MedTrace Logo

A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery

NCT04195399 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 31

Last updated 2026-04-15

No results posted yet for this study

Summary

This phase II trial studies the side effects and how well nirogacestat works in treating patients less than 18 years of age with desmoid tumors that has grown after at least one form of treatment by mouth or in the vein that cannot be removed by surgery. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Conditions

  • Desmoid Fibromatosis
  • Recurrent Desmoid Fibromatosis
  • Unresectable Desmoid Fibromatosis

Interventions

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

PROCEDURE

Computed Tomography

Undergo CT

PROCEDURE

Echocardiography Test

Undergo ECHO

PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

DRUG

Nirogacestat

Given PO

OTHER

Quality-of-Life Assessment

Ancillary studies

OTHER

Questionnaire Administration

Ancillary studies

PROCEDURE

X-Ray Imaging

Undergo x-ray

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    collaborator NIH

  • SpringWorks Therapeutics, Inc.

    collaborator INDUSTRY

  • Children's Oncology Group

    lead NETWORK

Principal Investigators

  • Fariba Navid · Children's Oncology Group

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-07
Primary Completion
2026-03-31
Completion
2027-04-10
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • New Zealand
  • Puerto Rico

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04195399 on ClinicalTrials.gov