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临床试验/NCT05253131
NCT05253131
尚未招募
2 期

Phase 1/2 Trial of the MEK Inhibitor Selumetinib and Bromodomain Inhibitor ZEN-3694 With Durvalumab (MEDI4736), a PD-L1 Antibody for Sarcomas Including Malignant Peripheral Nerve Sheath Tumors

University of Alabama at Birmingham1 个研究点 分布在 1 个国家目标入组 41 人2026年5月15日

概览

阶段
2 期
干预措施
Selumetinib + ZEN-3694 ± Durvalumab
疾病 / 适应症
MPNST
发起方
University of Alabama at Birmingham
入组人数
41
试验地点
1
主要终点
Part A: Safety and Tolerability Selumetinib with ZEN-3694
状态
尚未招募
最后更新
2个月前

概览

简要总结

A multi-institutional open-label phase 1/2 trial of selumetinib in combination with ZEN-3694 and durvalumab in refractory/unresectable sarcomas including MPNST. The phase 1 portion will be separated in two parts and will be open to all patients with refractory/relapsed sarcomas. The phase 2 portion will be for patients with refractory/unresectable NF1-associated MPNST.

详细描述

A multi-institutional open-label phase 1/2 trial of selumetinib in combination with bromodomain inhibitor (ZEN-3694) and durvalumab in refractory/unresectable sarcomas including MPNST. The phase 1 portion will be separated in two parts and will be open to all patients with refractory/relapsed sarcomas. The phase 2 portion will be for patients with refractory/unresectable NF1-associated MPNST. Part A will be a phase 1 dose escalation study of the combination with selumetinib and ZEN-3694. Part B will be phase 1 study combining the determined dose of selumetinib and ZEN-3694 from Part A with durvalumab. Part C will be a phase 2 study combining selumetinib, ZEN-3694 with durvalumab in MPNST patients at the recommended doses from part B. A Simon's two-stage design will be used in the phase 2 trial to determine the clinical benefit in patients with unresectable or metastatic NF associated MPNST. Statistical Plan Phase 1: Conventional dose escalation schema. Cohorts of 3 to 6 participants will be treated per dose level. At the RP2D or last dose level, the cohort may be expanded to up to an additional six participants for further pharmacokinetic and tolerability experience. The MTD/RP2D will be defined as the dose level immediately below the level at which ≥33% of participants in a cohort experience a DLT based on toxicities observed in the first drug therapy cycle. Phase 2: A Simon's two-stage phase 2 trial of selumetinib, ZEN-3694, and durvalumab to determine the safety and clinical benefit in patients with unresectable or metastatic MPNST Maximum Total Number of Subjects Phase 1: 6-24 participants Phase 2: 9 participants in first stage with additional 8 participants in stage 2. Target Population Individuals ≥ 18 years of age with relapsed or refractory histologically confirmed sarcoma including MPNST. This may be amended when tolerability is established. Anticipated Length of Study Maximum enrollment number for entire study is 41participants. It is expected that 15-25 participants will be enrolled per year, and enrollment is expected to be completed in 3 years with follow up after last participant accrual to be approximately 12 months.

注册库
clinicaltrials.gov
开始日期
2026年5月15日
结束日期
2032年5月15日
最后更新
2个月前
研究类型
Interventional
研究设计
Sequential
性别
All

研究者

责任方
Principal Investigator
主要研究者

Girish Dhall, MD

PI, NFCTC

University of Alabama at Birmingham

入排标准

入选标准

  • Inclusion Criteria AGE: ≥ 18 years of age Weight: \>30 kg Life expectancy of at least 12 weeks
  • Part A and B (Phase 1): Patients with histologically confirmed soft tissue or bone sarcoma of the following subtypes:
  • MFH/ undifferentiated pleomorphic sarcoma
  • Unclassified sarcoma
  • Rhabdomyosarcoma
  • Malignant peripheral nerve sheath tumor (MPNST)
  • Osteosarcoma
  • Ewing or Ewing-like sarcoma
  • Synovial sarcoma
  • Desmoplastic small round blue cell tumor (DSRCT)

排除标准

  • History of another primary malignancy except for
  • A malignancy treated with curative intent and with no known active disease ≥5 years prior to study entry
  • Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease
  • Adequately treated carcinoma in situ without evidence of disease
  • Stable optic pathway glioma or low grade glioma not receiving active therapy
  • History of leptomeningeal carcinomatosis.
  • Patients receiving other anti-cancer agents are not eligible.
  • Patients who cannot swallow whole pills.
  • History of allogeneic organ transplantation.
  • Current or prior use of immunosuppressive medications within 14 days prior to study entry. The following are exceptions to this criterion:

研究组 & 干预措施

Phase 1 and 2 Study of Selumentinib, ZEN-3694 and Durvalumab

Part A will be a phase 1 dose escalation study of the combination with selumetinib and ZEN-3694. Part B will be phase 1 study combining the determined dose of selumetinib and ZEN-3694 from Part A with durvalumab. Part C will be a phase 2 study combining selumetinib, ZEN-3694 with durvalumab in MPNST patients at the recommended doses from part B. A Simon's two-stage design will be used in the phase 2 trial to determine the clinical benefit in patients with unresectable or metastatic NF associated MPNST.

干预措施: Selumetinib + ZEN-3694 ± Durvalumab

结局指标

主要结局

Part A: Safety and Tolerability Selumetinib with ZEN-3694

时间窗: From first dose through the end of the first treatment cycle for each participant at each dose level (up to 28 days).

To determine the safety, tolerability, and recommended doses of selumetinib given in combination with ZEN-3694 in participants with refractory sarcomas including MPNST. The Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of selumetinib in combination with ZEN-3694 will be determined based on dose-limiting toxicities (DLTs) observed during the first cycle of therapy. DLTs are defined as grade ≥3 toxicities attributable to the study drugs, assessed according to CTCAE v5. Dose escalation will proceed in cohorts of 3-6 participants, with possible dose de-escalation if ≥33% of participants experience a DLT. At the RP2D, the cohort may be expanded to up to six additional participants to further evaluate pharmacokinetics and tolerability.

Part B: Safety and Tolerability of Durvalumab with Combination of Selumetinib and ZEN-3694

时间窗: From first dose through the end of the first treatment cycle for each participant at each dose level (up to 28 days).

To determine the safety, tolerability, and recommended doses of durvalumab when given in combination with selumetinib and ZEN-3694 in participants with refractory sarcomas including MPNST. The Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of the combination of selumetinib, ZEN-3694, and durvalumab will be determined based on dose-limiting toxicities (DLTs) observed during the first cycle of therapy. DLTs are defined as grade ≥3 toxicities attributable to the study drugs, assessed according to CTCAE v5. Dose escalation will proceed in cohorts of 3-6 participants, with possible dose de-escalation if ≥33% of participants experience a DLT. At the RP2D, the cohort may be expanded to up to six additional participants to further evaluate pharmacokinetics and tolerability.

Part C: Determine the Clinical Benefit of Selumetinib, ZEN-3694 and Durvalumab

时间窗: From first dose through completion of 4 treatment cycles for each participant (up to 112 days).

Clinical benefit rate defined as radiographic complete response, partial response, or stable disease, greater than or equal to four cycles. The primary endpoint is the clinical benefit rate, defined as the proportion of evaluable participants achieving a complete response (CR), partial response (PR), or stable disease (SD) for at least 4 treatment cycles. A Simon's optimal two-stage phase 2 design will be used to evaluate efficacy in participants with unresectable or metastatic NF1-associated MPNST. In the first stage, 9 participants will be enrolled; the trial will stop early if 0 participants respond. If the study continues to the second stage, a total of 17 participants will be enrolled, and the treatment will be considered ineffective if ≤2 participants respond. The target clinical benefit rate is 30% (p1 = 0.30), and a rate ≤5% (p0 = 0.05) is considered uninteresting.

研究点 (1)

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