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

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 4 Years - 17 Years
Gender All
More Inclusion & Exclusion 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. 2. The improvement of, or no worsening in stool frequency, sustained for 1 week or more, in the interval between the colonoscopy, flexible sigmoidoscopy or rectoscopy in inclusion criteria 4(a) and the screening visit. 3. No initiation or intensification of treatment with corticosteroids, immunosuppressants, or monoclonal antibody dose regimen between the colonoscopy, flexible sigmoidoscopy or rectoscopy in inclusion criteria 4(a) and the screening visit.

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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04701411
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Takeda
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Medical Director
Principal Investigator Affiliation Takeda
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Israel, Japan, Netherlands, Poland, Spain
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Crohn's Disease, Complex Perianal Fistula
Study Website: View Trial Website
Additional Details

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.

Arms & Interventions

Arms

Experimental: Darvadstrocel

Darvadstrocel (Cx601), 24 mL suspension of 120 million cells as a perilesional injection, once on Day 0.

Interventions

Biological: - Darvadstrocel

Darvadstrocel perilesional injection.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Shamir Medical Center (Assaf Harofeh), Be'er Ya'aqov, Israel

Status

Not yet recruiting

Address

Shamir Medical Center (Assaf Harofeh)

Be'er Ya'aqov, , 7033001

Site Contact

Site Contact

efratb@asaf.health.gov.il

972897797202

Rambam Health Care Campus, Haifa, Israel

Status

Not yet recruiting

Address

Rambam Health Care Campus

Haifa, , 3109601

Site Contact

Site Contact

r_shaoul@rambam.health.gov.il

97248542914

Wolfson Medical Center, Holon, Israel

Status

Not yet recruiting

Address

Wolfson Medical Center

Holon, , 58100

Site Contact

Site Contact

alevine@wolfson.health.gov.il

972305028808

Shaare Zedek Medical Center, Jerusalem, Israel

Status

Recruiting

Address

Shaare Zedek Medical Center

Jerusalem, , 91031

Site Contact

Site Contact

turnerd@szmc.org.il

97226666482

Hadassah University Hospital-Mt. Scopus, Jerusalem, Israel

Status

Recruiting

Address

Hadassah University Hospital-Mt. Scopus

Jerusalem, , 9124001

Site Contact

Site Contact

zevd@hadassah.org.il

972586645719

Schneider Children's Medical Center, Petach-Tikva, Israel

Status

Not yet recruiting

Address

Schneider Children's Medical Center

Petach-Tikva, , 4920235

Site Contact

Site Contact

shamirraanan@gmail.com

972309253672

Juntendo University Hospital, Bunkyo-ku, Japan

Status

Not yet recruiting

Address

Juntendo University Hospital

Bunkyo-ku, , 113-8431

Site Contact

Site Contact

t-kudo@juntendo.ac.jp

81338133111

Bunkyo-ku, Japan

Status

Not yet recruiting

Address

Medical Hospital, Tokyo Medical and Dental University

Bunkyo-ku, , 113-8519

Site Contact

Site Contact

nagahori.gast@tmd.ac.jp

81338136111

Miyagi Children's Hospital, Sendai-shi, Japan

Status

Not yet recruiting

Address

Miyagi Children's Hospital

Sendai-shi, , 989-3126

Site Contact

Site Contact

lastnamefirstname@email.jp

81223915111

Jichi Medical University Hospital, Shimotsuke-shi, Japan

Status

Not yet recruiting

Address

Jichi Medical University Hospital

Shimotsuke-shi, , 329-0498

Site Contact

Site Contact

lastnamefirstname@email.jp

81285442111

Mie University Hospital, Tsu-shi, Japan

Status

Not yet recruiting

Address

Mie University Hospital

Tsu-shi, , 514-8507

Site Contact

Site Contact

ucchie@clin.medic.mie-u.ac.jp

81592321111

Amsterdam UMC, Locatie AMC, Amsterdam, Netherlands

Status

Not yet recruiting

Address

Amsterdam UMC, Locatie AMC

Amsterdam, , 1105 AZ

Site Contact

Site Contact

j.e.vanlimbergen@amsterdamumc.nl

31205669111

Groningen, Netherlands

Status

Not yet recruiting

Address

Universitair Medisch Centrum Groningen (UMCG)

Groningen, , 9713 GZ

Site Contact

Site Contact

p.f.van.rheenen@umcg.nl

31503614717

Erasmus Medisch Centrum, Rotterdam, Netherlands

Status

Not yet recruiting

Address

Erasmus Medisch Centrum

Rotterdam, , 3000 CA

Site Contact

Site Contact

l.deridder@erasmusmc.nl

31107036049

Krakow, Poland

Status

Not yet recruiting

Address

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, , 30-663

Site Contact

Site Contact

misladek@cyf-kr.edu.pl

481265820111440

Gabinet Lekarski Bartosz Korczowski, Rzeszow, Poland

Status

Recruiting

Address

Gabinet Lekarski Bartosz Korczowski

Rzeszow, , 35-302

Site Contact

Site Contact

korczowski@op.pl

48604481752

Warszawa, Poland

Status

Recruiting

Address

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warszawa, , 04-730

Site Contact

Site Contact

j.kierkus@wip.waw.pl

48500111648

Badalona, Spain

Status

Recruiting

Address

Hospital Universitari Germans Trias i Pujol

Badalona, , 8916

Site Contact

Site Contact

moviedo.germanstrias@gencat.cat

34676588434

Hospital Universitari Vall d'Hebron, Barcelona, Spain

Status

Recruiting

Address

Hospital Universitari Vall d'Hebron

Barcelona, , 8035

Site Contact

Site Contact

osegarra@vhebron.net

34932746213

Hospital Sant Joan de Deu, Barcelona, Spain

Status

Recruiting

Address

Hospital Sant Joan de Deu

Barcelona, , 8950

Madrid, Spain

Status

Recruiting

Address

Hospital Infantil Universitario Nino Jesus

Madrid, , 28009

Site Contact

Site Contact

jlorenzo.alonso@salud.madrid.org

34680484490

Madrid, Spain

Status

Recruiting

Address

Hospital Universitario Fundacion Jimenez Diaz

Madrid, , 28040

Site Contact

Site Contact

maria.mibeas@quironsalud.es

34915504800

Hospital Materno-Infantil de Malaga, Malaga, Spain

Status

Recruiting

Address

Hospital Materno-Infantil de Malaga

Malaga, , 29011

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