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A Phase II Study of AP23573, an mTOR Inhibitor, in Patients With Relapsed or Refractory Hematologic Malignancies

Phase 2
18 Years
Not Enrolling
Hematologic Malignancies, Leukemia, Myelodysplastic Syndromes, Myeloid Metaplasia, Lymphoma

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Trial Information

A Phase II Study of AP23573, an mTOR Inhibitor, in Patients With Relapsed or Refractory Hematologic Malignancies

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.

Inclusion Criteria

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

- 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

- 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

- 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

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess efficacy of AP23573 in patients with relapsed or refractory hematologic malignancies.

Outcome Time Frame:

Duration of study

Safety Issue:


Principal Investigator

Frank Haluska, M.D., Ph.D.

Investigator Role:

Study Director

Investigator Affiliation:

Ariad Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

June 2004

Completion Date:

June 2006

Related Keywords:

  • Hematologic Malignancies
  • Leukemia
  • Myelodysplastic Syndromes
  • Myeloid Metaplasia
  • Lymphoma
  • Relapsed or refractory hematologic malignancies
  • Primary Myelofibrosis
  • Neoplasms
  • Leukemia
  • Lymphoma
  • Metaplasia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms



MD Anderson Cancer Center Houston, Texas  77030-4096
Gabrail Cancer Center Canton, Ohio  44718
The Cancer Institute of New Jersey New Brunswick, New Jersey  08901
University of Chicago Hospitals, Section of Hematology/Oncology Chicago, Illinois  60637
Washington University School of Medicine, Siteman Cancer Center, 4921 Parkview Place St. Louis, Missouri  63110
New York Presbyterian Hospital, Weill College of Cornell University New York, New York  10021
Morris Cancer Center - Duke University Medical Center, Adult Bone Marrow Transplant Clinic Durham, North Carolina  27705
Jeane's Hospital of TUHS Philadelphia, Pennsylvania  19111