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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00022451




Registration number
NCT00022451
Ethics application status
Date submitted
10/08/2001
Date registered
27/01/2003
Date last updated
15/03/2012

Titles & IDs
Public title
Tipifarnib in Treating Young Patients With Refractory Leukemia
Scientific title
A Phase I Trial and Pharmacokinetic Study of R115777 in Pediatric Patients With Refractory Leukemia
Secondary ID [1] 0 0
01-C-0196C
Secondary ID [2] 0 0
010196
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - tipifarnib

Treatment: Drugs: tipifarnib
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
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
Minimum age
No limit
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2001
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/03/2005
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC,WA
Recruitment hospital [1] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [2] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 0 0
6001 - Perth
Recruitment outside Australia
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Arkansas
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United States of America
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California
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District of Columbia
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Florida
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Georgia
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Illinois
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Indiana
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Kansas
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Louisiana
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Mississippi
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Missouri
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New Jersey
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New York
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North Carolina
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Ohio
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Oklahoma
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Oregon
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Pennsylvania
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South Carolina
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Tennessee
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Texas
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Utah
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Washington
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Wisconsin
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Canada
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Ontario
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Canada
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Quebec

Funding & Sponsors
Primary sponsor type
Government body
Name
National Institutes of Health Clinical Center (CC)
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Cancer Institute (NCI)
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Children's Oncology Group
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00022451
Trial related presentations / 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.
Public notes

Contacts
Principal investigator
Name 0 0
Brigitte C. Widemann, MD
Address 0 0
National Cancer Institute (NCI)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00022451