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

NCT04701411 | Terminated Phase 3 Results

• 5 other identifiers: Darvadstrocel-30042020-003193-48EMEA-001561-PIP01-13-M022023-503973-39-00jRCT2033200314
• Study Type: interventional
• Target: 7
• Locations: 5 countries
• Sites: 22

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).

Conditions

Crohn's Disease; Complex Perianal Fistula

Keywords

Drug therapy.

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Achieved Combined Remission

  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.

  Week 24

Secondary Outcomes (10)

• Percentage of Participants Who Achieved Clinical Remission

  Weeks 24 and 52

• Percentage of Participants Who Achieved Clinical Response

  Weeks 24 and 52

• Time to Clinical Remission

  Up to Week 52

• Time to Clinical Response

  Up to Week 52

• Percentage of Participants With Relapse in Participants With Combined Remission at Week 24

  From Week 24 to Week 52

Study Arms (1)

Darvadstrocel

EXPERIMENTAL

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

Biological: Darvadstrocel

Interventions

Darvadstrocel BIOLOGICAL

Darvadstrocel perilesional injection.

Also known as: Cx601

Darvadstrocel

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Has a CD diagnosis based on accepted clinical, endoscopic, histological and/or radiologic criteria at least 6 months before the screening visit.
• 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.
• 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.
• Has inactive or mildly active luminal CD defined by meeting all of the following criteria:
• 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.

You may not qualify if:

• Has received any investigational compound within 12 weeks/84 days before screening.
• Has received darvadstrocel/eASC in a previous clinical study or as a therapeutic agent.
• The participant weighs \<10 kg at screening.
• Has concomitant perianal fistula(s) with only internal or external opening(s).
• Has concomitant internal fistula(s) such as ileo-vesical, rectovaginal or ileo-colonic fistula(s).
• Has an abscess \>2 cm, unless resolved in the preparation procedure.
• Has rectal and/or anal stenosis, and/or active proctitis, which would restrict the surgical procedure.
• The participant underwent surgery for the fistula other than drainage or seton placement.
• Has diverting stomas.
• Has ongoing systemic corticosteroid treatment or has been treated with systemic corticosteroids within 4 weeks before screening.
• The participant requires new treatment with immunosuppressants/anti-TNF agents during the screening period.
• 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.
• 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.
• Has malignant tumor or a prior history of any malignant tumor, including any type of fistula carcinoma.
• 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.

Sponsors & Collaborators

• Takeda: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (22)

Shamir Medical Center (Assaf Harofeh)

Be’er Ya‘aqov, 7033001, Israel

Location

Rambam Health Care Campus

Haifa, 3109601, Israel

Location

Shaare Zedek Medical Center

Jerusalem, 91031, Israel

Location

Hadassah University Hospital-Mt. Scopus

Jerusalem, 9124001, Israel

Location

Schneider Children's Medical Center

Petah Tikva, 4920235, Israel

Location

Juntendo University Hospital

Bunkyō City, 113-8431, Japan

Location

Medical Hospital, Tokyo Medical and Dental University

Bunkyō City, 113-8519, Japan

Location

Miyagi Children's Hospital

Sendai, 989-3126, Japan

Location

Jichi Medical University Hospital

Shimotsuke-shi, 329-0498, Japan

Location

Mie University Hospital

Tsu, 514-8507, Japan

Location

Amsterdam UMC, Locatie AMC

Amsterdam, 1105 AZ, Netherlands

Location

Universitair Medisch Centrum Groningen (UMCG)

Groningen, 9713 GZ, Netherlands

Location

Erasmus Medisch Centrum

Rotterdam, 3000 CA, Netherlands

Location

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30-663, Poland

Location

Gabinet Lekarski Bartosz Korczowski

Rzeszów, 35-302, Poland

Location

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, 04-730, Poland

Location

Hospital Universitari Germans Trias i Pujol

Badalona, 8916, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 8035, Spain

Location

Hospital Sant Joan de Deu

Barcelona, 8950, Spain

Location

Hospital Infantil Universitario Nino Jesus

Madrid, 28009, Spain

Location

Hospital Universitario Fundacion Jimenez Diaz

Madrid, 28040, Spain

Location

Hospital Materno-Infantil de Malaga

Málaga, 29011, Spain

Location

Related Links

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

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2021
• First Posted: January 8, 2021
• Study Start: June 30, 2021
• Primary Completion: May 7, 2025
• Study Completion: May 7, 2025
• Last Updated: November 13, 2025
• Results First Posted: October 6, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

PL (3); ES (6); JP (5); NL (3); IL (5)