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A Multi-Center Phase I Study of Intravenous Deforolimus (AP23573, MK-8669) Administered QDX5 Every Other Week in Pediatric Patients With Advanced Solid Tumors

Phase 1
1 Year
17 Years
Not Enrolling
Solid Tumors

Thank you

Trial Information

A Multi-Center Phase I Study of Intravenous Deforolimus (AP23573, MK-8669) Administered QDX5 Every Other Week in Pediatric Patients With Advanced Solid Tumors

Inclusion Criteria:

- Male or female age 1 to <18 years at the time of study entry for the dose escalation
portion of the study

- Histologic diagnosis of a malignant lymphoma or solid tumor, including tumors of the
central nervous system that has progressed in the opinion of the investigator despite
standard therapy or for which no effective standard therapy is known

- Patients may have measurable or non-measurable disease as defined by RECIST

- Patients with brainstem glioma or intrinsic pontine glioma do not need biopsy proof
of the diagnosis, but must have documentation at their local institution that there
is agreement among the attending oncologist and/or neuro-oncologist, radiologist, and
neurosurgeon/pediatric neurosurgeon that the diagnostic imaging studies are
consistent with a diagnosis of brainstem or intrinsic pontine glioma

- Patients must have fully recovered from the acute toxic effects of all prior
chemotherapy, immunotherapy, surgery or radiotherapy prior to entering this study

- Performance Status: EGOG 0-2 for patients age 16 and older; Karnofsky >40% for
patients >10 years of age; Lansky Play Scale >40 for children < 10 years of age

- Life expectancy greater than or equal to 12 weeks

- There is no limit to the number of prior treatment regimens provided that performance
status, organ function, and life expectancy meet the study criteria

- No persistent toxicities from previous therapies > Grade 2 by NCI CTCAE version 3.
For patients with CNS tumors ONLY, if baseline neurotoxicity due to primary tumor
involvement or post-operative complications, Grade 3 neurotoxicity is allowed if

- Normal organ and marrow function

- For females of childbearing potential, a negative pregnancy test must be documented
prior to enrollment

- Patients who enter this study and their sexual partners who are of childbearing
potential must agree to use an effective form of contraception

Exclusion Criteria:

- Patients who have had chemotherapy or radiotherapy within three (3) weeks (or six
weeks for nitrosoureas or mitomycin C) prior to entering the study, or those who have
not recovered from adverse events due to agents administered more than 4 weeks

- Patients receiving any other investigational agents or using any investigational

- Patients with leukemia

- Patients who have previously received deforolimus or other rapamycin analogs

- History of allergic reactions (in opinion of the investigator) attributed to
compounds of similar chemical or biologic composition to deforolimus and its
excipients used in administration

- Uncontrolled intercurrent illness

- Pregnant women are excluded from this study because the teratogenic or abortifacient
effects of deforolimus are not known at this time

- Because patients with immune deficiency are at increased risk of lethal infections
when treated with marrow-suppressive therapy, known HIV-positive patients are
excluded from the study because of possible pharmacokinetic interactions with

- Autologous or allogeneic stem cell transplant <3 months prior to enrollment; any
evidence of on-going graft versus host disease (GVHD), or GVHD requiring
immunosuppressive therapy. Patients who have had prior stem cell transplant regimens
must be discussed with and approved by the principal investigator prior to

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To establish the DLT and the MTD of ridaforolimus administered daily x 5 every 14 days in pediatric patients with recurrent/refractory solid tumors, including lymphoma and tumors of the central nervous system.

Outcome Time Frame:

Duration of trial

Safety Issue:


Principal Investigator

Lisa Gore, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Colorado, Denver


United States: Food and Drug Administration

Study ID:




Start Date:

January 2008

Completion Date:

June 2010

Related Keywords:

  • Solid Tumors
  • Pediatric
  • Solid Tumor
  • CNS Tumor
  • Lymphoma
  • Neoplasms