NCT02238496

Brief Summary

The purpose of this study is to test the effectiveness of a drug called temsirolimus in combination with a drug called perifosine in treating brain tumors that have continued to grow after previous treatment. Temsirolimus is an intravenous drug approved by the FDA for treatment of other cancers (kidney cancer, certain types of lymphoma) but not for brain tumors. Perifosine is a pill that has not been approved by the FDA which blocks a messenger that tells cancer cells to grow. Research suggests that combined treatment with both drugs is better than either alone, and that it is reasonably safe.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Dec 2014

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 29, 2014

Completed
14 days until next milestone

First Posted

Study publicly available on registry

September 12, 2014

Completed
3 months until next milestone

Study Start

First participant enrolled

December 8, 2014

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 27, 2017

Completed
3.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 14, 2021

Completed
Last Updated

May 25, 2023

Status Verified

May 1, 2023

Enrollment Period

2.9 years

First QC Date

August 29, 2014

Last Update Submit

May 23, 2023

Conditions

Keywords

GlioblastomaAnaplastic AstrocytomaAnaplastic OligodendrogliomaMixed Gliomaakt-Oncogene ProteinmTOR ProteinGliomaBrain NeoplasmsBrain Tumor, Recurrent

Outcome Measures

Primary Outcomes (1)

  • Clinical Benefit Rate

    Clinical Benefit Rate is defined as the radiographic response rate plus 6-month progression-free survival (PFS) rate.

    Up to 6 months from the start of treatment

Secondary Outcomes (1)

  • Median Overall Survival Rate

    Up to 48 months from start of treatment

Study Arms (2)

Surgical Cohort - cytoreductive surgery

OTHER

Cytoreductive surgery planned (surgical cohort). After post-operative standard evaluations, patients will resume therapy. After anti-emetic prophylaxis, patients will receive the first divided dose of the perifosine loading dose after recovery from surgery. Patients will be observed for at least 30 minutes to ensure there has been adequate anti-emetic prophylaxis, and then patients will receive temsirolimus administered over 30-60 minutes IV. The remaining divided doses of the perifosine loading dose will then be administered. Patients will then return weekly for infusion of temsirolimus over 30-60 minutes IV. Dosing will be continuous although for the purposes of evaluation, a cycle will be defined as 4 weeks (28 days).

Procedure: Cytoreductive surgeryDrug: PerifosineDrug: Temsirolimus

Medical Cohort - no cytoreductive surgery

OTHER

No-Cytoreductive surgery planned (medical cohort). After anti-emetic prophylaxis, patients will receive the first divided dose of the perifosine loading dose. Patients will be observed for at least 30 minutes to ensure there has been adequate anti-emetic prophylaxis, and then patients will receive temsirolimus administered over 30-60 minutes IV. The remaining divided doses of the perifosine loading dose will then be administered. Patients will then return weekly for infusion of temsirolimus over 30-60 minutes IV. Dosing will be continuous although for the purposes of evaluation, a cycle will be defined as 4 weeks (28 days).

Drug: PerifosineDrug: Temsirolimus

Interventions

Standard of care/routine cytoreductive glioma resection surgery. Arm B only.

Also known as: Resection
Surgical Cohort - cytoreductive surgery

Perifosine is a pill that has not been approved by the FDA which blocks a messenger that tells cancer cells to grow.

Also known as: AEZS-104/D-21266, KRX-0401
Medical Cohort - no cytoreductive surgerySurgical Cohort - cytoreductive surgery

Temsirolimus is an intravenous drug approved by the FDA for treatment of other cancers (kidney cancer, certain types of lymphoma) but not for brain tumors.

Also known as: Torisel
Medical Cohort - no cytoreductive surgerySurgical Cohort - cytoreductive surgery

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histologically confirmed intracranial glioblastoma (GBM), including sub variants
  • At least 15 unstained slides or at least 1 tissue blocks must be collected from at least one prior surgery.
  • Received prior radiotherapy and prior temozolomide as treatment for the malignant glioma
  • Recovered from toxic effects of prior therapies and at least 2 weeks must have elapsed since any prior signaling pathway modulators; in general, at least 4 weeks must have elapsed from any other anticancer therapy
  • Able to undergo contrast enhanced magnetic resonance imaging (MRI) scans or CT scans
  • Shown unequivocal evidence for contrast enhancing tumor progression by MRI or CT in comparison to a prior scan
  • Age \> or = 18 years
  • Karnofsky Performance Status \> or = 70
  • Life expectancy of \> 8 weeks
  • Normal organ and marrow function, adequate liver function, and adequate renal function before starting therapy
  • Platelet count of at least 100,000/mm3 on at least 2 consecutive blood draws at least 1 week apart with results stable or trending upward
  • Normal coagulation
  • Cholesterol level \< or = 350 mg/dl and triglycerides level \< or = 400 mg/dl
  • Women of child-bearing potential and men must agree to use adequate contraception prior to study entry and for the duration of study participation
  • Women of childbearing potential must have a negative beta-human chorionic gonadotropin (B-hCG) pregnancy test documented within 7 days prior to treatment
  • +10 more criteria

You may not qualify if:

  • There is no limit on the number or type of prior chemotherapies except:
  • convection enhanced delivery, catheter based intra-tumoral treatment, or carmustine (BCNU)/Gliadel® wafers
  • stereotactic radiosurgery, or re-irradiation of any type
  • agent designed to inhibit mTOR or PI3K/AKT
  • direct Vascular Endothelial Growth Factor (VEGF)/Vascular Endothelial Growth Factor Receptors (VEGFR) inhibitors
  • Smoking or plan to smoke tobacco or marijuana during study therapy
  • Plan to eat grapefruit or drink grapefruit juice during study therapy
  • Receiving any other investigational agents concurrently with study treatment
  • Taking hepatic Enzyme Inducing Anti-Epileptic Drug (EIAED)
  • Taking medications that are inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4) for at least two weeks prior to study treatment
  • Uncontrolled intercurrent illness
  • HIV-positive patients on combination antiretroviral therapy
  • Other active concurrent malignancy
  • History of gout which can be exacerbated by perifosine
  • Known history of allergic reactions attributed to compounds of similar chemical or biologic composition to temsirolimus or perifosine
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

MeSH Terms

Conditions

Brain NeoplasmsGlioblastomaAstrocytomaOligodendrogliomaGlioma

Interventions

Cytoreduction Surgical Proceduresperifosinetemsirolimus

Condition Hierarchy (Ancestors)

Central Nervous System NeoplasmsNervous System NeoplasmsNeoplasms by SiteNeoplasmsBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Surgical Procedures, Operative

Study Officials

  • Andrew B. Lassman, MD

    Columbia University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Masking Details
No Masking
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
John Harris Associate Professor of Neurology

Study Record Dates

First Submitted

August 29, 2014

First Posted

September 12, 2014

Study Start

December 8, 2014

Primary Completion

October 27, 2017

Study Completion

February 14, 2021

Last Updated

May 25, 2023

Record last verified: 2023-05

Data Sharing

IPD Sharing
Will not share

Locations