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LeukemiaPhase 2NCT00391560

Phase II Study of Perifosine in Patients With Refractory and Relapsed Leukemia

A Phase 2 study testing perifosine in people with Leukemia, run by AEterna Zentaris.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a Phase II trial designed to determine the efficacy and safety of perifosine in patients with leukemia who develop progressive disease or recurrence while receiving therapy. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leukemia
What is being tested?
perifosine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
No sites listed yet
Study dates
Oct 2006to Oct 2011
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have relapsed/refractory leukemias for which no standard therapies are anticipated to result in a durable remission. Patients with poor-risk myelodysplasia (MDS) [i.e. refractory anemia with excess blasts (RAEB-1 or RAEB-2) by WHO classification] and chronic myelomonocytic leukemia (CMML) are also candidates for this protocol. Relapsed/refractory leukemias include acute non-lymphocytic leukemia (AML) by WHO classification, acute lymphocytic leukemia (ALL), chronic lymphocytic leukemia (CLL), or chronic myelogenous leukemia (CML) in blast crisis. Patients with agnogenic myeloid metaplasia (AMM) are also eligible.
  • ECOG performance status of 0-2
  • Sexually active men and women who are not surgically sterile or post menopausal must use acceptable contraceptive methods (physician will discuss acceptable methods) during the time on study and for 4 weeks following the completion of treatment. Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must have a negative serum or urine pregnancy test within 2 weeks prior to beginning treatment on this trial.
  • In the absence of rapidly progressing disease, the interval from prior treatment to time of study drug administration should be at least 2 weeks for cytotoxic agents, or at least five half-lives for noncytotoxic agents. Persistent chronic toxicities from prior chemotherapy must not be greater than grade 1.
  • Patients must have the following clinical laboratory values:
  • Serum creatinine: <= 2.0 mg/dl
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  • Total bilirubin: <=1.5x the upper limit of normal unless considered due to Gilbert's syndrome
  • Alanine aminotransferase (ALT), or aspartate aminotransferase (AST): <= 3x the upper limit of normal unless considered due to organ leukemic involvement
  • Must be able and willing to give written informed consent
  • Age equal to or greater than 18 years

Who cannot join

  • Uncontrolled intercurrent illness including, but not limited to uncontrolled infection, symptomatic congestive heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Active heart disease including myocardial infarction within previous 3 months, symptomatic coronary artery disease, arrhythmias not controlled by medication, or uncontrolled congestive heart failure
  • Patients with a history of severe hyper-reactive airway system (e.g. active asthma, COPD)
  • Patients receiving any other standard or investigational treatment for their hematologic malignancy
  • Pregnant and nursing patients are excluded because the effects of perifosine on a fetus or nursing child are unknown.

About this study

This is a Phase II trial designed to determine the efficacy and safety of perifosine in patients with leukemia who develop progressive disease or recurrence while receiving therapy.

Read the detailed description

This is a Phase II study of perifosine in patients with refractory and relapsed leukemia. After a one time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed.

Patients will be assessed for efficacy at the end of each 28 day cycle of therapy, +/- 7 days. Complete remissions, partial remissions and hematological improvements of any kind will be counted towards an objective response for all diseases.

A maximum total of 74 patients will be enrolled on the study, all of them assigned to the same experimental treatment scheme (arm) described above.

A maximum total of 37 evaluable patients will be entered in each of two diagnostic groups, which are being distinguished due to different anticipated rates of accrual. Group 1: AML, MDS, CML-BP non-lymphoid, CMML, Agnogenic Myeloid Metaplasia (AMM); Group 2: CLL, ALL, CML-BP lymphoid.

Conditions
Leukemia
Being tested
perifosine
Keywords
refractory leukemia
Medical subject headings
Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • perifosineDrug

    Also called D-21266, KRX-0401

    Identical intervention in both arms.

Study groups in NCT00391560
Study groupRoleWhat they receive
Group 1 on PerifosineExperimentalPatients with AML, MDS, CML-BP non-lymphoid, CMML, or Agnogenic Myeloid Metaplasia (AMM). After a one-time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed.
Receives: Drug: perifosine
Group 2 on PerifosineExperimentalPatients with CLL, ALL, or CML-BP lymphoid. After a one-time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed.
Receives: Drug: perifosine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (CR + PR)

    All patients included in the study must be assessed for response to treatment, unless there are major protocol treatment deviations or they are ineligible.

    Even a 5% response rate would be of interest for this agent, given the different nature and mechanism of action of this compound.

    Measured: Every 4 weeks

Secondary outcomes (2)

  1. Time to Tumor Progression

    "Time to Progression" is defined as the period of time from the date of first study drug administration to the date that the patient is withdrawn because of clinical or radiographic progressive disease or death from any cause.

    Measured: Every 4 weeks

  2. Hematologic Improvement

    Hematological improvements of any kind will be counted towards an objective response for all diseases.

    Measured: Every 4 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Oct 2006 (estimated)
  2. Study startOct 2006
  3. Primary completionOct 2010
  4. Study completionOct 2011

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
Single treatment arm. Two disease related analyses groups, i.e.: Group 1: AML, MDS, CML-BP non-lymphoid, CMML, Agnogenic Myeloid Metaplasia (AMM); Group 2: CLL, ALL, CML-BP lymphoid
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00391560
Sponsor's protocol ID
Perifosine 217
Status last verified
Feb 2012
First submitted
20 Oct 2006
Last updated
14 Mar 2018
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing perifosine in people with Leukemia, run by AEterna Zentaris.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (CR + PR) (time frame: Every 4 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00391560.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Mar 2018.

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