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Study of 72-Hour Infusion of ON 01910.Na in Patients With MDS or AML

2017年6月22日 更新者:Onconova Therapeutics, Inc.

A Phase 1/2, Single-Arm Study To Assess The Efficacy and Safety Of 72-Hour Continuous Intravenous Dosing Of ON 01910.Na Administered Every Other Week in Patients With Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML)

The primary objective of this study is to evaluate the efficacy and safety of ON 01910.Na Concentrate when it is administered as an intravenous continuous infusion (IVCI) over 72 hours once every 2 weeks in a broad population of MDS patients. Rationale for this study is based on the activity observed in another study with ON 01910.Na in patients with refractory anemia with excess blasts (RAEB) 1 and 2 MDS. This study will examine ON 01910.Na in a broader population of MDS and AML patients. This phase I/II study will establish the Maximum Tolerated Dose (MTD) starting with a dose of 800 mg/m2 per day administered over 24 hours for 2 consecutive days as a continuous intravenous infusion, once a week for 3 weeks of a 4-week cycle and examine the efficacy and safety profile at the MTD.

調査の概要

詳細な説明

The primary objective of this study is to evaluate the efficacy and safety of ON 01910.Na CIV 24-hour infusion administered three times a week every other week in achieving by week 25 a complete or partial response as defined per the 2000 International Working Group (IWG) Criteria in patients with MDS or as defined by Cheson et al. [JCO 21:4642 (2003)] in patients with AML.

The secondary objectives are to assess:

  • Time and duration of response
  • Blastic response in bone marrow
  • Improvement of dyspoiesis as evaluated by the change in aneuploidy in bone marrow
  • Change in International Prognostic Scoring System (IPSS) in MDS patients
  • Major and minor responses in absolute neutrophil count, according to IWG 2000 criteria
  • Major and minor responses in platelet count, according to IWG 2000 criteria
  • Major and minor erythroid response, according to IWG 2000 criteria
  • Time to progression
  • Overall survival at 25 and 50 weeks
  • Proportion of MDS patients transitioning to acute myeloid leukemia (AML) at 25 and 50 weeks

This is a phase 1/2 single arm study in which six to thirty-five patients with MDS or AML who meet all other inclusion/exclusion criteria will receive ON 01910.Na as an intravenous continuous infusion (IVCI) over 72 hours once a week every other week.

In the phase 1 portion of the study, a traditional dose escalation rule, also known as the "3+3" rule, will be used. Three patients will be treated at the 800 mg/m2/day dose level. If none of the patients experience a DLT during cycle 1, the next group of 3 patients will receive 1500 mg/m2/day. If no DLT is seen at the 1500 mg/m2/day dose level, then the dose used in the phase 2 portion of the study will be 1500 mg/m2/day. If there is DLT in one of the first three patients at the 800 mg/m2/day dose level, this dose level will be expanded to 6 patients. If ≤ 1 patient out of 6 experience DLT, then the dose will be escalated to the 1500 mg/m2/day dose level. If ≥ 2 patients experience DLT at the 800 mg/m2/day dose level, a full safety review will determine if further enrollment of patients will proceed. If there is DLT in one of the first three patients at the 1500 mg/m2/day dose level, this dose level will be expanded to 6 patients. If ≤ 1 patient out of 6 experience DLT at the 1500 mg/m2/day dose level, then 1500 mg/m2/day dose level will be considered the MTD. If ≥ 2 patients experience DLT at the 1500 mg/m2/day dose level, then 800 mg/m2/day will be designated as the MTD.

The total study duration is 29 weeks, which includes a 2-week screening phase, a 23-week dosing phase, and a 4-week follow-up phase that begins after the last dose of ON 01910.Na. Beginning at week 4, and every 2 weeks thereafter, patients will be assessed for response and followed up.

Patients who achieve by week 25 a complete or partial response or stabilization of their disease are eligible to receive an additional 24 weeks of ON 01910.Na 1800 mg/24 h for 72 h once a week every 2 weeks and will be followed up.

研究の種類

介入

入学 (実際)

36

段階

  • フェーズ2
  • フェーズ 1

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

    • New York
      • New York、New York、アメリカ、10032
        • Columbia University Medical Center

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

18年歳以上 (大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

Inclusion Criteria:

  • > 18 years
  • Diagnosis of MDS or AML via bone marrow aspirate and biopsy performed within 8 weeks prior to study entry according to WHO Criteria and FAB Classification. AML patients must be:

not eligible for standard chemotherapy, including newly diagnosed patients over 70 years; with relapsed or refractory AML; and, with AML secondary to prior cancer chemotherapy or evolving from a myeloproliferative/myelodysplastic syndrome.

  • Anemia requiring transfusion support with at least one unit of packed red blood cells per month for greater than or equal to 2 months or Hemoglobin < 10 g/dL OR Thrombocytopenia (platelet count < 100,000/µl) OR Neutropenia (absolute neutrophil count < 1,500/µl)
  • Failed to respond to, relapsed following, or opted not to participate in bone marrow transplantation
  • Off all other treatments for MDS or AML (including filgrastim (G-CSF) and erythropoietin) for at least four weeks. As an exception, filgrastim (G-CSF) can be used before, during and after the protocol treatment for patients with documented febrile neutropenia (<500/µl)
  • ECOG Performance Status 0, 1 or 2
  • Adequate contraceptive [including prescription oral contraceptives (birth control pills), contraceptive injections, intrauterine device (IUD), double-barrier method (spermicidal jelly or foam with condoms or diaphragm), contraceptive patch, or surgical sterilization] before entry and throughout the study for female patients of reproductive potential
  • Female patient with reproductive potential must have a negative serum beta-HCG pregnancy test at screening
  • Willing to adhere to the prohibitions and restrictions specified in this protocol
  • Patient (or his/her legally authorized representative) must have signed an informed consent document indicating that he/she understands the purpose of and procedures required for the study and is willing to participate in the study

Exclusion Criteria:

  • Anemia due to factors other than MDS or AML (including hemolysis or gastrointestinal bleeding)
  • Proliferative (WBC ≥ 12,000/mm3) chronic myelomonocytic leukemia
  • Any active malignancy within the past year except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast
  • History of HIV-1 seropositivity
  • Uncontrolled intercurrent illness including, but not limited to symptomatic congestive heart failure, unstable angina pectoris or cardiac arrhythmia
  • Active infection not adequately responding to appropriate therapy.
  • Total bilirubin > 1.5 mg/dL not related to hemolysis or Gilbert's disease, ALT or AST > 2 X ULN
  • Serum creatinine > 1.5 mg/dL or calculated creatinine clearance < 60 ml/min/1.73 m2
  • Ascites requiring active medical management including paracentesis, or hyponatremia (defined as serum sodium value of <134 Meq/L).
  • Women patients who are pregnant or lactating
  • Male patients with female sexual partners who are unwilling to follow the strict contraception requirements described in this protocol
  • Major surgery without full recovery or major surgery within 3 weeks of ON 01910.Na treatment start.
  • Uncontrolled hypertension (defined as a systolic pressure ≥ 160 and/or a diastolic pressure ≥ 110)
  • New onset seizures (within 3 months prior to the first dose of ON 01910.Na) or poorly controlled seizures
  • Any concurrent investigational agent or chemotherapy, radiotherapy or immunotherapy
  • Psychiatric illness/social situations that would limit the patient's ability to tolerate and/or comply with study requirements

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:なし
  • 介入モデル:単一グループの割り当て
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
実験的:ON 01910.Na
1800 mg/day of ON 01910.Na administered as a 24-hour continuous intravenous infusion on days 1, 2 and 3 of 14-day cycle.
1800 mg/day as a 24-hour continuous intravenous infusion administered three times a week every 2 weeks
他の名前:
  • リゴセルチブ
  • ON 01910

この研究は何を測定していますか?

主要な結果の測定

結果測定
時間枠
Response as defined per the 2000 International Working Group (IWG) Criteria in patients with MDS.
時間枠:6 -12 months
6 -12 months

二次結果の測定

結果測定
時間枠
Hematologic improvement.
時間枠:6 - 12 months
6 - 12 months

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

捜査官

  • 主任研究者:Azra Raza, MD、Columbia University

出版物と役立つリンク

研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始

2009年1月1日

一次修了 (実際)

2013年9月1日

研究の完了 (実際)

2014年2月1日

試験登録日

最初に提出

2009年3月2日

QC基準を満たした最初の提出物

2009年3月2日

最初の投稿 (見積もり)

2009年3月3日

学習記録の更新

投稿された最後の更新 (実際)

2017年6月26日

QC基準を満たした最後の更新が送信されました

2017年6月22日

最終確認日

2017年6月1日

詳しくは

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