A phase 2 study of temsirolimus (CCI-779) in patients with soft tissue sarcomas: a study of the Mayo phase 2 consortium (P2C)
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Published on: July 28, 2020
Scott Okuno1, Howard Bailey, Michelle R Mahoney
1Department of Oncology, Mayo Clinic College of Medicine, Rochester, MN 55905, USA. Okuno.scott@mayo.edu
Background:
The primary goal of this trial was to evaluate the confirmed response rate of temsirolimus (CCI-779), a mammalian target of rapamycin in patients with advanced soft tissue sarcomas (STS).
Methods:
Patients ≥18 years with measurable advanced STS, no prior chemotherapy for metastatic disease (adjuvant and neoadjuvant chemotherapy allowed), adequate organ function, and performance status of ≤2 were eligible. After premedication with an antihistamine, CCI-779 was given intravenously at 25 mg over 30 minutes on Days 1, 8, 15, and 22, repeated every 4 weeks. The primary endpoint was confirmed response rate per Response Evaluation Criteria in Solid Tumors.
Results:
Between June 2004 and November 2005, a total of 41 patients were enrolled and began treatment; 40 patients are evaluable for response and adverse events. The median age was 62 years (range, 28-72 years) with 56% women. Eighty percent had high-grade STS, and 22% had prior adjuvant chemotherapy. There were 2 patients (5%; 95% confidence interval [CI], 1-17) (undifferentiated fibrosarcoma and uterine leiomyosarcoma) who achieved a confirmed partial response lasting 3 and 17 months, respectively. Thirty-nine (95%) patients have progressed, with a median time to progression of 2.0 months (95% CI, 1.8-3.5). The median overall survival was 7.6 months (95% CI, 6.1-15.9). Forty-three percent experienced grade 3+ adverse events that were possibly related to therapy.
Conclusions:
Temsirolimus in this patient population of STS had limited clinical activity and had moderate toxicities.
Insights
Temsirolimus showed limited clinical activity in advanced soft tissue sarcomas (STS), with only 5% partial response rate. The drug also presented moderate toxicities in patients with STS.
Area of Science:
- Oncology
- Pharmacology
Background:
- Advanced soft tissue sarcomas (STS) represent a heterogeneous group of malignancies.
- Current treatment options for advanced STS often have limited efficacy.
- Mammalian target of rapamycin (mTOR) inhibitors are being investigated for various cancers.
Purpose of the Study:
- To evaluate the confirmed response rate of temsirolimus (CCI-779) in patients with advanced soft tissue sarcomas (STS).
- To assess the safety and tolerability of temsirolimus in this patient population.
Main Methods:
- A phase II clinical trial was conducted involving patients with measurable advanced STS.
- Eligible patients received intravenous temsirolimus at 25 mg on a weekly schedule for 4 weeks.
- The primary endpoint was the confirmed response rate, assessed using Response Evaluation Criteria in Solid Tumors (RECIST).
Main Results:
- Forty patients were evaluable for response and adverse events.
- Two patients (5%) achieved a confirmed partial response.
- The median time to progression was 2.0 months, and median overall survival was 7.6 months.
- Grade 3+ adverse events possibly related to therapy occurred in 43% of patients.
Conclusions:
- Temsirolimus demonstrated limited clinical activity in patients with advanced STS.
- The drug exhibited moderate toxicities in this patient population.
- Further investigation may be needed to identify potential subsets of STS patients who could benefit from temsirolimus.
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