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A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia

This study has been terminated.
(Despite considerable efforts to boost recruitment during the final year of the study, no new patients were enrolled.)
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00866281
First received: March 19, 2009
Last updated: November 18, 2015
Last verified: November 2015
Results First Received: September 8, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Intervention: Drug: midostaurin

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was conducted at 8 centers in 5 countries.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 22 participants were enrolled in the study.

Reporting Groups
  Description
Cohort 1: Midostaurin (30 Milligrams/Meters^2) Participants received bodyweight and body surface area (BSA) stratified dose of midostaurin 30 mg/m^2 twice daily (bid) through oral route. The total daily dose in 30 mg/m^2 bid cohort was 60 mg/m^2.
Cohort 2: Midostaurin (60 mg/m^2) Participants received bodyweight and BSA stratified dose of midostaurin 60 mg/m^2 bid through oral route. The total daily dose in 60 mg/m^2 bid cohort was 120 mg/m^2.

Participant Flow:   Overall Study
    Cohort 1: Midostaurin (30 Milligrams/Meters^2)   Cohort 2: Midostaurin (60 mg/m^2)
STARTED   7   15 
COMPLETED   0   0 
NOT COMPLETED   7   15 
Adverse Event                1                0 
Withdrawal by participants                3                0 
Disease progression                3                11 
New cancer therapy                0                4 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The analysis was performed in full analysis set (FAS) population, defined as all participants to whom study treatment was assigned.

Reporting Groups
  Description
Cohort 1: Midostaurin (30 Milligrams/Meters^2) Participants received body-weight and BSA stratified dose of midostaurin 30 mg/m^2 bid through oral route. The total daily dose in 30 mg/m^2 bid cohort was 60 mg/m^2.
Cohort 2: Midostaurin (60 mg/m^2) Participants received body-weight and BSA stratified dose of midostaurin 60 mg/m^2 bid through oral route. The total daily dose in 60 mg/m^2 bid cohort was 120 mg/m^2.
Total Total of all reporting groups

Baseline Measures
   Cohort 1: Midostaurin (30 Milligrams/Meters^2)   Cohort 2: Midostaurin (60 mg/m^2)   Total 
Overall Participants Analyzed 
[Units: Participants]
 7   15   22 
Age 
[Units: Years]
Mean (Standard Deviation)
 9.65  (7.479)   6.50  (6.903)   7.50  (7.070) 
Age, Customized 
[Units: Participants]
     
Infants and toddlers (28 days-23 months)   2   9   11 
Children (2-­11 years)   1   1   2 
Adolescents (12-­17 years)   4   5   9 
Gender 
[Units: Participants]
     
Female   5   10   15 
Male   2   5   7 


  Outcome Measures
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1.  Primary:   Maximum Tolerated Dose (MTD) of Midostaurin- Posterior Probability of DLT   [ Time Frame: Baseline, End of dose escalation phase (6 months) ]

2.  Secondary:   Percentage of Participants With Best Overall Response by Indication   [ Time Frame: Baseline, Day 15 (Day 1 of Cycle 2), Day 22 (Day 8 of Cycle 2), Day 29(Day 1 of Cycle 9), End of treatment (up to 24 months after last dose or until death whichever occurred first) ]

3.  Secondary:   Time to Response With Midostaurin   [ Time Frame: Baseline, End of treatment (up to 24 months after last dose or until death whichever occurred first) ]

4.  Secondary:   Overall Survival With Midostaurin   [ Time Frame: Baseline, End of treatment (up to 24 months after last dose or until death whichever occurred first) ]

5.  Secondary:   Plasma Concentrations of Midostaurin and Its Metabolites CGP52421 and CGP62221   [ Time Frame: Day 1, Day 5, Day 7, Day 15 (Day 1 of Cycle 2), Day 29 (Day 1 of Cycle 3) ]

6.  Secondary:   Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment Related AEs or SAEs and Death During the Study   [ Time Frame: Baseline (start of study treatment) up to End of treatment (up to 24 months after last dose or until death whichever occurred first) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The study was terminated early since despite considerable efforts to boost recruitment, no new participants were enrolled in the final year of this study.


  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00866281     History of Changes
Other Study ID Numbers: CPKC412A2114
2008-006931-11 ( EudraCT Number )
Study First Received: March 19, 2009
Results First Received: September 8, 2015
Last Updated: November 18, 2015
Health Authority: United States: Food and Drug Administration
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: Ministry of Health
Netherlands: Ministry of Health, Welfare and Sport
Sweden: Medical Products Agency