Study of AP23573 in Patients With Relapsed or Refractory Hematologic Malignancies (8669-024)(COMPLETED)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00086125
Recruitment Status : Completed
First Posted : June 28, 2004
Last Update Posted : August 19, 2015
Ariad Pharmaceuticals
Information provided by (Responsible Party):
Merck Sharp & Dohme Corp.

Brief Summary:
The purpose of this phase II study is to assess the efficacy of AP23573 in patients with specified relapsed or refractory hematological malignancies.

Condition or disease Intervention/treatment Phase
Hematologic Malignancies Leukemia Myelodysplastic Syndromes Myeloid Metaplasia Lymphoma Drug: ridaforolimus Phase 2

Detailed Description:

The primary objective of the trial is to assess the efficacy of AP23573 in patients with specified relapsed or refractory hematological malignancies by evaluating the response rates as defined for the individual malignancy categories. Secondary objectives include evaluating time to disease progression, progression-free survival and duration of response; evaluating the pharmacokinetic and pharmacodynamic characteristics of AP23573; describing health-related quality of life measurements; and exploring the safety and tolerability of AP23573 at the specified dose level.

Protocol Outline: Open label, non-randomized parallel cohorts of five disease-specific cohorts with a minimum of 21 patients per cohort. Each patient receives a fixed dose of AP23573 administered intravenously (IV) over 30 minutes daily for five days (QDx5) to be repeated every 2 weeks.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 57 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of AP23573, an mTOR Inhibitor, in Patients With Relapsed or Refractory Hematologic Malignancies
Study Start Date : June 2004
Primary Completion Date : June 2006
Study Completion Date : June 2006

Arm Intervention/treatment
Experimental: 1
AP23573 12.5 mg IV as monotherapy once daily for 5 days, every 2 weeks
Drug: ridaforolimus
AP23573 12.5 mg IV as monotherapy once daily for 5 days, every 2 weeks
Other Names:
  • deforolimus
  • AP23573
  • MK-8669
  • ridaforolimus was also known as deforolimus until May 2009

Primary Outcome Measures :
  1. To assess efficacy of AP23573 in patients with relapsed or refractory hematologic malignancies. [ Time Frame: Duration of study ]

Secondary Outcome Measures :
  1. Evaluating time to progression, progression-free survival, duration of response and safety [ Time Frame: Duration of study ]

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria (Patients must meet each of the following criteria to be eligible for participation in the trial):

  • Male or female patients ≥ 18 years of age
  • Patients must have histologically-confirmed diagnoses of relapsed or refractory hematologic malignancy as specified in the protocol
  • Patients must have an ECOG performance status of 0 to 2
  • Patients must have adequate renal and liver function as demonstrated by laboratory values performed within 5 days, inclusive, prior to administration of the first dose of AP23573
  • Patients must be able to understand and give written informed consent

Exclusion Criteria (Patients meeting any of the following criteria are ineligible for participation in the study):

  • Women who are pregnant or lactating
  • Patients may not have had cytotoxic chemotherapy or radiotherapy within 14 days prior to study entry
  • Patients may not receive any investigational anti-cancer agent while on this study or within 14 days prior to the first dose of AP23573
  • Patients with known or suspected hypersensitivity to drugs formulated with polysorbate 80 (Tween) or any other excipient contained in the study drug formulation
  • Patients with known Grade 3 or 4 hypersensitivity to macrolide antibiotics (e.g., clarithromycin, erythromycin, azithromycin)
  • Patients with significant uncontrolled cardiovascular disease
  • Patients with known HIV infection
  • Patients with any uncontrolled infection
  • Patients receiving immunosuppressive agents other than prescribed corticosteroids
  • Patients who have had prior therapy with rapamycin, any rapamycin analog or tacrolimus
  • Patients with inadequate recovery from any prior surgical procedure or patients having undergone any major surgical procedure within 14 days prior to the first dose of AP23573
  • Patients with any other life-threatening illness or organ system dysfunction which, in the opinion of the Investigator, would either compromise the patient's safety or interfere with evaluation of the safety of the study drug
  • Patients with a psychiatric disorder or altered mental status that would preclude understanding of the informed consent process and/or completion of the necessary studies
  • Patients with another primary malignancy within the past three years (except for non-melanoma skin cancer and cervical carcinomas in situ)
  • Patients with the inability, in the opinion of the Investigator, to comply with the protocol requirements

Drugs and Other Treatments to be Excluded (The following drugs and other treatments are not permitted for patients enrolling in the study, either during or within two weeks prior to the first dose of AP23573, unless otherwise specified):

  • Chemotherapeutic agents with the following exception that therapy with nitrosoureas (including generics) cannot be given within six weeks prior to the first dose of AP23573
  • Other antineoplastic agents
  • Immunotherapy (including vaccines) or biological response modifier therapy
  • Systemic hormonal therapy with exceptions as specified in the protocol
  • Herbal preparations or related OTC preparations containing herbal ingredients (e.g., St John's Wort)
  • Radiotherapy for the primary malignancy
  • Any other investigational agent during the course of the trial should be discussed with the Sponsor prior to use

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00086125

United States, Illinois
University of Chicago Hospitals, Section of Hematology/Oncology
Chicago, Illinois, United States, 60637
United States, Missouri
Washington University School of Medicine, Siteman Cancer Center, 4921 Parkview Place
St. Louis, Missouri, United States, 63110
United States, New Jersey
The Cancer Institute of New Jersey
New Brunswick, New Jersey, United States, 08903
United States, New York
New York Presbyterian Hospital, Weill College of Cornell University
New York, New York, United States, 10021
United States, North Carolina
Morris Cancer Center - Duke University Medical Center, Adult Bone Marrow Transplant Clinic
Durham, North Carolina, United States, 27705
United States, Ohio
Gabrail Cancer Center
Canton, Ohio, United States, 44718
United States, Pennsylvania
Jeane's Hospital of TUHS
Philadelphia, Pennsylvania, United States, 19111
United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Merck Sharp & Dohme Corp.
Ariad Pharmaceuticals
Study Director: Frank Haluska, M.D., Ph.D. Ariad Pharmaceuticals

Publications of Results:
Responsible Party: Merck Sharp & Dohme Corp. Identifier: NCT00086125     History of Changes
Other Study ID Numbers: 8669-024
First Posted: June 28, 2004    Key Record Dates
Last Update Posted: August 19, 2015
Last Verified: August 2015

Keywords provided by Merck Sharp & Dohme Corp.:
Relapsed or refractory hematologic malignancies

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Primary Myelofibrosis
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Pathologic Processes
Myeloproliferative Disorders
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs