Clinical trial · no report yet · Phase 3
DeFi
Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)
- NCT03785964 ↗
- Completed
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Deep
We haven't built a report for this trial yet. This page shows the registry record and the public commentary we found that names the trial. A report reads that commentary and sets out what clinicians said about the trial, each point linked to its source. How reports are made.
Preliminary discovery
Commentary in our index that names this trial so far
Podcasts, YouTube videos and X posts we have indexed. A report screens them, searches for more and cites what clinicians said when it is built, so final coverage is set at build.
| Channel | Found | |
|---|---|---|
| YouTube | 20 | |
| X threads | 15 | |
| Podcasts | 14 | |
| Bluesky threads | 1 |
The trial
ClinicalTrials.gov- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
- Phase
- Phase 3
- Status
- Completed
- Conditions
- Desmoid Tumor; Aggressive Fibromatosis
- Interventions
- Nirogacestat oral tablet; Placebo Oral Tablet
- Arms
- Double-Blind Phase - Nirogacestat · Experimental
- Double-Blind Phase - Placebo · Placebo comparator
- Open-Label Phase - Nirogacestat · Experimental
- Enrollment
- 142
- Primary outcomes
- Progression Free Survival (PFS) Defined as the Time From Randomization Until Date of Assessment of Progression or Death by Any Cause.
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Start
- Apr 17, 2019
- Primary completion
- Apr 7, 2022
- Results posted
- Yes
- Registry record
- NCT03785964 · updated Aug 20, 2026
Publications
linked from the registry- Ratan R, Kasper B, Alcindor T, Schoffski P, van der Graaf WTA, Federman N, Bui NQ, D'Amato G, Riedel RF, Attia S, Chawla S, Lim A, Tumminello B, Oton AB, Chu Y, Zhou S, Gounder M. Efficacy and Safety of Long-Term Continuous Nirogacestat Treatment in Adults With Desmoid Tumors: Results From the DeFi Trial. J Clin Oncol. 2025 Dec;43(34):3646-3651. doi: 10.1200/JCO-25-00582. Epub 2025 Oct 20. PubMed 41115259
- Gounder MM, Atkinson TM, Bell T, Daskalopoulou C, Griffiths P, Martindale M, Smith LM, Lim A. GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS(c)): psychometric properties and clinically meaningful thresholds as assessed in the Phase 3 DeFi randomized controlled clinical trial. Qual Life Res. 2023 Oct;32(10):2861-2873. doi: 10.1007/s11136-023-03445-7. Epub 2023 Jun 22. PubMed 37347393
- Gounder M, Ratan R, Alcindor T, Schoffski P, van der Graaf WT, Wilky BA, Riedel RF, Lim A, Smith LM, Moody S, Attia S, Chawla S, D'Amato G, Federman N, Merriam P, Van Tine BA, Vincenzi B, Benson C, Bui NQ, Chugh R, Tinoco G, Charlson J, Dileo P, Hartner L, Lapeire L, Mazzeo F, Palmerini E, Reichardt P, Stacchiotti S, Bailey HH, Burgess MA, Cote GM, Davis LE, Deshpande H, Gelderblom H, Grignani G, Loggers E, Philip T, Pressey JG, Kummar S, Kasper B. Nirogacestat, a gamma-Secretase Inhibitor for Desmoid Tumors. N Engl J Med. 2023 Mar 9;388(10):898-912. doi: 10.1056/NEJMoa2210140. PubMed 36884323