Perifosine With Temsirolimus for Recurrent Pediatric Solid Tumors
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|ClinicalTrials.gov Identifier: NCT01049841|
Recruitment Status : Completed
First Posted : January 15, 2010
Last Update Posted : March 10, 2017
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|Condition or disease||Intervention/treatment||Phase|
|Pediatric Solid Tumors||Drug: perifosine + temsirolimus||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||23 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study of Perifosine With Temsirolimus for Recurrent Pediatric Solid Tumors|
|Actual Study Start Date :||January 2010|
|Actual Primary Completion Date :||March 2017|
|Actual Study Completion Date :||March 2017|
Experimental: perifosine + temsirolimus
This is a single arm, phase I study. Eligible patients will receive a loading dose of oral perifosine on the first day, followed by a maintenance dose starting on the second day until progression. Each patient is assigned to a group according to their body surface area (BSA). Temsirolimus will be combined with perifosine at four dose levels to determine the MTD for the combination therapy. Temsirolimus dosing will start on the same day as the perifosine load.
Drug: perifosine + temsirolimus
The patient will take oral tablets of perifosine at a dose and frequency to be determined their height, weight and time when they enter the study as those entering early will be treated with lower dose levels (dosing frequency will vary from once a week to daily). The patient will receive an intravenous injection of temsirolimus once a week at a dose to be determined by their height, weight and time when they enter the study as those entering early will be treated with lower doses. In addition they will be asked to keep a medicine diary.
- Maximum Tolerated Dose (MTD) of perifosine + temsirolimus combination in children with cancer. [ Time Frame: 2 years ]
- To determine whether pharmacokinetic serum levels of both perifosine and temsirolimus correlate with toxicity. [ Time Frame: 2 years ]
- To record preliminary data on the efficacy of the perifosine + temsirolimus combination. [ Time Frame: 2 years ]
- If previously resected tissue is available, determine whether molecular features predict response including, Elevated PI3K/AKT/mTOR signaling, Elevated RAS/MAPK signaling, Cell cycle markers [ Time Frame: 2 years ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||up to 21 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Any solid tumor that has failed standard therapy
- Patient must have evidence of tumor by CT, MRI, MIBG scan, serum markers, or tissue sampling.
- Age ≤ 21 years (age ≤ 35 years for biopsy proven medulloblastoma or neuroblastoma)
- Karnofsky/Lansky performance status ≥ 50% (Karnofsky score for age> 16 years and Lansky score for age ≤ 16 years)
- ANC≥ 1000 at least 24 hours off GCSF
- Platelets ≥ 100K at least one week off platelet transfusions
- Hg≥ 8g/dL at least one week off PRBC transfusion
- AST ≤ 2 x the upper limit of normal
- ALT ≤ 2 x the upper limit of normal
- Total bilirubin ≤ 2.0 mg/dl
- Patients must have cholesterol level < 350 mg/dl and triglycerides level < 400 mg/dl because temsirolimus can induce hyperlipidemia.
- Serum creatinine ≤ 1.5 x the upper limit of normal for age, or calculated creatinine clearance or nuclear GFR ≥ 70 ml/min/1.73 m2.
- ≥ 3 weeks since last non-nitrosourea chemotherapy
- ≥ 6 weeks since last nitrosoureas
- ≥ 4 weeks since last RT
- Patients must agree to practice adequate contraception. Females of childbearing potential must have a negative serum B-HCG pregnancy test documented within 14 days prior to registration. Females must not be breast feeding.
- Patients must be able to swallow tablets whole
- Patients that participated in the phase I single agent perifosine study for recurrent pediatric solid tumors and did not experience a DLT are eligible to participate in this study and can start ≥ 2 weeks since last dose of perifosine
- Patients that have been previously treated with an mTOR inhibitor can still enroll in this trial as long as they did not experience a DLT in the single agent mTOR inhibitor trial
- Patients must not have an uncontrolled active infection.
- HIV-Positive patients receiving combination anti-retroviral therapy are excluded from the study due to possible retro-viral drug interactions. HIV testing not required.
- Patients must not be taking EIAEDs. If patients were previously on EIAEDs that have been discontinued, patients must have been off the agent for at least 2 weeks prior to registration.
- History of or known pulmonary hypertension or history of or known pneumonitis.
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 ClinicalTrials.gov identifier (NCT number): NCT01049841
|United States, New York|
|Memorial Sloan-Kettering Cancer Center|
|New York, New York, United States, 10065|
|Principal Investigator:||Ira Dunkel, MD||Memorial Sloan Kettering Cancer Center|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Memorial Sloan Kettering Cancer Center|
|Other Study ID Numbers:||
|First Posted:||January 15, 2010 Key Record Dates|
|Last Update Posted:||March 10, 2017|
|Last Verified:||March 2017|
Physiological Effects of Drugs