## Protocol Section
### Identification Module
**NCT ID:** NCT00398879

**Brief Title:** Placebo-Controlled Study of Perifosine + Single Agent Chemotherapy for Metastatic Cancer Patients

**Official Title:** A Randomized Placebo-Controlled Study of Perifosine in Combination With Single Agent Chemotherapy for Metastatic Cancer Patients

#### Organization Study ID Info
**ID:** Perifosine 211
#### Organization
**Class:** INDUSTRY
**Full Name:** AEterna Zentaris
### Status Module
#### Completion Date
**Date:** 2011-10

**Type:** ACTUAL

#### Disp First Post Date
**Date:** 2018-03-14

**Type:** ACTUAL

**Disp First Submit Date:** 2018-03-12
**Disp First Submit QC Date:** 2018-03-12

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2018-03-14

**Type:** ACTUAL

**Last Update Submit Date:** 2018-03-12

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2010-12

**Type:** ACTUAL

#### Start Date
**Date:** 2005-08

**Status Verified Date:** 2012-02

#### Study First Post Date
**Date:** 2006-11-14
**Type:** ESTIMATED
**Study First Submit Date:** 2006-11-06
**Study First Submit QC Date:** 2006-11-10
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** AEterna Zentaris

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** This is an exploratory phase 2, randomized placebo-controlled trial with stratification for disease and chemotherapy type. The study is subsequently closed to enrollment in all arms except patients with metastatic colorectal cancer which would be randomized to either capecitabine plus perifosine or capecitabine alone.

The effects of perifosine may be manifested by increased time to progression, tumor regression reflected in partial or complete responses, or a combination of these outcomes. The primary goal of this trial is to obtain a preliminary and objective assessment of the effects of perifosine on time to progression.

**Detailed Description:** This is an exploratory phase 2, randomized placebo-controlled trial with stratification for disease and chemotherapy type. If there is any evidence of improved time to progression in any tumor type with any of the drugs to be evaluated, the initial study or component(s) of the study will be expanded to increase the certainty that this is an effect of perifosine. If there is compelling evidence of benefit from this study, a phase 3 trial will be conducted to obtain proof of principle.

Primary Study Objectives:

To determine the time to tumor progression when receiving single agent chemotherapy (capecitabine) in combination with perifosine in comparison to patients receiving single agent chemotherapy (capecitabine) alone (i.e., with placebo).

Secondary Study Objectives:

* To determine the toxicity of single agent chemotherapy in combination with perifosine.
* To compare the time to progression of chemotherapy in combination with placebo to historical experience.
* Overall survival will also be evaluated.

### Conditions Module

**Conditions:**

- Colon Cancer

**Keywords:**

- Capecitabine

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** SINGLE

**Who Masked:**

- PARTICIPANT

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 381

**Type:** ACTUAL

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Perifosine 50 mg/d qd + Capecitabine 825 mg/m\^2 BID days 1 - 14 q 3 weeks until progression

**Intervention Names:**

- Drug: Perifosine

- Drug: Capecitabine

**Label:** Arm 1: Perifosine + Capecitabine

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Perifosine Placebo 50 mg/d qd + Capecitabine 825 mg/m\^2 BID days 1 - 14 q 3 weeks until progression

**Intervention Names:**

- Drug: Capecitabine

- Other: Perifosine Placebo

**Label:** Arm 2: Perifosine Placebo + Capecitabine

**Type:** PLACEBO_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Arm 1: Perifosine + Capecitabine

**Description:** Perifosine 50 mg/d qd

**Name:** Perifosine

**Other Names:**

- D-21266

- KRX-0401

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Arm 1: Perifosine + Capecitabine

- Arm 2: Perifosine Placebo + Capecitabine

**Description:** Capecitabine 825 mg/m\^2 BID days 1 - 14 q 3 weeks

**Name:** Capecitabine

**Type:** DRUG

#### Intervention 3

**Arm Group Labels:**

- Arm 2: Perifosine Placebo + Capecitabine

**Description:** Placebo to Perifosine 50 mg/d qd

**Name:** Perifosine Placebo

**Other Names:**

- placebo

**Type:** OTHER

### Outcomes Module

#### Primary Outcomes

**Description:** Time to progression will be measured from the first day of study drug until progression.

**Measure:** Effects of perifosine on time to progression

**Time Frame:** Every 12 weeks

#### Secondary Outcomes

**Description:** Determination of the toxicity of single agent chemotherapy in combination with perifosine. Toxicity evaluation is to be performed throughout the study.

**Measure:** Toxicity

**Time Frame:** Every 12 weeks

**Description:** To compare the time to progression of chemotherapy in combination with placebo to historical experience.

**Measure:** Comparison of time to progression to historical experience

**Time Frame:** Every 12 weeks

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. In the opinion of the treating physician, treatment with one of the following regimens should represent an appropriate treatment for the patient.

   - Capecitabine 825 mg/m2 BID days 1 - 14 q 3 weeks
2. Patients should have a histologically or cytologically confirmed diagnosis of colorectal cancer.
3. Patients must have received at least one but no more than two prior chemotherapy regimen(s) for the treatment of metastatic or recurrent disease.
4. ECOG performance status 0 or 1.

   * Leukocytes \>= 4,000/μL
   * absolute neutrophil count \>= 1,500/ μL
   * platelets \>= 100,000/ μL
   * 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
5. Patients must have recovered from acute toxicity-excluding alopecia-related to prior therapy, including surgery or radiotherapy.
6. Patients with brain metastases may be admitted, provided the disease has been treated and been stable for 2 months.
7. Patients must have ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

1. Patients receiving any other investigational agents or devices.
2. History of allergic reactions attributed to compounds of similar chemical or biologic composition to perifosine (miltefosine or edelfosine).
3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, psychiatric illness, or social situations that would limit compliance with study requirements.
4. HIV-positive patients receiving combination anti-retroviral therapy are excluded from the study due to potential pharmacokinetic interactions with perifosine.
5. 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.
6. Female patients who are pregnant or lactating are ineligible.

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Tucson

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Arizona

**Zip:** 85704

**Location 2:**

**City:** Beverly Hills

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 90211

**Location 3:**

**City:** Deer Park

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 94574

**Location 4:**

**City:** Monterey

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 93940

**Location 5:**

**City:** Newport Beach

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 92663

**Location 6:**

**City:** Pomona

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 91767

**Location 7:**

**City:** Santa Rosa

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 95403

**Location 8:**

**City:** Soquel

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 95703

**Location 9:**

**City:** Stockton

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** California

**Zip:** 95207

**Location 10:**

**City:** Colorado Springs

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Colorado

**Zip:** 80909

**Location 11:**

**City:** Greeley

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Colorado

**Zip:** 80631

**Location 12:**

**City:** Middletown

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Connecticut

**Zip:** 06457

**Location 13:**

**City:** Norwich

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Connecticut

**Zip:** 06360

**Location 14:**

**City:** Aventura

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 33180

**Location 15:**

**City:** Coral Springs

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 33065

**Location 16:**

**City:** Lake City

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 32055

**Location 17:**

**City:** Miami

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 33176

**Location 18:**

**City:** Ormond Beach

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 32174

**Location 19:**

**City:** Sebastian

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 32958

**Location 20:**

**City:** Vero Beach

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Florida

**Zip:** 32960

**Location 21:**

**City:** Augusta

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Georgia

**Zip:** 30904

**Location 22:**

**City:** Lawrenceville

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Georgia

**Zip:** 30045

**Location 23:**

**City:** Marietta

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Georgia

**Zip:** 30060

**Location 24:**

**City:** Galesburg

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Illinois

**Zip:** 61401

**Location 25:**

**City:** Park Ridge

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Illinois

**Zip:** 60068

**Location 26:**

**City:** Louisville

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Kentucky

**Zip:** 40202

**Location 27:**

**City:** Lafayette

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Louisiana

**Zip:** 70506

**Location 28:**

**City:** Grand Rapids

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Michigan

**Zip:** 49546

**Location 29:**

**City:** Kalamazoo

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Michigan

**Zip:** 49048

**Location 30:**

**City:** Branson

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Missouri

**Zip:** 65616

**Location 31:**

**City:** Billings

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Montana

**Zip:** 59103

**Location 32:**

**City:** Great Falls

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Montana

**Zip:** 59405

**Location 33:**

**City:** Albany

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** New York

**Zip:** 12208

**Location 34:**

**City:** Armonk

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** New York

**Zip:** 10504

**Location 35:**

**City:** Great Neck

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** New York

**Zip:** 11042

**Location 36:**

**City:** Wilmington

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** North Carolina

**Zip:** 28401

**Location 37:**

**City:** Dayton

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Ohio

**Zip:** 45409

**Location 38:**

**City:** Pottsville

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Pennsylvania

**Zip:** 17901

**Location 39:**

**City:** Sayre

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Pennsylvania

**Zip:** 18840

**Location 40:**

**City:** Greenville

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** South Carolina

**Zip:** 29605

**Location 41:**

**City:** Chattanooga

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Tennessee

**Zip:** 37404

**Location 42:**

**City:** Memphis

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Tennessee

**Zip:** 38120

**Location 43:**

**City:** Dallas

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Texas

**Zip:** 75231

**Location 44:**

**City:** Dallas

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Texas

**Zip:** 75246

**Location 45:**

**City:** Tyler

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Texas

**Zip:** 75702

**Location 46:**

**City:** Chesapeake

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Virginia

**Zip:** 23320

**Location 47:**

**City:** Norfolk

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Virginia

**Zip:** 23502

**Location 48:**

**City:** Spokane

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Washington

**Zip:** 99218

**Location 49:**

**City:** Appleton

**Country:** United States

**Facility:** AOI Pharmaceuticals Investigative Site

**State:** Wisconsin

**Zip:** 54915

#### Overall Officials

**Official 1:**

**Affiliation:** Online Collaborative Oncology Group

**Name:** Craig Henderson, MD

**Role:** STUDY_CHAIR

### References Module

#### References

**Citation:** Bendell JC, Nemunaitis J, Vukelja SJ, Hagenstad C, Campos LT, Hermann RC, Sportelli P, Gardner L, Richards DA. Randomized placebo-controlled phase II trial of perifosine plus capecitabine as second- or third-line therapy in patients with metastatic colorectal cancer. J Clin Oncol. 2011 Nov 20;29(33):4394-400. doi: 10.1200/JCO.2011.36.1980. Epub 2011 Oct 3.

**PMID:** 21969495

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009369

- Term: Neoplasms

- ID: D000009385

- Term: Neoplastic Processes

- ID: D000010335

- Term: Pathologic Processes

### Condition Browse Module - Browse Branches

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

### Condition Browse Module - Browse Leaves

- ID: M12307

- Name: Neoplasm Metastasis

- Relevance: HIGH

- As Found: Metastatic Cancer

- ID: M12330

- Name: Neoplastic Processes

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000009362

- Term: Neoplasm Metastasis

### Intervention Browse Module - Ancestors

- ID: D000000964

- Term: Antimetabolites, Antineoplastic

- ID: D000000963

- Term: Antimetabolites

- ID: D000045504

- Term: Molecular Mechanisms of Pharmacological Action

- ID: D000000970

- Term: Antineoplastic Agents

### Intervention Browse Module - Browse Branches

- Abbrev: ANeo

- Name: Antineoplastic Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M377

- Name: Capecitabine

- Relevance: HIGH

- As Found: Function

- ID: M4281

- Name: Antimetabolites

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: D000069287

- Term: Capecitabine

### Misc Info Module

- Version Holder: 2024-05-24
