TABLE 2.
Clinical trials of investigational agents for patients with desmoid tumor (searched April 27, 2022)
| Trial identifier | Agent | Status | Phase | No. of patients | Key inclusion criteria | Primary endpoint | Estimated completion |
|---|---|---|---|---|---|---|---|
| NCT04871282 (RINGSIDE) | AL102 | Recruiting | 2/3 | 192 | Aged ≥18 years, TN or R/R DT | PFS | February 2025 |
| NCT01981551 (Kummar 2017 89 ) | Nirogacestat | Active, not recruiting | 2 | 17 | Aged ≥ 18 years, DT progressing after one or more prior systemic therapy and not amenable to surgery | ORR | September 2022 |
| NCT04195399 | Nirogacestat | Recruiting | 2 | 35 | Aged 1–18 years, progressing DT not amenable to surgery, one or more prior systemic therapy | PFS | December 2024 |
| NCT03785964 (DeFi) | Nirogacestat | Active, not recruiting | 3 | 142 | Aged ≥18 years, progressing TN and not amenable to surgery, or R/R DT | PFS | March 2023 |
| NCT03459469 | Tegavivint | Active, not recruiting | 1 | 24 | Aged ≥18 years, TN unresectable DT or progressing or symptomatic R/R DT | Safety, tolerability | November 2021 |
| NCT03802084 | Vactosertib + imatinib | Recruiting | 1/2 | 24 | Aged ≥19 years, DT not amenable to surgery or RT | Adverse events | December 2021 |
| NCT02834013 | Nivolumab + ipilimumab | Recruiting | 2 | 818 | Aged ≥18 years, histologically confirmed rare cancer, including DT | ORR | October 2023 |
| NCT01265030 | Sirolimus | Completed | 1/2 | 9 | Aged ≤29 years, TN or R/R DT planning to undergo surgery | mTOR pathway activation | June 2021 |
Abbreviations: DT, desmoid tumor; mTOR, mammalian target of rapamycin; NCT, ClinicalTrials.gov identification number; ORR, overall response rate; PFS, progression‐free survival; R/R, relapsed or refractory; TN, treatment‐naive.