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Study Summary
No. 2008-0052:.......Brain; Lymphoma; Pediatrics; Phase I Studies; Solid Tumors......Cynthia E. Herzog......Pediatrics
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Study Summary Title
Study Summary
Number:
2008-0052
Study Title:A Multi-Center Phase 1 Study of Intravenous Deforolimus (AP23573, MK-8669) Administered Daily for Five Days Every Other Week in Pediatric Patients with Advanced Solid Tumors
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Physician New Patient Referral
Name:Cynthia E. HerzogPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:PediatricsReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-6620
Contact us about clinical trials
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General Information
Disease Group:Brain
Lymphoma
Pediatrics
Phase I Studies
Solid Tumors
Supported By:ARIAD Pharmaceuticals, Inc.
H. Lee Moffitt Cancer Center
Phase of Study:Phase IReturn
Visit:
On Days 1-5 of each 14-day course of treatment.
Treatment
Agents:
AP23573
Deforolimus
MK-8669
Home Care:None.
Treatment Loc:Both at MDACC & outside MDACC at one or more Collaborating Sites or Institutions
Estimated
Length of Stay
in Houston:
Hospitalization is not anticipated.
Description/
Intervention:
The goal of this clinical research study is to find the highest tolerable dose
of deforolimus that can be given to children with lymphoma or solid tumors
whose tumors have come back or did not go away after earlier treatment(s). The
safety of this drug will also be studied.
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Study Objectives / Outcomes
Primary Objective

The primary objective of this study is to determine the safety, tolerability and maximum
tolerated dose of deforolimus (AP23573) in pediatric patients with advanced lymphomas and solid
tumors, including tumors of the central nervous system.

Secondary Objectives are:
  • To determine the pharmacokinetic and pharmacodynamic properties of deforolimus in pediatric patients with recurrent/refractory solid tumors, including tumors of the central nervous system.
  • To further evaluate the safety and preliminary efficacy data of deforolimus when administered daily x 5 every 14 days in an expanded cohort at the recommended phase 2 dose.
  • To assess pharmacogenomic parameters that may correlate with response to deforolimus
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Study Status Information
Study Activation / Registration Date:01/22/2009
IRB Review and Approval Date:05/29/2008
Study Type:Phase I
Recruitment Status:Terminated
Projected Accrual:36
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Male or female age 1 to < 18 years at the time of study entry for the dose escalation portion of the study, and age 1 and less than 18 years for enrollment on the expanded cohort phase.

2) 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.

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

4) 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. Documentation of the conferring professionals must be provided along with the eligibility sheets at patient registration.

5) Patients must have fully recovered from the acute toxic effects of myelosuppressive chemotherapy: Three (3) weeks must have elapsed since the administration of previous chemotherapy. Six (6) weeks must have elapsed since administration of nitrosoureas or mitomycin C.

6) Patients must have fully recovered from the acute toxic effects of biologic agents: At least 14 days must have elapsed since the completion of therapy with a biologic agent such as a monoclonal antibody. Seven days must have elapsed since the last dose of retinoids.

7) Patients must have fully recovered from the acute toxic effects of radiation therapy (XRT): > 4 weeks must have elapsed for local XRT (small port); > 6 months must have elapsed if prior craniospinal irradiation or irradiation to > 50% of the pelvis or other substantial bone marrow irradiation, including total body irradiation.

8) Patients must have fully recovered from the acute toxic effects of surgery: > 4 weeks must have elapsed from any major surgical procedure. Simple surgical procedures including biopsy, central line placement or similar are allowed less than 4 weeks of study entry if the patient has recovered to baseline.

9) Patients must have fully recovered from the acute toxic effects of stem cell transplant (SCT): Patients who have undergone prior stem cell transplantation will not be excluded from study entry as long as adequate marrow reserve is demonstrated (refer to hematologic parameters). At least 3 months must have elapsed since autologous or allogeneic stem cell transplantation. Patients must be off of all immunosuppressive therapy and have no evidence of active graft versus host disease.

10) Patients must have fully recovered from the acute toxic effects of Growth Factors: Seven (7) days must have elapsed since the administration of conventional G-CSF and/or GM-CSF. Fourteen (14) days must have elapsed since the administration of PEGylated GCSF (Neulasta).

11) Patients must have fully recovered from the acute toxic effects of steroids: Patients requiring steroids should be on a stable or decreasing dose for > 7 days prior to study entry.

12) 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.

13) Life expectancy greater than or equal to 12 weeks.

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

15) 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 stable.

16) Adequate Bone Marrow function defined as: 1) Peripheral absolute neutrophil (ANC) > 1000/uL; 2) Platelet count > 75,000/uL (transfusion-independent); 3) Hemoglobin > 8.5 gm/dL (transfusion permitted).

17) Adequate Renal Function defined as: 1) Serum creatinine < 1.5x upper limit of normal (ULN) for age; 2) If the serum creatinine is above these values, the calculated creatinine clearance or radioisotope GFR must be> 60 mL/min/1.73m^2.

18) Adequate Hepatic Function defined as: 1) Total bilirubin < 1.5 mg/dL; 2) ALT and AST < 2.5x (< 5x if liver involvement with primary tumor) ULN for institution; 3) Prothrombin time < 1.5x ULN.

19) Cholesterol < 350 mg/dL and Triglyceride Levels less than 400 mg/dL.

20) Adequate Cardiac Function defined as Ejection Fraction (EF) > 50% or SF > 28% by any method of the investigator's choice.

21) Adequate Pulmonary Function defined as: 1) No history of pulmonary fibrosis; 2) Room air oxygen saturation of > 90% at altitude > 5000 feet, or > 93% at altitude < 5000 feet; 3) Patients who have received prior therapy with bleomycin and are able to comply with pulmonary function testing must have a DLCO (corrected for hemoglobin) of > 50% predicted values appropriate for the patient's age, gender, and ethnicity within one month of study entry.

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

23) Patients who enter this study and their sexual partners who are of childbearing potential must agree to use an acceptable form of contraception that is agreed upon by the patient and their physician throughout participation in this study.

Exclusion Criteria:1) 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 earlier.

2) Patients receiving any other investigational agents or using any investigational devices

3) Patients with leukemia.

4) Patients who have previously received deforolimus or other rapamycin analogs.

5) 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.

6) Uncontrolled intercurrent illness including, but not limited to the following: 1) Ongoing or active infection; 2) Uncontrolled seizure disorder; 3) Symptomatic congestive heart failure (patients with congestive heart failure who are well-controlled on medication may be enrolled); 4) cardiac arrhythmia; 5) psychiatric or social circumstances that would limit compliance with study requirements.

7) Pregnant women are excluded from this study because the teratogenic or abortifacient effects of deforolimus are not known at this time. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with deforolimus, breastfeeding should be discontinued if the mother is treated with deforolimus. These potential risks may also apply to other agents used in this study.

8) 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 deforolimus.

9) 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 registration.

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Links
Registration Number: NCT00704054
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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