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AzTrials

Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic SyndromePhase 1NCT00101296

Tipifarnib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1 study testing tipifarnib, pharmacological study and laboratory biomarker analysis in people with Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome and Adult Acute Erythroid Leukemia (M6), run by National Cancer Institute (NCI) 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

Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. This phase I trial is studying the side effects and best dose of tipifarnib in treating patients with relapsed or refractory acute myeloid leukemia Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
tipifarnib, pharmacological study and laboratory biomarker analysis
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteUnited States
Study dates
Oct 2004to May 2011
Phase
Phase 1
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

  • Adult patients with acute myeloid leukemia (AML), excluding the M3 subtype (acute promyelocytic leukemia), that are not likely to respond to conventional therapy, including:
  • Relapsed or refractory AML after one to three prior induction regimens (not counting consolidation therapies while in CR, such as autologous transplant),
  • Newly diagnosed AML in patients up to age-70 with poor risk features (unfavorable cytogenetics or findings suggestive of prior myelodysplasia) not fit for standard induction therapy, and
  • Newly diagnosed AML patients age 70 - 75 not fit for standard therapy (without history of prior myelodysplastic syndrome [MDS])
  • Bone marrow and peripheral blood studies must be available for confirmation of diagnosis; cytogenetics, flow cytometry, and molecular studies (such as fms-related tyrosine kinase 3 [Flt-3] status) will be obtained as per standard practice
  • Patients with active central nervous system (CNS) leukemia are NOT eligible
Show all 21Show fewer
  • Performance status of 60% or greater by the Karnofsky scale
  • If induction chemotherapy has been attempted, a minimum of 4 weeks must have elapsed since the completion of prior chemotherapy in order to be eligible for this study; hydroxyurea for control of blasts is not counted as chemotherapy, and may be given up until 24 hours before starting R115777
  • Patients may have had prior autologous transplant; they must be at least 100 days post transplant, and have had recovery of their counts with ANC > 1000 and platelets greater than 100K at some point post transplant, and be without active CMV or fungal disease
  • Patients may have received prior radiation therapy as part of a transplant conditioning regimen; radiotherapy must have been completed at least 100 days prior to starting on R115777
  • There are no minimum hematological parameter requirements prior to the first two cycles of R115777, as patients with AML and MDS are understood to have low ANC and platelet counts when the disease is active; however, patients with WBC greater than 30,000 will receive hydroxyurea to reduce WBC to below 30,000 at which point they may begin treatment with R115777
  • A pretreatment calculated creatinine clearance (absolute value) of >= 60 ml/minute or serum creatinine of < 1.5 x upper limit of normal is required
  • Serum bilirubin =< 2.0 mg/dl
  • SGOT and SGPT =< 2.5 times the institutional upper limits of normal
  • Any condition causing inability to swallow pills given by mouth will render patients ineligible for the trial
  • There must be no plans for the patient to receive concurrent hormonal, biologic, or radiation therapy
  • Patients eligible, at the time of starting the drug, for curative therapeutic approaches (such as allogeneic transplant) are not eligible for the trial; however, patients who achieve CR or PR as a result of R115777 may go on to allogeneic transplant
  • Patients must be informed of the investigational nature of this study and must sign and give written informed consent in accordance with institutional and federal guidelines
  • Prior to the first patient registration, the notification of institutional review board approval for this study must be provided to the Data Coordinating Center at City of Hope
  • Antacids are allowed but must not be taken concurrently with R115777; (there must be at least 2 hours between antacid intake and R115777 dosing)
  • Pregnant or lactating women are excluded from this trial; all patients of child-bearing potential, both male and female, must be advised to practice adequate contraception; premenopausal women must have a negative pregnancy test prior to entry on this study

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • City of Hope

    Duarte, California, 91010

Other trials in United States

About this study

Official title: Phase I Trial of R115777 (NSC 702818) in Relapsed, Refractory or High Risk Myeloid Leukemia

Tipifarnib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. This phase I trial is studying the side effects and best dose of tipifarnib in treating patients with relapsed or refractory acute myeloid leukemia

Read the detailed description

PRIMARY OBJECTIVES:

  1. To define the maximum tolerated dose (MTD) of R115777 (tipifarnib) in patients with relapsed, refractory, or high risk myeloid leukemias treated according to this regimen.

II. To assess the toxicity and preliminary assessment of efficacy of R115777 in patients with relapsed, refractory, or high risk myeloid leukemias.

OUTLINE: This is a dose-escalation, multicenter study.

Patients receive oral tipifarnib twice daily on days 1-7 and 15-21. Courses repeat every 28 days in the absence of unacceptable toxicity or disease progression. Patients achieving a complete response (CR) receive 2 additional courses beyond CR. Patients experiencing relapse after previously achieving CR may receive additional tipifarnib at the current dose level for newly registered patients.

Cohorts of 3-6 patients receive escalating doses of tipifarnib until the MTD is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed every 6 months for survival.

PROJECTED ACCRUAL: A total of 20-30 patients will be accrued for this study.

Being tested
tipifarnib, pharmacological study, laboratory biomarker analysis
Medical subject headings
Leukemia, Erythroblastic, Acute, Leukemia, Megakaryoblastic, Acute, Leukemia, Monocytic, Acute, Leukemia, Myeloid, Acute, Congenital Abnormalities, Leukemia, Myelomonocytic, Acute

What participants receive

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

  • tipifarnibDrug

    Also called R115777, Zarnestra

    Given orally

  • pharmacological studyOther

    Also called pharmacological studies

    Correlative studies

  • laboratory biomarker analysisOther

    Correlative studies

Study groups in NCT00101296
Study groupRoleWhat they receive
Treatment (tipifarnib)ExperimentalPatients receive oral tipifarnib twice daily on days 1-7 and 15-21. Courses repeat every 28 days in the absence of unacceptable toxicity or disease progression. Patients achieving a CR receive 2 additional courses beyond CR. Patients experiencing relapse after previously achieving CR may receive additional tipifarnib at the current dose level for newly registered patients.
Receives: Drug: tipifarnib; Other: pharmacological study; Other: laboratory biomarker analysis

What is being measured

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

  1. MTD defined as the highest dose level in which six patients have been evaluated for toxicity with no more than one patient experiencing dose limiting toxicity (DLT) assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 3.0

    The toxicities observed at each dose level will be summarized in terms of type, severity, time of onset, duration, and reversibility or outcome. Tables will be created to summarize these toxicities and side effects by dose and by course.

    Measured: 28 days

  2. Objective tumor response

    Will be summarized at each dose level, and the number and percent responding combined across dose levels.

    Measured: Up to 6.5 years

  3. Survival

    Will be summarized with Kaplan-Meier plots.

    Measured: From registration to time of death due to any cause, assessed up to 6.5 years

  4. Time to treatment failure

    Will be summarized with Kaplan-Meier plots.

    Measured: From registration to the first observation of disease progression, death due to any cause, or early discontinuation of treatment, assessed up to 6.5 years

  5. Duration of response

    Will be summarized with Kaplan-Meier plots.

    Measured: Up to 6.5 years

Timeline

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

  1. First posted10 Jan 2005 (estimated)
  2. Study startOct 2004
  3. Primary completionMay 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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00101296
Sponsor's protocol ID
NCI-2012-03078
Other IDs
PHI-47, U01CA062505, CDR0000404151
Status last verified
Jan 2013
First submitted
7 Jan 2005
Last updated
4 Feb 2013
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing tipifarnib, pharmacological study and laboratory biomarker analysis in people with Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome and Adult Acute Erythroid Leukemia (M6), run by National Cancer Institute (NCI) 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 21 inclusion and 0 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 4 Feb 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

MTD defined as the highest dose level in which six patients have been evaluated for toxicity with no more than one patient experiencing dose limiting toxicity (DLT) assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 (time frame: 28 days). The study lists 5 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 NCT00101296.

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 4 Feb 2013.

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