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Chronic Lymphocytic LeukemiaPhase 2NCT00873457

Perifosine in Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

A Phase 2 study testing perifosine in people with Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma, run by Daphne Friedman at 1 site in United States.

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

Perifosine inhibits the AKT pathway (a way cells communicate with each other). This pathway is felt to be important in the development of several types of cancers including chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Read more about this study

Who is it for?
Ages 18 years and older
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
16 participants
Where
1 siteUnited States
Study dates
Aug 2009to Apr 2013
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • A diagnosis of CLL or SLL based on iwCLL diagnostic criteria.
  • Prior therapy for CLL (no limit on number of prior regimens).
  • Patients requiring therapy, based on at least one of the iwCLL criteria.
  • 18 years of age or older.
  • Performance status ECOG 0, 1, or 2.
  • An estimated or measured creatinine clearance ≥30 ml/min at study enrollment.
  • AST, ALT, and total bilirubin ≤ 2.5 times the upper limit of normal, unless due to CLL/SLL.
  • Female subject who is either post-menopausal or surgically sterilized or male or female subject willing to use an acceptable method of birth control for the duration of the study therapy and for 2 weeks after study therapy completion.

Who cannot join

  • Female subject is pregnant or lactating.
  • Patient has received other investigational drugs for this disease within 14 days of enrollment.
  • Patient with known HIV prior to enrollment.
  • Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
  • Patients with another malignancy within the last three years (from documentation of remission) other than basal or squamous cell skin cancer or CIS of the cervix or early stage prostate cancer not requiring systemic treatment.
  • Patients who underwent allogeneic stem cell transplant and have at least 2% donor cells engrafted will be excluded.
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  • Significant cardiac or vascular events within 6 months: acute MI, unstable angina, severe peripheral vascular disease (ischemic pain at rest class 3 or worse, non-healing ulcers/wounds, congestive heart failure (NHYA class ≥ 2), uncontrolled cardiac arrhythmias.
  • Known severe hypersensitivity to perifosine or any component of the formulation.
  • Life expectancy less than six months due to co-morbid illness
  • Active autoimmune hemolytic anemia or immune thrombocytopenia, requiring current steroid therapy.
  • De novo prolymphocytic leukemia (PLL) or PLL arising from CLL (≥ 55% prolymphocytes).
  • Richter's transformation

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Duke University Medical Center

    Durham, North Carolina, 27710

Other trials in United States

About this study

Official title: Phase II Trial of Perifosine in Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Perifosine inhibits the AKT pathway (a way cells communicate with each other). This pathway is felt to be important in the development of several types of cancers including chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). It is thought perifosine may be able to block this pathway and lead to an improvement in the CLL or SLL. The purpose of this trial is to see if perifosine is an effective treatment for relapsed or refractory CLL or SLL. Another purpose of this study is to look at the effect perifosine has on cells.

Read the detailed description

Chronic lymphocytic leukemia and small B-cell lymphocytic lymphoma represent different manifestations of the same disease. CLL/SLL (hereafter denoted by CLL) is a clonal disorder of small B lymphocytes expressing a characteristic morphology and immunophenotype. The B cells express CD19, dim CD 20, dim CD 5, CD 23, CD 43, CD 79a, and weakly express surface immunoglobin. CLL can present asymptomatically in 25% of patients when diagnosed on a complete blood count. It also can present with diffuse painless lymphadenopathy and, in a smaller number of patients, B symptoms.

CLL is characterized by accumulation of circulating B cells predominantly in the G0 phase of the cell cycle. These cells are resistant to apoptosis. CLL has been found to have aberrant signaling in several pathways including NF-kB, Akt/PI3K, and JNK/STAT pathways. Akt is important in promoting CLL survival and viability, as seen in in vitro experiments where blocking its activity results in apoptosis. Thus an AKT inhibitor may lead to increased apoptosis and may have a role in the treatment of this disease.

Treatment options for CLL range from a watch and wait approach to high dose chemotherapy with stem cell support. Currently, there is no consensus on the best treatment regimen, since no treatment has been shown to improve survival in randomized prospective clinical trials. New approaches to treatment, especially those with lower toxicity rates, are needed.

Perifosine has been shown to inhibit or otherwise modify signaling through a number of different signal transduction pathways, including Akt, MAPK, and JNK. These pathways are involved in the development of cancers and resistance to chemotherapy. Perifosine is of particular interest, especially due to the difficulty in discovery of drugs that inhibit these pathways with minimal toxicity. The effect of perifosine on CLL cells has been tested in the laboratory and has been shown to be an active agent against primary CLL cells in vitro.

Being tested
perifosine
Keywords
B-Cell Chronic Lymphocytic Leukemia, Leukemia
Medical subject headings
Leukemia, Lymphocytic, Chronic, B-Cell, 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

    Perifosine 50 mg will be taken orally twice a day for a maximum of six 28-day cycles

Study groups in NCT00873457
Study groupRoleWhat they receive
PerifosineExperimentalPerifosine 50 mg twice a day for a total of six 28-day cycles.
Receives: Drug: perifosine

What is being measured

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

  1. Overall Response

    Per International Workshop on Chronic Lymphocytic Leukemia, Complete Response (CR):normal CBC; absence of the following: clonal lymphocytes in blood and marrow, lymphadenopathy, hepatomegaly or splenomegaly, and constitutional symptoms; and bone marrow has <30% lymphocytes, is normocellular, and is without B-lymphoid nodules. Partial Response(PR): one of the following: decrease lymphadenopathy ≥ 50%; decrease of liver and/or spleen size ≥ 50%, any constitutional symptoms, Polymorphonuclear leukocytes ≥ 1,500/µl or a 50% improvement, or decrease of circulating clonal B lymphocytes ≥ 50% AND one of the following: Platelets ≥ 100,000/µl or a 50% improvement, Hemoglobin ≥ 11.0 g/dl or a 50% improvement, Bone marrow has ≥ 30% lymphocytes, or B-lymphoid nodules, or not done. Overall Response (OR)= CR+PR

    Measured: after 3 months of treatment

  2. Overall Response

    Per International Workshop on Chronic Lymphocytic Leukemia, Complete Response (CR):normal CBC; absence of the following: clonal lymphocytes in blood and marrow, lymphadenopathy, hepatomegaly or splenomegaly, and constitutional symptoms; and bone marrow has <30% lymphocytes, is normocellular, and is without B-lymphoid nodules. Partial Response(PR): one of the following: decrease lymphadenopathy ≥ 50%; decrease of liver and/or spleen size ≥ 50%, any constitutional symptoms, Polymorphonuclear leukocytes ≥ 1,500/µl or a 50% improvement, or decrease of circulating clonal B lymphocytes ≥ 50% AND one of the following: Platelets ≥ 100,000/µl or a 50% improvement, Hemoglobin ≥ 11.0 g/dl or a 50% improvement, Bone marrow has ≥ 30% lymphocytes, or B-lymphoid nodules, or not done. Overall Response (OR)= CR+PR

    Measured: after 6 months of treatment

Secondary outcomes (2)

  1. Overall Survival

    Overall survival is defined as the length of time between discontinuation of perifosine until death or 2 year's followup, whichever comes first.

    Measured: up to a maximum of 2 years

  2. Event-free Survival

    Event-free survival will be defined as the length of time between the discontinuation of study treatment and disease progression, next therapy, or death,whichever comes first, up to a maximum of 2 years.

    Measured: up to a maximum of 2 years

Timeline

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

  1. First posted1 Apr 2009 (estimated)
  2. Study startAug 2009
  3. Primary completionOct 2011
  4. Study completionApr 2013

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00873457
Sponsor's protocol ID
Pro00015060
Status last verified
May 2013
First submitted
30 Mar 2009
Last updated
27 May 2013
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing perifosine in people with Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma, run by Daphne Friedman at 1 site in United States.

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 8 inclusion and 12 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 27 May 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

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?

Yes. Results are posted in the registry (first posted 24 Jan 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 (time frame: after 3 months of treatment). The study lists 2 primary outcomes in all. 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 NCT00873457.

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 27 May 2013.

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