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LeukemiaPhase 1NCT00022451

Tipifarnib in Treating Young Patients With Refractory Leukemia

A Phase 1 study testing tipifarnib in people with Leukemia, run by National Institutes of Health Clinical Center (CC) at 61 sites in 3 countries.

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

RATIONALE: Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. PURPOSE: Phase I trial to study the effectiveness of tipifarnib in treating young patients who have refractory leukemia. Read more about this study

Who is it for?
Ages Up to 21 years
Conditions the study is for
Leukemia
What is being tested?
tipifarnib
How many people
Not given
Where
61 sitesin 3 countries
Study dates
Jun 2001to Mar 2005
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 Up to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

DISEASE CHARACTERISTICS:

  • Histologically confirmed acute lymphoblastic leukemia, acute nonlymphoblastic leukemia, juvenile myelomonocytic leukemia (JMML), or chronic myelogenous leukemia (CML) in blast crisis
  • Refractory to standard curative therapy
  • Acute promyelocytic leukemia refractory to tretinoin and arsenic trioxide
  • Philadelphia chromosome-positive CML refractory to imatinib mesylate
  • Greater than 25% blasts in bone marrow (M3 bone marrow) except for patients with JMML
  • Active extramedullary disease allowed
  • No active leptomeningeal leukemia

PATIENT CHARACTERISTICS:

Age:

  • 21 and under

Performance status:

  • Karnofsky 50-100% (over 10 years of age)
  • Lansky 50-100% (10 years of age and under)

Life expectancy:

  • Not specified

Hematopoietic:

  • Not required to be normal

Hepatic:

  • Bilirubin normal
  • SGPT and SGOT normal
  • No significant hepatic dysfunction
  • No grade 3 or 4 liver function test results within the past month

Renal:

  • Creatinine normal OR
  • Creatinine clearance at least 60 mL/min
  • No significant renal dysfunction

Cardiovascular:

  • No significant cardiac dysfunction

Pulmonary:

  • No significant pulmonary dysfunction

Neurologic:

  • No history of grand mal seizures grade 3 or greater except febrile seizures
  • No persistent sensory or motor neuropathy greater than grade 2

Other:

  • No clinically significant unrelated systemic illness
  • No serious infection
  • No organ dysfunction that would preclude study participation
  • No requirement for total parenteral nutrition
  • No known allergy to azoles (e.g., clotrimazole, fluconazole, ketoconazole, voriconazole)
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • At least 1 week since prior colony-stimulating factor therapy (e.g., filgrastim [G-CSF] or sargramostim [GM-CSF]) except epoetin alfa
  • At least 3 months since prior myeloablative therapy followed by bone marrow or stem cell transplantation
  • No concurrent immunotherapy
  • No concurrent GM-CSF or interleukin-11

Chemotherapy:

  • At least 2 weeks since prior chemotherapy
  • No concurrent intrathecal chemotherapy
  • No other concurrent chemotherapy

Endocrine therapy:

  • At least 1 week since prior corticosteroids
  • No concurrent corticosteroids (except for acute allergic reaction)

Radiotherapy:

  • At least 4 weeks since prior radiotherapy
  • No concurrent radiotherapy

Surgery:

  • Not specified

Other:

  • Recovered from nonhematologic toxicity of all prior therapy
  • At least 1 week since prior retinoids
  • No antacids (magnesium- or aluminum-containing formulations) within 2 hours of study drug
  • No other concurrent investigational agents
  • No concurrent retinoids
  • No concurrent anticonvulsants

Trial locations and contacts

61 sites in 3 countries.

61 sites in 3 countries

United States56 sites

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109-0914

  • AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish Rite Campus

    Atlanta, Georgia, 30342

  • MBCCOP-Medical College of Georgia Cancer Center

    Augusta, Georgia, 30912-4000

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-7223

  • Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support

    Bethesda, Maryland, 20892-1182

  • Floating Hospital for Children

    Boston, Massachusetts, 02111

  • Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Hollings Cancer Center at Medical University of South Carolina

    Charleston, South Carolina, 29425-0721

  • Children's Memorial Hospital - Chicago

    Chicago, Illinois, 60614

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039

  • Columbus Children's Hospital

    Columbus, Ohio, 43205-2696

  • Simmons Cancer Center at University of Texas Southwestern Medical Center - Dallas

    Dallas, Texas, 75390-9063

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010-3000

  • Duke Comprehensive Cancer Center

    Durham, North Carolina, 27710

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104

  • Shands Cancer Center at the University of Florida Health Science Center

    Gainesville, Florida, 32610-0296

  • Cancer Center at Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Texas Children's Cancer Center

    Houston, Texas, 77030-2399

  • University of Texas - MD Anderson Cancer Center

    Houston, Texas, 77030-4009

  • Riley Children Cancer Center at Riley Hospital for Children

    Indianapolis, Indiana, 46202-5225

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216-4505

  • Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center

    Kansas City, Kansas, 66160-7357

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • Rebecca and John Moores UCSD Cancer Center

    La Jolla, California, 92093-0658

  • Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027-0700

  • Jonsson Comprehensive Cancer Center, UCLA

    Los Angeles, California, 90095-1781

  • University of Wisconsin Comprehensive Cancer Center

    Madison, Wisconsin, 53792-6164

  • CCOP - Marshfield Clinic Research Foundation

    Marshfield, Wisconsin, 54449

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105-2794

  • Midwest Children's Cancer Center

    Milwaukee, Wisconsin, 53226

  • University of Minnesota Cancer Center

    Minneapolis, Minnesota, 55455

  • Vanderbilt Children's Hospital

    Nashville, Tennessee, 37232-6310

  • Cancer Institute of New Jersey at Robert Wood Johnson University Hospital

    New Brunswick, New Jersey, 08901

  • MBCCOP - LSU Health Sciences Center

    New Orleans, Louisiana, 70112

  • NYU School of Medicine's Kaplan Comprehensive Cancer Center

    New York, New York, 10016

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10021

  • Herbert Irving Comprehensive Cancer Center at Columbia University

    New York, New York, 10032

  • Oklahoma University Medical Center

    Oklahoma City, Oklahoma, 73126

  • Children's Hospital of Orange County

    Orange, California, 92868

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213-2583

  • CCOP - Columbia River Oncology Program

    Portland, Oregon, 97225

  • Mayo Clinic Cancer Center

    Rochester, Minnesota, 55905

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78207

  • MBCCOP - South Texas Pediatrics

    San Antonio, Texas, 78229-3900

  • UCSF Comprehensive Cancer Center

    San Francisco, California, 94143

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105

  • Cardinal Glennon Children's Hospital

    St Louis, Missouri, 63104

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110

  • Stanford Cancer Center at Stanford University Medical Center

    Stanford, California, 94305-5208

  • SUNY Upstate Medical University Hospital

    Syracuse, New York, 13210

  • CCOP - Scott and White Hospital

    Temple, Texas, 76508

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010-2970

Other trials in United States

Canada3 sites

  • Montreal Children's Hospital at McGill University Health Center

    Montreal, Quebec, H3G 1A4

  • Hopital Sainte Justine

    Montreal, Quebec, H3T 1C5

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

Australia2 sites

  • Royal Children's Hospital

    Parkville, Victoria, 3052

  • Princess Margaret Hospital for Children

    Perth, Western Australia, 6001

Other trials in Australia

About this study

Official title: A Phase I Trial and Pharmacokinetic Study of R115777 in Pediatric Patients With Refractory Leukemia

RATIONALE: Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth.

PURPOSE: Phase I trial to study the effectiveness of tipifarnib in treating young patients who have refractory leukemia.

Read the detailed description

OBJECTIVES:

Primary

  • Determine the maximum tolerated dose and toxicity profile of tipifarnib in pediatric patients with refractory leukemia.
  • Determine the pharmacokinetics of this drug in these patients.
  • Determine the toxicity profile of this drug in these patients.

Secondary

  • Analyze the gene expression profile of leukemic blasts from these patients before and after treatment with this drug.
  • Determine circulating levels of nerve growth factor and correlate these levels with clinical neurotoxicity from this drug in these patients.

OUTLINE: This is an open-label, dose-escalation study.

Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of tipifarnib until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 6 patients experience dose-limiting toxicity. At least 9 additional patients are treated at the MTD.

PROJECTED ACCRUAL: A total of 12-34 patients will be accrued for this study within 1-2 years.

Conditions
Leukemia
Being tested
tipifarnib
Keywords
recurrent childhood acute lymphoblastic leukemia, recurrent childhood acute myeloid leukemia, relapsing chronic myelogenous leukemia, blastic phase chronic myelogenous leukemia, chronic myelogenous leukemia, BCR-ABL1 positive, childhood acute promyelocytic leukemia (M3), acute undifferentiated leukemia, juvenile myelomonocytic leukemia, childhood chronic myelogenous leukemia
Medical subject headings
Leukemia, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Blast Crisis, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Biphenotypic, Acute, Leukemia, Myelomonocytic, Juvenile

What participants receive

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

  • tipifarnibDrug

Timeline

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

  1. First posted27 Jan 2003 (estimated)
  2. Study startJun 2001
  3. Study completionMar 2005

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
Masking
None (open label)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00022451
Sponsor's protocol ID
010196
Other IDs
01-C-0196C, COG-ADVL0116, NCI-1930, CDR0000068819
Status last verified
Mar 2012
First submitted
10 Aug 2001
Last updated
15 Mar 2012
Results posted
No

Publications

  • de Nigris F, Balestrieri ML, Napoli C. Targeting c-Myc, Ras and IGF cascade to treat cancer and vascular disorders. Cell Cycle. 2006 Aug;5(15):1621-8. doi: 10.4161/cc.5.15.3138. Epub 2006 Aug 1.

    PubMed 16921263

  • Widemann BC, Arceci RJ, Jayaprakash N, Fox E, Zannikos P, Goodspeed W, Goodwin A, Wright JJ, Blaney SM, Adamson PC, Balis FM. Phase 1 trial and pharmacokinetic study of the farnesyl transferase inhibitor tipifarnib in children and adolescents with refractory leukemias: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2011 Feb;56(2):226-33. doi: 10.1002/pbc.22775. Epub 2010 Sep 21.

    PubMed 20860038

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing tipifarnib in people with Leukemia, run by National Institutes of Health Clinical Center (CC) at 61 sites in 3 countries.

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

Who may be eligible to take part?

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 15 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 61 sites in United States, Canada, Australia. 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.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00022451, which also lists 2 linked publications.

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 15 Mar 2012.

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