam Health Care Campus

Haifa, , Israel

Shaare Zedek Medical Center

Jerusalem, , Israel

Hadassah University Hospital-Mt. Scopus

Jerusalem, , Israel

Schneider Children's Medical Center

Petah Tikva, , Israel

Juntendo University Hospital

Bunkyō City, , Japan

Medical Hospital, Tokyo Medical and Dental University

Bunkyō City, , Japan

Miyagi Children's Hospital

Sendai, , Japan

Jichi Medical University Hospital

Shimotsuke-shi, , Japan

Mie University Hospital

Tsu, , Japan

Amsterdam UMC, Locatie AMC

Amsterdam, , Netherlands

Universitair Medisch Centrum Groningen (UMCG)

Groningen, , Netherlands

Erasmus Medisch Centrum

Rotterdam, , Netherlands

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, , Poland

Gabinet Lekarski Bartosz Korczowski

Rzeszów, , Poland

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, , Poland

Hospital Universitari Germans Trias i Pujol

Badalona, , Spain

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Hospital Sant Joan de Deu

Barcelona, , Spain

Hospital Infantil Universitario Nino Jesus

Madrid, , Spain

Hospital Universitario Fundacion Jimenez Diaz

Madrid, , Spain

Hospital Materno-Infantil de Malaga

Málaga, , Spain

Countries

Israel; Japan; Netherlands; Poland; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://clinicaltrials.takeda.com/study-detail/60096520565ce300294c6adf

To obtain more information on the study, click here/on this link

Other Identifiers

2020-003193-48

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EMEA-001561-PIP01-13-M02

Identifier Type: OTHER

Identifier Source: secondary_id

2023-503973-39-00

Identifier Type: CTIS

Identifier Source: secondary_id

jRCT2033200314

Identifier Type: REGISTRY

Identifier Source: secondary_id

Darvadstrocel-3004

Identifier Type: -

Identifier Source: org_study_id