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Gamma Secretase Inhibitor

Nirogacestat for Aggressive Fibromatosis (DeFi Trial)

Phase 3
Waitlist Available
Led By Bernd Kasper, MD
Research Sponsored by SpringWorks Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
Awards & highlights

DeFi Trial Summary

This trial will evaluate if nirogacestat is effective in treating desmoid tumor/aggressive fibromatosis. Half the participants will receive nirogacestat, the other half placebo, in the double-blind phase. All participants will receive nirogacestat in the open-label phase.

Who is the study for?
This trial is for adults with a type of tumor called desmoid tumor/aggressive fibromatosis (DT/AF) that has grown by at least 20% in the past year. Candidates must have finished any previous treatments for DT/AF at least 28 days before starting the study and resolved all treatment side effects to mild levels. They should be able to perform daily activities with slight limitations or better, as assessed by their ECOG performance status.Check my eligibility
What is being tested?
The trial is testing nirogacestat, an oral tablet, against a placebo (a pill without active medication). Participants are randomly assigned to one of these two groups in a double-blind phase where neither they nor the researchers know who's receiving which treatment. After this phase, all participants will receive nirogacestat during an open-label phase.See study design
What are the potential side effects?
While specific side effects for nirogacestat aren't listed here, common types of side effects from medications like it may include digestive issues, fatigue, skin reactions, changes in liver function tests and potentially increased risk of infections.

DeFi Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of progression free survival (PFS) events as defined as the time from randomization until date of assessment of progression or death by any cause.
Secondary outcome measures
Change from baseline in PRO scores using the Brief Pain Inventory (BPI) short form.
Change from baseline in PRO scores using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC) QLQ-C30.
Change from baseline in PRO scores using the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Impact Scale (GODDESS);
+6 more

DeFi Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Open-Label Phase - NirogacestatExperimental Treatment1 Intervention
Nirogacestat 150 mg by mouth, twice daily
Group II: Double-Blind Phase - NirogacestatExperimental Treatment1 Intervention
Nirogacestat 150 mg by mouth, twice daily
Group III: Double-Blind Phase - PlaceboPlacebo Group1 Intervention
Placebo 150 mg by mouth, twice daily

Find a Location

Who is running the clinical trial?

SpringWorks Therapeutics, Inc.Lead Sponsor
12 Previous Clinical Trials
824 Total Patients Enrolled
Bernd Kasper, MDPrincipal InvestigatorMannheim University Medical Center
1 Previous Clinical Trials
52 Total Patients Enrolled

Media Library

Nirogacestat (Gamma Secretase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03785964 — Phase 3
Aggressive Fibromatosis Research Study Groups: Double-Blind Phase - Placebo, Double-Blind Phase - Nirogacestat, Open-Label Phase - Nirogacestat
Aggressive Fibromatosis Clinical Trial 2023: Nirogacestat Highlights & Side Effects. Trial Name: NCT03785964 — Phase 3
Nirogacestat (Gamma Secretase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03785964 — Phase 3
~23 spots leftby May 2025
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