CompletedNCT00590954Updated Oct 19, 2020Results posted

Clinical and Molecular-Metabolic Phase II Trial of Perifosine for Recurrent/Progressive Malignant Gliomas

A Phase 2 interventional study of Perifosine in Malignant Gliomas, CNS and Brain Cancer, sponsored by Memorial Sloan Kettering Cancer Center. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-10-19.

Sponsored by Memorial Sloan Kettering Cancer Center · Phase 2, Interventional, and Treatment

From the registry’s dates

Registered 1 year 7 months after the study started (first participant enrolled May 2006, registered Dec 2007).

Phase: Phase 2
Study type: Interventional
Enrollment: 32
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

The purpose of this study is to test the effectiveness of perifosine in preventing further tumor growth using the established optimal dose of the drug. A second goal is to determine if perifosine can block the molecules in the tumor that drive it to divide and grow.

Read the detailed description

This is a phase II study of the small molecule inhibitor perifosine (NSC 639966, D21266, KRX-0401) in the treatment of patients with recurrent glioblastoma multiforme (GBM) and other recurrent malignant gliomas. The goal of the phase II study is to determine efficacy as measured by the progressionfree survival rate after 6 months of treatment. Secondary goals include determination of molecular and metabolic effects of perifosine by tissue analysis and PET imaging.

In addition, when cytoreductive surgery is recommended as part of the standard of care at study entry, patients will be considered for a "surgical arm." In this case, patients will receive perifosine for 5-10 days before surgery during which tumor will be aliquoted both for diagnostic purposes and for molecular effects of the drug in vivo and for analysis of drug penetration into tumor tissue.

Conditions studied

Malignant Gliomas
CNS
Brain Cancer
Cancer

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Keywords

Malignant Gliomas
CNS
Brain Cancer
Cancer
Perifosine

In context

Glioma

1,397 studies on the registry are indexed under Glioma; 351 are open to participants now.

This study's enrollment of 32 is close to the median of 32 across 1,065 interventional studies indexed under Glioma.

Browse Glioma studies →

Lead sponsor

Memorial Sloan Kettering Cancer Center is the lead sponsor of 1,930 studies on the registry; 328 are open to participants now.

Of its 129 completed or terminated interventional studies of FDA-regulated products, 66 (51%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Patients must have shown unequivocal evidence for tumor progression by MRI or CT scan.
Patients must be on a stable or decreasing dose of corticosteroids for a minimum of 5 days before the baseline MRI and PET scans.
Patients must have failed prior radiation therapy.
Patients with prior therapy that included interstitial brachytherapy or stereotactic radiosurgery (including gamma-knife or cyber-knife) must have confirmation of true progressive disease rather than radiation necrosis based upon either PET or Thallium scanning, and/or MR spectroscopy, and/or MR Perfusion, and/or surgical documentation of disease.
All patients must sign an informed consent indicating that they are aware of the investigational nature of this study. Patients must have signed an authorization for the release of their protected health information.
Age > 18 years old, and with a life expectancy > 8 weeks.
Karnofsky Performance Status ≥ 50%
Patients must have recovered from all acute toxicities from prior therapies. At least 28 days must have elapsed since prior radiation.
Patients must have adequate bone marrow function
Patients must agree to practice adequate contraception.

Exclusion criteria

Exclusion Criteria:

Patients must not be taking EIAEDs
Patients must not have any significant medical illnesses or other history that in the investigator's opinion cannot be adequately controlled with appropriate therapy or would compromise the patient's ability to tolerate this therapy.
Patients with a history of any other cancer (except non-melanoma skin cancer or carcinoma in-situ of the cervix), unless in complete remission and off of all therapy for that disease for a minimum of 3 years are ineligible.
Patients must not have active infection or serious intercurrent medical illness.
HIV-Positive patients receiving combination anti-retroviral therapy are excluded from the study due to possible retro-viral drug interactions.
Patients must not have any disease that will obscure toxicity or dangerously alter drug metabolism.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 32 participants (actual)

Study arms

Experimental
Treatment
Following a diagnosis of tumor recurrence or progression, all patients will receive perifosine monotherapy until toxicity, progression, or death.
Drug: Perifosine

Interventions

DrugPerifosine
Dosing will be continuous, and for the purpose of this trial a cycle will be defined as 28 days. Perifosine will be given as a 600 mg loading dose on day 1. The loading dose will be divided into 4 equal doses of 150 mg each. The first 3 doses should be given with food in the adult day hospital to allow intravenous antiemetic prophylaxis, and 4th dose at bedtime at home. The interval between doses of perifosine should be no less than 4 hours. On day 2, patients will start the maintenance dose of 100 mg daily at bedtime at home. In addition to baseline serum, all patients will have weekly serum drawn during weeks 2-4.
Also known as: NSC 639966, D21266, KRX-0401

What researchers measure

Primary outcomes

Determine the Efficacy of Perifosine in Patients With Recurrent/Progressive GBMs Not Taking EIAEDs as Measured by 6 Month Progression Free Survival/PFS.
Time frame: 6 months

Secondary outcomes

Determine Metabolic Effects of Perifosine on Malignant Gliomas by PET Imaging
Time frame: 2 years

Results

Posted Oct 19, 2020

Participant flow

Participant flow — Overall Study
Milestone	Treatment
Started	32
Completed	30
Not completed	2
Withdrew: Inevaluable	2

Outcome measures

PrimaryDetermine the Efficacy of Perifosine in Patients With Recurrent/Progressive GBMs Not Taking EIAEDs as Measured by 6 Month Progression Free Survival/PFS.

Time frame:: 6 months
Reported as:: Median · months

Determine the Efficacy of Perifosine in Patients With Recurrent/Progressive GBMs Not Taking EIAEDs as Measured by 6 Month Progression Free Survival/PFS.
months	Glioblastoma	Anaplastic Glioma
Determine the Efficacy of Perifosine in Patients With Recurrent/Progressive GBMs Not Taking EIAEDs as Measured by 6 Month Progression Free Survival/PFS.	1.58 (1.08 to 1.84)	2.12 (1.84 to 12.79)

SecondaryDetermine Metabolic Effects of Perifosine on Malignant Gliomas by PET Imaging

Time frame:: 2 years

No measurements were reported for this outcome.

Adverse events

Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Treatment	30/32 (93.8%)	22/32 (68.8%)	32/32 (100%)

Most frequent serious events

Showing 10 of 30

Most frequent serious events
Event	Treatment
SeizureNervous system disorders	13/32
Extremity-lower (gait/walking)General disorders	4/32
Muscle weakness - Left-sidedMusculoskeletal and connective tissue disorders	4/32
Death not assoc w CTCAE term-Disease prog NOSGeneral disorders	3/32
Muscle weakness - Whole body/generalMusculoskeletal and connective tissue disorders	3/32
Neurology - Other (specify)Nervous system disorders	3/32
Somnolence/dprssd level of consciousNervous system disorders	3/32
Speech impairmentNervous system disorders	3/32
ConfusionPsychiatric disorders	2/32
Secondary malig-poss related to ca txt specifyNeoplasms benign, malignant and unspecified (incl cysts and polyps)	2/32

Most frequent other events

Showing 10 of 18

Most frequent other events
Event	Treatment
HyperglycemiaMetabolism and nutrition disorders	16/32
Increased alanine aminotransferaseInvestigations	13/32
FatigueGeneral disorders	10/32
ThrombocytopeniaInvestigations	9/32
DiarrheaGastrointestinal disorders	7/32
Elevated aspartate transaminase (AST)Investigations	6/32
HypophosphatemiaMetabolism and nutrition disorders	5/32
LeukopeniaInvestigations	5/32
NauseaGastrointestinal disorders	5/32
LymphopeniaInvestigations	3/32

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Treatment
Median	51 (23 to 78)

Sex: Female, Male
Sex: Female, Male(Participants)	Treatment
Female	15
Male	17

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Treatment
Hispanic or Latino	2
Not Hispanic or Latino	30
Unknown or Not Reported	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Treatment
American Indian or Alaska Native	0
Asian	1
Native Hawaiian or Other Pacific Islander	0
Black or African American	1
White	30
More than one race	0
Unknown or Not Reported	0

Region of Enrollment
Region of Enrollment(Participants)	Treatment
United States	32

Study locations

1 site

Memorial Sloan-Kettering Cancer Center
New York, New York 10065, United States

References and documents

Study documents

Protocol and statistical analysis plan · Oct 25, 2011

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Oct 19, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT00590954
Lead sponsor: Memorial Sloan Kettering Cancer Center
Collaborators: Keryx Biopharmaceuticals, Keryx / AOI Pharmaceuticals, Inc., Online Collaborative Oncology Group, University of Wisconsin, Madison, Columbia University
Responsible party: Sponsor
First posted: Jan 11, 2008
Start date: May 2006
Primary completion: Nov 11, 2019
Completion: Nov 11, 2019
Results posted: Oct 19, 2020
Last update: Oct 19, 2020

Study contacts

Thomas Kaley, MD

principal investigator · Memorial Sloan Kettering Cancer Center

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Nov 2019. You cannot join it, but the record below documents what was studied.

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