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Tipifarnib in Treating Older Patients With Acute Myeloid Leukemia

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ClinicalTrials.gov Identifier: NCT01361464
Recruitment Status : Completed
First Posted : May 26, 2011
Results First Posted : January 6, 2014
Last Update Posted : April 8, 2015
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Adult Acute Megakaryoblastic Leukemia
Adult Acute Monoblastic Leukemia
Adult Acute Monocytic Leukemia
Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11
Adult Acute Myeloid Leukemia With Maturation
Adult Acute Myeloid Leukemia With Minimal Differentiation
Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1
Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL
Adult Acute Myeloid Leukemia Without Maturation
Adult Acute Myelomonocytic Leukemia
Adult Erythroleukemia
Adult Pure Erythroid Leukemia
Alkylating Agent-Related Acute Myeloid Leukemia
Secondary Acute Myeloid Leukemia
Untreated Adult Acute Myeloid Leukemia
Interventions Drug: Tipifarnib
Other: Laboratory Biomarker Analysis
Enrollment 21
Recruitment Details The Southeast Phase II Consortium (SEP2C) enrolled participants at 3 cancer centers in the United States. The study opened to accrual on 5/24/2011 and closed to accrual 07/25/2012. Further development of Tipifarnib in acute myeloid leukemia (AML) was terminated after the study failed to meet the primary endpoint.
Pre-assignment Details  
Arm/Group Title R115777 Therapy
Hide Arm/Group Description Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Period Title: Overall Study
Started 21
Completed 18
Not Completed 3
Reason Not Completed
Death             1
Withdrawal by Subject             2
Arm/Group Title R115777 Therapy
Hide Arm/Group Description Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
<=18 years
0
   0.0%
Between 18 and 65 years
0
   0.0%
>=65 years
21
 100.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants
75
(66 to 84)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
10
  47.6%
Male
11
  52.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
1.Primary Outcome
Title Complete Remission (CR) Rate
Hide Description Complete Remission (CR) rate in Acute Myelogenous Leukemia (AML) patients prospectively selected for R115777R115777 (ZARNESTRA) treatment on the basis of a 2-gene signature (RASGRP1:APTX ratio) in bone marrow aspirates. AML Complete Remission: Bone marrow aspiration - Less than 5% leukemic blasts, Auer rods not detected; Peripheral blood counts - Absolute neutrophil count >/= 1,000/mm^3, Platelet count >/= 100,000/mm^3, Leukemic blasts not present; Blood-product transfusion independence; Absence of extramedullary leukemia.
Time Frame From first treatment through follow up period, an expected average of 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All evaluable participants
Arm/Group Title R115777 Therapy
Hide Arm/Group Description:
Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: percentage of participants
11
2.Secondary Outcome
Title Median Overall Survival (OS)
Hide Description Overall survival is calculated from the first day of R115777 treatment and lasts until the date of death recorded on the case report form (CRF).
Time Frame From first treatment through follow up period, an expected average of 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All evaluable participants
Arm/Group Title R115777 Therapy
Hide Arm/Group Description:
Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Overall Number of Participants Analyzed 18
Median (95% Confidence Interval)
Unit of Measure: months
6.6 [1] 
(4.2 to NA)
[1]
Not Reached. Data is through through 12/2012. Updates will be submitted as appropriate.
3.Secondary Outcome
Title Median 1-Year Survival Rate
Hide Description Prior to the early discontinuation of the study (for not meeting the primary endpoint of at least 3 CR/CRi after 2 cycles), investigators had planned to calculate one year survival from Kaplan Meier estimates.
Time Frame 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Evaluable participants at planned study completion date
Arm/Group Title R115777 Therapy
Hide Arm/Group Description:
Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Number of Participants With Relapse Free Survival
Hide Description Relapse-free survival is calculated from the date of documentation of complete remission/morphologic complete remission with incomplete blood count recovery (CR/CRi) until disease relapse or death from any cause.
Time Frame 7 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All evaluable participants
Arm/Group Title R115777 Therapy
Hide Arm/Group Description:
Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: participants
2
Time Frame 19 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title R115777 Therapy
Hide Arm/Group Description Each participant will begin R115777 treatment with an orally dosed regimen of 300 mg twice a day (BID) for the first 21 consecutive days of a 28-day cycle.
All-Cause Mortality
R115777 Therapy
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
R115777 Therapy
Affected / at Risk (%) # Events
Total   7/21 (33.33%)    
Blood and lymphatic system disorders   
Febrile neutropenia  1  4/21 (19.05%)  5
Cardiac disorders   
Sinus tachycardia  1  1/21 (4.76%)  1
Gastrointestinal disorders   
Nausea  1  1/21 (4.76%)  2
Vomiting  1  1/21 (4.76%)  2
General disorders   
Chills  1  1/21 (4.76%)  1
Fatigue  1  1/21 (4.76%)  1
Fever  1  1/21 (4.76%)  1
General disorders and administration site conditions - Other  1  1/21 (4.76%)  1
Infections and infestations   
Infections and infestations - Other  1  1/21 (4.76%)  1
Lung infection  1  2/21 (9.52%)  3
Upper respiratory infection  1  1/21 (4.76%)  1
Injury, poisoning and procedural complications   
Fall  1  1/21 (4.76%)  1
Investigations   
Neutrophil count decreased  1  1/21 (4.76%)  1
Musculoskeletal and connective tissue disorders   
Generalized muscle weakness  1  1/21 (4.76%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE v4.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
R115777 Therapy
Affected / at Risk (%) # Events
Total   17/21 (80.95%)    
Blood and lymphatic system disorders   
Febrile neutropenia  1  8/21 (38.10%)  11
Anemia  1  4/21 (19.05%)  5
Gastrointestinal disorders   
Nausea  1  7/21 (33.33%)  10
Diarrhea  1  6/21 (28.57%)  9
Vomiting  1  6/21 (28.57%)  8
Constipation  1  3/21 (14.29%)  3
Mucositis - oral  1  2/21 (9.52%)  2
General disorders   
Fatigue  1  8/21 (38.10%)  10
General disorders and administration site conditions - Other  1  3/21 (14.29%)  3
Chills  1  2/21 (9.52%)  3
Edema - face  1  2/21 (9.52%)  3
Fever  1  2/21 (9.52%)  2
Non-cardiac chest pain  1  2/21 (9.52%)  3
Pain  1  2/21 (9.52%)  4
Infections and infestations   
Lung infection  1  4/21 (19.05%)  4
Sepsis  1  3/21 (14.29%)  3
Injury, poisoning and procedural complications   
Fall  1  3/21 (14.29%)  3
Bruising  1  2/21 (9.52%)  3
Investigations   
White blood cell decreased  1  7/21 (33.33%)  9
Platelet count decreased  1  5/21 (23.81%)  10
Neutrophil count decreased  1  2/21 (9.52%)  2
Metabolism and nutrition disorders   
Anorexia  1  7/21 (33.33%)  8
Hypokalemia  1  2/21 (9.52%)  4
Musculoskeletal and connective tissue disorders   
Back pain  1  2/21 (9.52%)  3
Pain in extremity  1  2/21 (9.52%)  3
Nervous system disorders   
Dizziness  1  4/21 (19.05%)  6
Headache  1  2/21 (9.52%)  2
Syncope  1  2/21 (9.52%)  2
Psychiatric disorders   
Confusion  1  3/21 (14.29%)  4
Insomnia  1  3/21 (14.29%)  5
Delirium  1  2/21 (9.52%)  2
Renal and urinary disorders   
Urinary frequency  1  3/21 (14.29%)  4
Urinary incontinence  1  2/21 (9.52%)  2
Respiratory, thoracic and mediastinal disorders   
Dyspnea  1  6/21 (28.57%)  6
Epistaxis  1  2/21 (9.52%)  2
Wheezing  1  2/21 (9.52%)  2
Skin and subcutaneous tissue disorders   
Rash maculo-papular  1  5/21 (23.81%)  7
Pruritus  1  4/21 (19.05%)  6
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE v4.0
Due to trial not meeting primary endpoint of at least 3 CR/CRi after 2 cycles, accrual was suspended. 1 year survival was not calculated, not relevant in the setting of a median survival of 6.6 months and with study not meeting its primary endpoint.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Jeffrey Lancet, M.D.
Organization: H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-6841
Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT01361464     History of Changes
Obsolete Identifiers: NCT01364038
Other Study ID Numbers: NCI-2011-02589
NCI-2011-02589 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
16572
CDR0000699713
8977 ( Other Identifier: Moffitt Cancer Center )
8977 ( Other Identifier: CTEP )
P30CA076292 ( U.S. NIH Grant/Contract )
N01CM00071 ( U.S. NIH Grant/Contract )
U01CA070095 ( U.S. NIH Grant/Contract )
N01CM00100 ( U.S. NIH Grant/Contract )
First Submitted: May 24, 2011
First Posted: May 26, 2011
Results First Submitted: November 15, 2013
Results First Posted: January 6, 2014
Last Update Posted: April 8, 2015