NCT03279081

Brief Summary

The purpose of this study is to evaluate the combined remission of complex perianal fistulas, defined as the clinical assessment at Week 24 of closure of all treated external openings that were draining at baseline despite gentle finger compression, and absence of collections greater than (\>) 2 centimeter (cm) (in at least 2 dimensions) confirmed by blinded central magnetic resonance imaging (MRI) assessment at Week 24.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
568

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Sep 2017

Longer than P75 for phase_3

Geographic Reach
15 countries

159 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 21, 2017

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 12, 2017

Completed
3 days until next milestone

Study Start

First participant enrolled

September 15, 2017

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 23, 2023

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 26, 2023

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

September 19, 2024

Completed
Last Updated

September 19, 2024

Status Verified

August 1, 2024

Enrollment Period

5.4 years

First QC Date

July 21, 2017

Results QC Date

July 12, 2024

Last Update Submit

August 22, 2024

Conditions

Keywords

Crohn's diseasecomplex perianal fistula(s)

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Combined Remission at Week 24

    Combined remission was 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 blinded central magnetic resonance imaging (MRI) assessment. Percentages are rounded off to whole number at the nearest decimal.

    Week 24

Secondary Outcomes (13)

  • Percentage of Participants With Clinical Remission at Week 24

    Week 24

  • Time to Clinical Remission at Week 24

    Week 24

  • Percentage of Participants With Combined Remission at Week 52

    Week 52

  • Percentage of Participants With Clinical Remission at Week 52

    Week 52

  • Percentage of Participants With Clinical Response at Week 24

    Week 24

  • +8 more secondary outcomes

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo (saline) 24 milliliters (mL) was administered once by local injection.

Other: Placebo

Cx601

EXPERIMENTAL

Cx601 expanded adipose-derived stem cells (eASCs) 120 million cells (5 million cells per mL) was administered once by local injection.

Drug: Cx601

Interventions

Cx601DRUG

Cx601 eASCs intralesional injection.

Cx601
PlaceboOTHER

Cx601 placebo-matching eASCs intralesional injection.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Signed informed consent.
  • Participants of either gender greater than or equal to (\>=) 18 years and less than or equal to (\<=) 75 years of age.
  • Participants with CD diagnosed at least 6 months prior to Screening visit in accordance with accepted clinical, endoscopic, histological and/or radiological criteria.
  • Presence of complex perianal fistula(s) with a maximum of 2 internal openings and a maximum of 3 external openings based on clinical assessment; a central reading of a locally performed contrast enhanced (gadolinium) pelvic MRI will be performed to confirm location of the fistula and potential associated perianal abscess(es). Fistula(s) must have been draining for at least 6 weeks prior to Screening visit. Actively draining simple subcutaneous fistula(s), at the time of Screening visit, are not allowed in this study. A complex perianal fistula is defined as a fistula that meets one or more of the following criteria :
  • High inter-sphincteric, high trans-sphincteric, extra-sphincteric or suprasphincteric.
  • Presence of \>=2 external openings.
  • Associated perianal abscess(es). Note: Abscesses that are larger than 2 cm at least 2 dimensions on MRI must be confirmed to have been drained adequately by the surgeon during the preparation curettage in order to be eligible.
  • Clinically controlled, nonactive or mildly active CD, during the last six months prior to Screening visit with:
  • A patient reported outcomes (PRO-2) score \<14 at Screening, AND
  • A colonoscopy documenting the absence of ulcers larger than 0.5 cm in the colonic mucosa:
  • \- If colonoscopy data are not available within 6 months prior to Screening:
  • A simple endoscopic score for Crohn's Disease (SES-CD) \<=6 with absence of rectal ulcers larger than 0.5 cm must be documented in a colonoscopy performed at Screening before randomization.
  • \- If colonoscopy data are available within 6 months prior to Screening, the following must be documented, otherwise a new colonoscopy (as above) will be mandatory:
  • The absence of ulcers larger than 0.5 cm in the colonic mucosa AND
  • the improvement or no worsening in abdominal pain and/or in the diarrhea, sustained for one week or more, since the last colonoscopy was performed in the clinical records until Screening visit.
  • +13 more criteria

You may not qualify if:

  • Concomitant rectovaginal or rectovesical fistula(s).
  • Participant naïve to prior specific medical treatment for complex perianal fistula(s) including immunosuppressant (IS) or anti-TNFs.
  • Presence of a perianal collection \>2 cm in at least two dimensions on the central reading MRI at Screening visit that was not adequately drained as confirmed by the surgeon during the preparation procedure (week -3 to day 0).
  • Severe rectal and/or anal stenosis and/or severe proctitis (defined as the presence of large \>0.5 cm ulcers in the rectum) that make impossible to follow the surgery procedure manual.
  • Participant with diverting stomas.
  • Active, uncontrolled infection requiring parenteral antibiotics.
  • Participant with ongoing systemic or rectal steroids for CD in the last 2 weeks prior to the Preparation visit.
  • Participants with major alteration on any of the following laboratory tests or increased risk for the surgical procedure:
  • Serum creatinine levels \>1.5 times the ULN
  • Total bilirubin \>1.5 ULN
  • Aspartate Transaminase (AST)/ Alanine Transaminase (ALT) \>3 times ULN
  • Hemoglobin \<10.0 g/dL
  • Platelets \<75.0\*10\^9/L
  • Albuminemia \<3.0 g/dL
  • Suspected or documented infectious enterocolitis within two weeks prior to Screening visit.
  • +14 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (159)

Scottsdale Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

UC San Diego Health Systems

La Jolla, California, 92037, United States

Location

University of Southern California (USC) Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

UC Irvine Medical Center - Chao Family Comprehensive Cancer

Orange, California, 92868, United States

Location

Inland Empire Liver Foundation

Rialto, California, 92377, United States

Location

Kaiser Permamente

San Francisco, California, 91115, United States

Location

University of California San Francisco

San Francisco, California, 94115, United States

Location

Vallejo Hospital and Medical Offices

Vallejo, California, 94589, United States

Location

Cedar-Sinai Medical Center

West Hollywood, California, 90048, United States

Location

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

Hartford Hospital - Gastroenterology

Farmington, Connecticut, 06032, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06519, United States

Location

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20017, United States

Location

Mayo Clinic - Gastroenterology

Jacksonville, Florida, 32224, United States

Location

University of Miami Hospital

Miami, Florida, 33136, United States

Location

Florida Hospital Orlando

Orlando, Florida, 32804, United States

Location

USF Health South Tampa Center for Advanced Healthcare

Tampa, Florida, 33606, United States

Location

Florida Hospital Tampa

Tampa, Florida, 33613, United States

Location

Cleveland Clinic Florida

Weston, Florida, 33331, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

The University of Chicago Medicine - Colon & Rectal Surgery

Chicago, Illinois, 60637, United States

Location

Carle Foundation Hospital

Urbana, Illinois, 61801, United States

Location

Indiana University - Colon and Rectal

Indianapolis, Indiana, 46237, United States

Location

University of Kansas Sxchool of Medicine

Kansas City, Kansas, 66160, United States

Location

University of Louisville

Louisville, Kentucky, 40292, United States

Location

Digestive Health Center of Louisiana

Baton Rouge, Louisiana, 70809, United States

Location

Colon and Rectal Surgery Associates

Metairie, Louisiana, 70001, United States

Location

University Medical Center - New Orleans

New Orleans, Louisiana, 71103, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins Medicine - The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Massachussetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Medical Center

Boston, Massachusetts, 08807, United States

Location

Lahey Hospital & Medical Center

Burlington, Massachusetts, 01803, United States

Location

University of Massachusetts - colon & rectal surgery

Worcester, Massachusetts, 01605, United States

Location

Mayo Clinic College of Medicine - Division of Colon and Rectal Surgery - Division of Colon and Rectal Surgery

Rochester, Minnesota, 55905, United States

Location

Barnes-Jewish Hospital - Gastroenterology

St Louis, Missouri, 63110, United States

Location

University of Nevada School of Medicine

Las Vegas, Nevada, 89154, United States

Location

Dartmouth Hitchcock Medical Center - Cancer Center

Lebanon, New Hampshire, 03756, United States

Location

Morristown Medical Center - Gastroenterology

Morristown, New Jersey, 07960, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

North Shore University Hospital - Gastroenterology

Manhasset, New York, 11030, United States

Location

NYU Langone Medical Center

New York, New York, 10016, United States

Location

Stony Brook University Medical Center

New York, New York, 10016, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10028, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Lenox Hill Hospital

New York, New York, 10075, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44106, United States

Location

OHSU Digestive Health Center

Portland, Oregon, 97239, United States

Location

Harvard Medical School-Beth Israel Deaconess Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Penn State Hershey Medical Center - Surgery

Hershey, Pennsylvania, 17033, United States

Location

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

University Surgical Associates-Rhode Island Hospital

Providence, Rhode Island, 02904, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Rapid City Medical Center

Rapid City, South Dakota, 57701, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

UT Southwestern Medical Center - Gastroenterology - Gastroenterology

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine (BCM) - Gastroenterology

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

Carilion Clinic

Roanoke, Virginia, 24016, United States

Location

Virginia Mason Medical Center - Gastroenterology

Seattle, Washington, 98101, United States

Location

Swedish Medical Center

Seattle, Washington, 98104, United States

Location

Aurora St. Luke's Medical Center

Milwaukee, Wisconsin, 53215, United States

Location

Medical College of Wisconsin Hub for Collaborative Medicine - Gastroenterology and Hepatology

Milwaukee, Wisconsin, 53226, United States

Location

UZ Leuven - Campus Gasthuisberg

Leuven, Vlaams Brabant, 3000, Belgium

Location

AZ Groeninge - Campus Kennedylaan - Gastro-enterology

Kortrijk, West-Vlaanderen, 8500, Belgium

Location

AZ Delta vzw - Maag-darm-leverziekten

Roeselare, West-Vlaanderen, 8800, Belgium

Location

GZA ziekenhuizen - Campus Sint-Vincentius - Gastro-enterology

Antwerp, 2018, Belgium

Location

Imelda Ziekenhuis

Antwerp, 2820, Belgium

Location

UZ Gent - Gastroenterology

Ghent, 9000, Belgium

Location

CHU de Liege - Domaine Universitaire du Sart Tilman

Liège, 4000, Belgium

Location

Clinique Saint-Joseph (CHC)

Liège, 4000, Belgium

Location

CHU Dinant Godinne UCL Namur

Namur, 5530, Belgium

Location

University of Alberta

Edmonton, Alberta, T6G 2X8, Canada

Location

(G.I.R.I.) GI Research Institute

Vancouver, British Columbia, V6Z 2K5, Canada

Location

Ottawa Hospital

Ottawa, Ontario, K1H 8L6, Canada

Location

Mount Sinai Hospital - Toronto - Gastroenterology

Toronto, Ontario, M5G 1X5, Canada

Location

Kensington Screening Clinic - Gastroenterology

Toronto, Ontario, M5T 3A9, Canada

Location

Centre Hospitalier de l'Universite de Montreal

Montreal, Quebec, H2X 3J4, Canada

Location

McGill University Health Centre - Montreal General Hospital

Montreal, Quebec, H3G 1A4, Canada

Location

NH Hospital a.s.

Hořovice, Beroun, 268 31, Czechia

Location

FN Hradec Kralove

Hradec Králové, 500 05, Czechia

Location

Bispebjerg Hospital

Copenhagen, Copenhague, 2400, Denmark

Location

Aarhus University Hospital - Department of Hepatology and Gastroenterology, Lever-Mave- og Tarmsygdomme Klinik, krydspunkt C216

Aarhus, 8200, Denmark

Location

Odense Universitetshospital

Odense, Denmark

Location

CHU de Nice

Nice, Alpes-Maritimes, 06202, France

Location

CHU de Clermont-Ferrand - Estaing

Clermont-Ferrand, Auvergne, 63003, France

Location

Centre Hospitalier Universitaire De Toulouse - Hopital De Ra

Toulouse, Haute-Garonne, 31059, France

Location

CHRU Hopital De Pontchaillou

Rennes, Ille-et-Vilaine, 35033, France

Location

CHRU de Nancy -Hopital Brabois Adultes - Service d'Hepato- Gastroenterologie

Vandœuvre-lès-Nancy, Lorraine, 54511, France

Location

CHRU De Lille - Hopital Claude Huriez - Hepato-Gastro-Enterologie

Lille, Nord, 59000, France

Location

CHU Saint Etienne

Saint Priest En Jarez, Pays de la Loire Region, 42270, France

Location

CHU Amiens-Picardie

Amiens, Picardie, 80054, France

Location

Centre Hospitalier Lyon Sud

Pierre-Bénite, Rhone, 69495, France

Location

Paris St. Joseph Hospital

Paris, 75014, France

Location

Hopital Beaujon

Clichy, Île-de-France Region, 92110, France

Location

Hopital Saint Louis - Gastro-hepatoenterologie

Paris, Île-de-France Region, 75010, France

Location

Universitatsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

Location

Klinikum der Universitat Munchen - Campus Grosshadern

München, Bavaria, 81377, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitat

Frankfurt am Main, Hesse, 69590, Germany

Location

Universitatsklinikum Dresden

Dresden, Saxony, 01307, Germany

Location

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont - I. sz. Belgyogyaszati Klinika

Szeged, Csongrád megye, 6720, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, Hajdú-Bihar, H-4032, Hungary

Location

MH Egeszsegugyi Kozpont - Gasztroenterologiai Osztaly

Budapest, Pest County, 1062, Hungary

Location

Semmelweis Egyetem

Budapest, Pest County, H-1088, Hungary

Location

Rabin Med Ctr Beilinson Hosp

Petah Tikva, Central District, 4941492, Israel

Location

Shaare Zedek Medical Center - Gastroenterology

Jerusalem, Jerusalem, 9103102, Israel

Location

Hadassah Medical Organization, Hadassah Medical Center, Ein-

Jerusalem, Jerusalem, 91120, Israel

Location

Rambam Medical Centre

Haifa, Northern District, 31096, Israel

Location

Chaim sheba Medical Center

Tel Litwinsky, Tel Aviv, 5262000, Israel

Location

Ospedale Santissima Annunziata

Cento, Ferrara, 44042, Italy

Location

Istituto Clinico Humanitas Rozzano, IRCCS - IBD Center

Rozzano, Milano, 20089, Italy

Location

Policlinico S. Orsola Malpighi, AOU di Bologna-U.O. di Medicina Interna.

Bologna, 40138, Italy

Location

AOU Policlinico di Modena - Gastroenterologia

Modena, 41124, Italy

Location

II Universita degli Studi di Napoli

Napoli, 80131, Italy

Location

Gastroenterology Section

Palermo, 90127, Italy

Location

Universita degli studi di Pisa

Pisa, 56126, Italy

Location

Policlinico Universitario Campus Biomedico - UOC di Gastroenterologia

Roma, 00128, Italy

Location

A.O. San Camillo Forlanini

Roma, 00152, Italy

Location

Complesso Integrato Columbus

Roma, 00168, Italy

Location

Policlinico Universitario Agostino Gemelli

Roma, 00168, Italy

Location

Azienda Ospedaliero Universitaria S.Maria della Misericordia - Gastroenterologia

Udine, 33100, Italy

Location

Azienda Ospedaliera Universitaria Integrata Verona (AOUI) -

Verona, 37134, Italy

Location

Centrum Medyczne PROMED

Krakow, Lesser Poland Voivodeship, 31-411, Poland

Location

Centrum Medyczne Melita Medical

Wroclaw, Lower Silesian Voivodeship, 50-449, Poland

Location

Wielospecjalistyczny Szpital Medicover

Warsaw, Masovian Voivodeship, 03-984, Poland

Location

Endoskopia Sp z o.o.

Sopot, Pomeranian Voivodeship, 81-756, Poland

Location

COPERNICUS Podmiot Leczniczy Sp. z o.o.

Gdansk, 80-803, Poland

Location

Centralny Szpital Kliniczny MSWiA w Warszawie

Warsaw, 02-507, Poland

Location

Uniwersytecki Szpital Kliniczny im. Wojskowej Akademii Medycznej Centralny Szpital Weteranow

Lodz, Łódź Voivodeship, 90-647, Poland

Location

University of Puerto Rico School of Medicine

San Juan, 00936, Puerto Rico

Location

Hospital Universitario Son Espases

Palma de Mallorca, Balearic Islands, 07120, Spain

Location

H.U. G.Trias i Pujol

Badalona, Barcelona, 08916, Spain

Location

Corporacio Sanitaria Parc Tauli

Sabadell, Barcelona, 8208, Spain

Location

Hospital Universitario de Fuenlabrada

Fuenlabrada, Madrid, 28942, Spain

Location

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, 28222, Spain

Location

Hospital de Sagunto

Sagunto, Valencia, 46520, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital del Mar

Barcelona, 8003, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Universitario Fundacion Jimenez Diaz

Madrid, 28040, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

C.H.U. de Pontevedra

Pontevedra, 36071, Spain

Location

H.U.V. del Rocio

Seville, 21005, Spain

Location

Linkoping University Hospital - Department of Surgery

Linköping, Ostergotlands Lan [se-05], 581 85, Sweden

Location

Addenbrooke's Hospital

Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

Location

NHS Greater Glasgow and Clyde - Glasgow Royal Infirmary (GRI)

Glasgow, Glasgow City, G52 3NQ, United Kingdom

Location

St. Mark's Hospital

Harrow, London, City of, HA1 3UJ, United Kingdom

Location

Guys & St Thomas

London, London, City of, SE1 7EH, United Kingdom

Location

Nottingham University Hospitals NHS Trust - Surgery

Nottingham, Nottinghamshire, NG7 2UH, United Kingdom

Location

University Colleague London Hospital (UCLH)

London, NW1 2BU, United Kingdom

Location

Wythenshawe Hospital - Gastroenterology

Manchester, M13 9WL, United Kingdom

Location

Northern General Hospital

Sheffield, S10 2JF, United Kingdom

Location

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

Inflammatory Bowel DiseasesGastroenteritisGastrointestinal DiseasesDigestive System DiseasesIntestinal Diseases

Results Point of Contact

Title
Study Director
Organization
Takeda

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 21, 2017

First Posted

September 12, 2017

Study Start

September 15, 2017

Primary Completion

January 23, 2023

Study Completion

July 26, 2023

Last Updated

September 19, 2024

Results First Posted

September 19, 2024

Record last verified: 2024-08

Data Sharing

IPD Sharing
Will share

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
More information

Locations