Table 4.
Overview of selected clinical trials with rubitecan in patients with sarcoma
| Study reference | Study phase | Tumor type | Treatment | Patients, n | Most frequent AE | Efficacy ORR | mPFS | mOS |
|---|---|---|---|---|---|---|---|---|
| Phase 1 studies in patients with sarcoma | ||||||||
| NCT00492141 | 1–2 | Ewing sarcoma | L9-NC aerosol alone | 10 | NA | NA | NA | NA |
| L9-NC aerosol + temozolomide | ||||||||
| In patients over the age of 10 years | ||||||||
| Phase 2 studies in patients with sarcoma | ||||||||
| Patel et al. [52] 2003 | 2 | Advanced STS | 9-NC 1.5 mg/m2/day (D1–5) orally q4w | 56 | Fatigue, anemia, neutropenia, thrombocytopenia, diarrhea* | 5% | 2 mo | 12 mo |
| In adult patients | (17 GI leiomyosarcoma; 39 other STS) | (8% [3/39] PR in other STS) | ||||||
| (CBR: 31%) | ||||||||
| Churgh et al. [98] (2005) | 2 | Advanced chordoma, STS, and GIST | 9-NC 1.25 mg/m2/day (D1–5) orally followed by 2 days rest | 51 | Anemia, hyperglycemia, nausea, leukopenia | Chordoma: 7% | mTTP: | NA |
| Chordoma: 9.9 wks | ||||||||
| In adult patients | (15 chordoma 23 STS, 13 GIST) | STS: 4% | STS: 8.0 wks | |||||
| GIST: 8.3 wks | ||||||||
9-NC, 9-nitrocamptothecin; AE, adverse event; CBR, clinical benefit rate (CR + PR + SD); CR, complete response; D, day; GI, gastrointestinal; GIST, gastrointestinal stromal tumor; L9-NC, liposomal 9-nitro-20-(S)-camptothecin; m, median; mo, months; n, number of patients; NA, not available; ORR, objective response rate; OS, overall survival; PD, progressive disease; PFS, progression-free survival; PR, partial response; q, every; q4w, every 4 weeks; SD, stable disease; STS, soft tissue sarcoma; TTP, time to progression; wk, week.
*Grade 3/4 AEs.