A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)

Who is this study for? Patients with Desmoid Tumor, Aggressive Fibromatosis
What treatments are being studied? Nirogacestat Oral Tablet
Status: Active, not recruiting
Location: See all (52) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This study evaluates nirogacestat (PF-03084014) in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). In the double-blind phase, half of the participants will receive nirogacestat while the other half will receive placebo. Once participants are eligible to roll into the open-label phase, they will receive nirogacestat.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: No
View:

• Participant has histologically confirmed DT/AF (by local pathologist prior to informed consent) that has progressed by ≥ 20% as measured by RECIST v1.1 within 12 months of the screening visit scan.

• Participant has:

• Treatment naïve, measurably progressing DT/AF that is deemed not amenable to surgery without the risk of significant morbidity; OR

• Recurrent, measurably progressing DT/AF following at least one line of therapy; OR

• Refractory, measurably progressing DT/AF following at least one line of therapy.

• Participant has a DT/AF tumor where continued progressive disease will not result in immediate significant risk to the participant.

• Participant agrees to provide archival or new tumor tissue for re-confirmation of disease.

• If participant is currently being treated with any therapy for the treatment of DT/AF, this must be completed at least 28 days (or 5 half-lives, whichever is longer) prior to first dose of study treatment. All toxicities from prior therapy must be resolved to ≤ Grade 1 or clinical baseline.

• Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 at screening.

• Participant has adequate organ and bone marrow function.

Locations
United States
Arkansas
Arkansas Children's Hospital
Little Rock
California
Ronald Regan UCLA Medical Center
Los Angeles
USC/Norris Comprehensive Cancer Center
Los Angeles
Stanford Cancer Center
Palo Alto
UCSF Mission Bay
San Francisco
Sarcoma Oncology Research Center
Santa Monica
Colorado
University of Colorado Hospital-Anschutz Cancer Pavillion (ACP)
Aurora
Connecticut
Smilow Cancer Hospital at Yale-New Haven
New Haven
Washington, D.c.
Washington Cancer Institute at MedStar Washington Hospital Center
Washington
Florida
Mayo Clinic Florida
Jacksonville
Sylvester Comprehensive Cancer Center
Miami
Illinois
Northwestern Memorial Hospital
Chicago
Massachusetts
Dana-Farber Cancer Institute (DFCI)
Boston
Massachusetts General Hosptial (MGH)
Boston
Maryland
Johns Hopkins Hospital
Baltimore
Michigan
University of Michigan
Ann Arbor
Minnesota
Mayo Clinic Rochester
Rochester
Missouri
Washington Univerisity School of Medicine
Saint Louis
North Carolina
DUMC/Duke Cancer Center
Durham
New York
Northwell Health
Lake Success
Columbia University Medical Center
New York
Memorial Sloan Kettering Cancer Center
New York
Ohio
Cincinnati Childrens's Hospital Medical Center
Cincinnati
James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
Columbus
Oregon
Oregon Health & Science Univeristy-Center for Health & Healing
Portland
Pennsylvania
Abramson Cancer Center at Pennsylvania Hospital
Philadelphia
Fox Chase Cancer Center
Philadelphia
UPMC Hillman Cancer Ceter
Pittsburgh
Tennessee
Henry-Joyce Cancer Clinic
Nashville
Texas
UT Southwestern Medical Center
Dallas
The University of Texas MD Anderson Cancer Center
Houston
Washington
Seattle Cancer Care Alliance
Seattle
Wisconsin
University of Wisconsin Clinical Science Center
Madison
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee
Other Locations
Belgium
Cliniques Universitaires Saint-Luc, Institut Roi Albert II
Brussels
Institut Jules Bordet-Medical Onocology
Brussels
UZ Gent
Gent
UZ Leuven
Leuven
Canada
McGill University Health Centre
Montréal
Princess Margaret Cancer Centre
Toronto
Germany
Helios Klinikum Berlin-Buch
Berlin
Universitaetsklinikum Hamburg-Eppendorf
Hamburg
Universitätsmedizin Mannheim
Mannheim
Italy
IRCCS Istituto Ortopedico Rizzoli
Bologna
Istituto di Candiolo IRCCS Oncologia Medica
Candiolo
Fondazione IRCCS Instituto Nazionale dei Tumori di Milano
Milano
Policlinico Unvrsitario Campus Bio-Medico
Roma
Netherlands
The Netherlands Cancer Institute
Amsterdam
Leiden University Medical Center (LUMC)
Leiden
Radboud University Medical Centre
Nijmegen
United Kingdom
Department of Oncology, University College of London Hospital
London
The Royal Marsden NHS Foundation Trust
London
Time Frame
Start Date: May 15, 2019
Estimated Completion Date: December 31, 2023
Participants
Target number of participants: 142
Treatments
Experimental: Double-Blind Phase - Nirogacestat
Nirogacestat 150 mg by mouth, twice daily
Placebo Comparator: Double-Blind Phase - Placebo
Placebo 150 mg by mouth, twice daily
Experimental: Open-Label Phase - Nirogacestat
Nirogacestat 150 mg by mouth, twice daily
Related Therapeutic Areas
Sponsors
Leads: SpringWorks Therapeutics, Inc.

This content was sourced from clinicaltrials.gov

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