NCT03649971

Brief Summary

The purpose of this study is to determine the effect of treatment with guselkumab in participants with familial adenomatous polyposis (FAP) on rectal/pouch polyp burden.

Trial Health

93
On Track

Trial Health Score

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

Enrollment
77

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Nov 2018

Typical duration for phase_1

Geographic Reach
9 countries

32 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

August 27, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 28, 2018

Completed
3 months until next milestone

Study Start

First participant enrolled

November 19, 2018

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 13, 2021

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 23, 2022

Completed
Last Updated

February 3, 2025

Status Verified

January 1, 2025

Enrollment Period

2.8 years

First QC Date

August 27, 2018

Last Update Submit

January 31, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Percentage Change from Baseline in Rectal/pouch Polyp Burden at Week 24

    Percentage change from baseline in rectal/pouch polyp burden (sum of the polyp diameters) at Week 24 will be determined through endoscopy.

    Baseline, Week 24

Secondary Outcomes (14)

  • Percentage Change from Baseline in Number of Colorectal Polyps

    Baseline, Weeks 24 and 52

  • Percentage Change from Baseline in Number of J-pouch Polyps

    Baseline, Weeks 24 and 52

  • Percentage Change from Baseline in J-pouch Polyp Burden

    Baseline, Weeks 24 and 52

  • Percentage Change from Baseline in Number of Duodenal Polyps

    Baseline, Weeks 24 and 52

  • Percentage Change from Baseline in Duodenal Polyp Burden

    Baseline, Weeks 24 and 52

  • +9 more secondary outcomes

Study Arms (3)

Guselkumab Dose 1

EXPERIMENTAL

Participants will receive guselkumab Dose 1 subcutaneous (SC), 6 doses every 4 weeks from Week 0 to Week 20. Participants who respond to guselkumab may continue treatment at the same dose level through Week 48.

Drug: Guselkumab

Guselkumab Dose 2

EXPERIMENTAL

Participants will receive guselkumab Dose 2 SC, 6 doses every 4 weeks from Week 0 to Week 20. Participants who respond to guselkumab may continue treatment at the same dose level through Week 48.

Drug: Guselkumab

Placebo

PLACEBO COMPARATOR

Participants will receive placebo SC, 6 doses every 4 weeks from Week 0 to Week 20.

Drug: Placebo

Interventions

Guselkumab SC will be administered every 4 weeks.

Also known as: Tremfya
Guselkumab Dose 1Guselkumab Dose 2

Placebo SC will be administered every 4 weeks.

Placebo

Eligibility Criteria

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

You may qualify if:

  • Phenotypic familial adenomatous polyposis (FAP) with disease involvement of the colorectum by either genetic or clinical diagnosis: Adenomatous polyposis coli (APC) germline mutation with or without family history, or with greater than (\>)100 adenomas in large intestine and a family history of FAP, attenuated FAP is allowed. FAP phenotype post colectomy for polyposis with a family history of FAP may be allowed
  • Post-colectomy or subtotal colectomy
  • Polyps with a sum of diameters greater than or equal to (\>=)10 millimeter (mm) in the rectum or pouch on biopsy at screening
  • A woman of childbearing potential must agree not to get pregnant during the study and at least 12 weeks after the last dose of study administration
  • A woman must agree not to breast feed or donate eggs (ova, oocytes) during the study and for a period of 12 weeks after the last administration of study drug

You may not qualify if:

  • Prior use of any biologic therapy targeting interleukin (IL)-12/23, IL-17, or IL-23 receptor
  • Use of non-steroidal anti-inflammatory drugs other than aspirin during the study. The use 100 milligram (mg) of aspirin a day or 700 mg of aspirin per week is allowed
  • Treatment with other FAP-directed drug therapy (including NSAID \[Nonsteroidal anti-inflammatory drug\] drugs), unless completes a 4-week washout period prior to randomization
  • High grade dysplasia or cancer on biopsy at screening in GI tract (including stomach, duodenum, and colon/rectum/pouch)
  • Duodenal, colorectal, or pouch polyp: \>2 centimeter (cm) unless excised at the screening evaluation; and 1 to 2 cm with evidence of high-grade dysplasia upon biopsy unless excised

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (32)

Mayo Clinic

Phoenix, Arizona, 85054, United States

Location

City of Hope

Duarte, California, 91010, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

Ochsner Medical Center

New Orleans, Louisiana, 70121, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Washington University School Of Medicine

St Louis, Missouri, 63110, United States

Location

Herbert Irving Comprehensive Cancer Center Columbia University Medical Center

New York, New York, 10032, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Wexner Medical Center at the Ohio State University

Columbus, Ohio, 43210, United States

Location

University of Pennsylvania - Perelman School of Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Utah Huntsman Cancer Institute

Salt Lake City, Utah, 84132, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Hopital Edouard Herriot - CHU Lyon

Lyon, 69437, France

Location

APHM Hopital Timone

Marseille, 13385, France

Location

Universitatsklinikum Bonn

Bonn, 53127, Germany

Location

Universitatsklinikum Ulm

Ulm, 89070, Germany

Location

Sourasky MC

Tel Aviv, 64239, Israel

Location

Academisch Medisch Centrum Universiteit van Amsterdam

Amsterdam, 1105 AZ, Netherlands

Location

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

Szpital Kliniczny im Heliodora Swiecickiego Uniwersytetu Medycznego im Karola Marcinkowskiego w Po

Poznan, 60 355, Poland

Location

Narodowy Instytut Onkologii im Marii Sklodowskiej Curie Panstwowy Instytut Badawczy

Warsaw, 02-781, Poland

Location

Pan American Center for Oncology Trials LLC

Rio Piedras, 00935, Puerto Rico

Location

Hosp Univ Vall D Hebron

Barcelona, 8035, Spain

Location

Hosp Clinic de Barcelona

Barcelona, 8036, Spain

Location

Karolinska Universitetssjukhuset

Stockholm, 171 76, Sweden

Location

Related Publications (1)

  • Stone JK, Mehta NA, Singh H, El-Matary W, Bernstein CN. Endoscopic and chemopreventive management of familial adenomatous polyposis syndrome. Fam Cancer. 2023 Oct;22(4):413-422. doi: 10.1007/s10689-023-00334-3. Epub 2023 Apr 29.

MeSH Terms

Conditions

Adenomatous Polyposis Coli

Interventions

guselkumab

Condition Hierarchy (Ancestors)

Adenomatous PolypsAdenomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsColorectal NeoplasmsIntestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteNeoplastic Syndromes, HereditaryDigestive System DiseasesGastrointestinal DiseasesColonic DiseasesIntestinal DiseasesIntestinal PolyposisGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Janssen Research & Development, LLC Clinical Trial

    Janssen Research & Development, LLC

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 27, 2018

First Posted

August 28, 2018

Study Start

November 19, 2018

Primary Completion

September 13, 2021

Study Completion

March 23, 2022

Last Updated

February 3, 2025

Record last verified: 2025-01

Locations