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A Trial of Perifosine in Patients With Chemo-Insensitive Sarcomas

Primary Purpose

Chondrosarcomas, Alveolar Soft Part Sarcomas, Extra Skeletal Myxoid Chondrosarcomas

Status

Completed

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

Perifosine

Loperamide

Allopurinol

Antiemetics

About this trial

This is an interventional treatment trial for Chondrosarcomas focused on measuring Chemo Insensitive Sarcomas, Chondrosarcomas, Alveolar soft part sarcomas, Extra skeletal myxoid chondrosarcomas, Perifosine

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients must have histologically or cytologically confirmed diagnosis of chondrosarcoma, extra-skeletal myxoid chondrosarcoma or alveolar soft part sarcoma.
Patients may have had prior chemotherapy, but if the patient has had three or more forms of prior chemotherapy for metastases, the patient's clinical course should be discussed with the study chairman before the patient is enrolled on study
Patients must have progression of disease by Choi criteria.
ECOG performance status 0-1. Patients with ECOG PS of 2 may be admitted with approval from the study chairman.
At least 13 years of age.
Patients must have measurable disease.
Patients who have brain metastases that have not progressed for at least 2 months following surgery or radiotherapy will be considered after discussion with the study chairman.
Patients must have a life expectancy of more than 3 months.
Patients must have normal organ and marrow function, unless in the opinion of the treating investigator, the abnormality is related to tumor, and the study chairman agree the abnormality is unlikely to affect the safety of perifosine use. Normal organ and marrow function is described below:
- ANC >1.5 x 109 /L
- Platelets >75,000/ mm3
- HCT > 28% (with or without growth factor support)
- Creatinine <= 2.5 mg/dl
- Total bilirubin < 1.5 x upper limit of normal
- Transaminase <= 2.5 x upper limit of normal
Patients must have recovered from acute toxicity related to prior therapy, including surgery or radiotherapy to grade <= 1 (excluding alopecia) at the time of enrollment.
Patients must be able to ingest oral medications or to obtain them through a gastrostomy tube.
Female patients who are pregnant or lactating are ineligible. All females of childbearing potential must have a negative urine or serum pregnancy test within 72 hours of treatment. Men and women of childbearing potential must agree to employ adequate contraception to prevent pregnancy while on therapy and for four weeks after the completion of treatment.
Patients must have the ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

Patients receiving investigational or commercial agents or therapies administered with the intent to treat the patient's malignancy, except bisphosphonates.
History of allergic reactions attributed to compounds of similar chemical or biologic composition to perifosine (miltefosine or edelfosine).
Uncontrolled intercurrent illness-including, but not limited to, ongoing or active infection-and psychiatric illness/social situations that would limit compliance with study requirements.
Patients with a history of unstable or newly diagnosed angina pectoris, recent myocardial infarction (within 6 months of enrollment) or New York Heart Assoc. class II-IV congestive heart failure.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Group A: chondrosarcoma

Group B: alveolar soft part sarcoma

Group C: extra-skeletal myxoid

Arm Description

Patients with sarcoma subtype: histologically or cytologically confirmed diagnosis of chondrosarcoma. Supportive Care Guidelines for perifosine include antiemetic prophylaxis (antiemetics will be administered at the treating investigator's discretion), diarrhea management (loperamide), and hyperuricemia prophylaxis (allopurinol).

Patients with sarcoma subtype: histologically or cytologically confirmed diagnosis of alveolar soft part sarcoma. Supportive Care Guidelines for perifosine include antiemetic prophylaxis (antiemetics will be administered at the treating investigator's discretion), diarrhea management (loperamide), and hyperuricemia prophylaxis (allopurinol).

Patients with sarcoma subtype: histologically or cytologically confirmed diagnosis of extra-skeletal myxoid chondrosarcoma. Supportive Care Guidelines for perifosine include antiemetic prophylaxis (antiemetics will be administered at the treating investigator's discretion), diarrhea management (loperamide), and hyperuricemia prophylaxis (allopurinol).

Outcomes

Primary Outcome Measures

Response rate

To investigate whether perifosine has a response rate of > 20% in this group of patients with chemo-insensitive sarcomas.

Secondary Outcome Measures

Best overall response

Best response recorded from the start of the treatment until disease progression/recurrence.

Stable disease of six months or greater

Stable disease is measured from the start of treatment until the criteria for progression are met, taking as reference the smallest measurements recorded since the treatment started.

Full Information

NCT ID

NCT00401388

First Posted

November 17, 2006

Last Updated

March 6, 2018

Sponsor

AEterna Zentaris

Collaborators

Sarcoma Alliance for Research through Collaboration

1. Study Identification

Unique Protocol Identification Number

NCT00401388

Brief Title

A Trial of Perifosine in Patients With Chemo-Insensitive Sarcomas

Official Title

Sarcoma Alliance for Research Through Collaboration (SARC) Multicenter Trial: A Phase II Trial of Perifosine in Patients With Chemo-Insensitive Sarcomas

Study Type

Interventional

2. Study Status

Record Verification Date

February 2012

Overall Recruitment Status

Completed

Study Start Date

November 2006 (undefined)

Primary Completion Date

January 2011 (Actual)

Study Completion Date

October 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

AEterna Zentaris

Collaborators

Sarcoma Alliance for Research through Collaboration

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a phase II study of perifosine in patients with chondrosarcomas, alveolar soft part sarcomas and extra-skeletal myxoid chondrosarcomas. Patients will receive perifosine 100 mg orally qhs with food until disease progression. The goals of this study include: In this study a daily dose of perifosine previously determined to be relatively non-toxic will be evaluated in patients with chondrosarcomas, alveolar soft part sarcomas and extra-skeletal myxoid chondrosarcomas. Response to therapy will be based on regression of measurable disease according to Choi criteria. Time to progression and duration of stable disease will be measured as secondary endpoints of the study.

Detailed Description

This is a phase II study of perifosine in patients with chondrosarcomas, alveolar soft part sarcomas and extra-skeletal myxoid chondrosarcomas. Patients will receive perifosine 100 mg orally qhs with food until disease progression. Perifosine is available in 50 mg tablets. Patients will take two perifosine 50 mg tablets orally once a day at bedtime with food. Administering the drug qhs has been shown to decrease gastrointestinal toxicity in some patients. Patients may need anti-emetics and/or anti-diarrheals. All patients may continue therapy unless disease progression is documented on two occasions at least 4 weeks apart. Patients who experience toxicity may continue on treatment with doses delayed or reduced. Evaluation of all lesions for progression or response will be made at 3-month intervals. STUDY KEY POINTS Treatment will be administered on an outpatient basis in 28-day cycles. Growth factors should not be needed, however, use by patients on this trial is NOT prohibited. A favorable outcome will be defined as a complete or partial response according to the Choi criteria or stable disease by the Choi criteria for 6 months or longer.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Chondrosarcomas, Alveolar Soft Part Sarcomas, Extra Skeletal Myxoid Chondrosarcomas

Keywords

Chemo Insensitive Sarcomas, Chondrosarcomas, Alveolar soft part sarcomas, Extra skeletal myxoid chondrosarcomas, Perifosine

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

One treatment arm: Perifosine. Three groups related to sarcoma subtype: histologically or cytologically confirmed diagnosis of (a) chondrosarcoma, (b) alveolar soft part sarcoma and (c) extra-skeletal myxoid chondrosarcoma.

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

72 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Group A: chondrosarcoma

Arm Type

Experimental

Arm Description

Arm Title

Group B: alveolar soft part sarcoma

Arm Type

Experimental

Arm Description

Arm Title

Group C: extra-skeletal myxoid

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Perifosine

Other Intervention Name(s)

D-21266, KRX-0401

Intervention Description

Perifosine is available in 50 mg tablets. Patients will take two perifosine 50 mg tablets orally once a day at bedtime with food.

Intervention Type

Other

Intervention Name(s)

Loperamide

Intervention Description

All patients should be instructed to take loperamide at the earliest signs of diarrhea and/or abdominal cramping after beginning perifosine.

Intervention Type

Other

Intervention Name(s)

Allopurinol

Intervention Description

Patients with a known history of hyperuricemia and/or gout should receive prophylactic treatment with allopurinol 300 mg po daily.

Intervention Type

Other

Intervention Name(s)

Antiemetics

Intervention Description

Antiemetic prophylaxis will be administered at the treating investigator's discretion.

Primary Outcome Measure Information:

Title

Response rate

Description

To investigate whether perifosine has a response rate of > 20% in this group of patients with chemo-insensitive sarcomas.

Time Frame

>= 6 months

Secondary Outcome Measure Information:

Title

Best overall response

Description

Best response recorded from the start of the treatment until disease progression/recurrence.

Time Frame

>= 6 months

Title

Stable disease of six months or greater

Description

Stable disease is measured from the start of treatment until the criteria for progression are met, taking as reference the smallest measurements recorded since the treatment started.

Time Frame

>= 6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

13 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patients must have histologically or cytologically confirmed diagnosis of chondrosarcoma, extra-skeletal myxoid chondrosarcoma or alveolar soft part sarcoma. Patients may have had prior chemotherapy, but if the patient has had three or more forms of prior chemotherapy for metastases, the patient's clinical course should be discussed with the study chairman before the patient is enrolled on study Patients must have progression of disease by Choi criteria. ECOG performance status 0-1. Patients with ECOG PS of 2 may be admitted with approval from the study chairman. At least 13 years of age. Patients must have measurable disease. Patients who have brain metastases that have not progressed for at least 2 months following surgery or radiotherapy will be considered after discussion with the study chairman. Patients must have a life expectancy of more than 3 months. Patients must have normal organ and marrow function, unless in the opinion of the treating investigator, the abnormality is related to tumor, and the study chairman agree the abnormality is unlikely to affect the safety of perifosine use. Normal organ and marrow function is described below: ANC >1.5 x 109 /L Platelets >75,000/ mm3 HCT > 28% (with or without growth factor support) Creatinine <= 2.5 mg/dl Total bilirubin < 1.5 x upper limit of normal Transaminase <= 2.5 x upper limit of normal Patients must have recovered from acute toxicity related to prior therapy, including surgery or radiotherapy to grade <= 1 (excluding alopecia) at the time of enrollment. Patients must be able to ingest oral medications or to obtain them through a gastrostomy tube. Female patients who are pregnant or lactating are ineligible. All females of childbearing potential must have a negative urine or serum pregnancy test within 72 hours of treatment. Men and women of childbearing potential must agree to employ adequate contraception to prevent pregnancy while on therapy and for four weeks after the completion of treatment. Patients must have the ability to understand and the willingness to sign a written informed consent document. Exclusion Criteria: Patients receiving investigational or commercial agents or therapies administered with the intent to treat the patient's malignancy, except bisphosphonates. History of allergic reactions attributed to compounds of similar chemical or biologic composition to perifosine (miltefosine or edelfosine). Uncontrolled intercurrent illness-including, but not limited to, ongoing or active infection-and psychiatric illness/social situations that would limit compliance with study requirements. Patients with a history of unstable or newly diagnosed angina pectoris, recent myocardial infarction (within 6 months of enrollment) or New York Heart Assoc. class II-IV congestive heart failure.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Dejka Araujo, MD

Organizational Affiliation

MD Anderson Cancer Center, Dept of Sarcoma

Official's Role

Study Chair

12. IPD Sharing Statement

Learn more about this trial

A Trial of Perifosine in Patients With Chemo-Insensitive Sarcomas

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