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Efficacy and Safety Dose Finding Study of Givinostat to Treat Polyarticular Course Juvenile Idiopathic Arthritis

This study has been terminated.
(The primary reason for the decision to discontinue the study is lack of enrolment; this decision is not related to any tolerability concerns with Givinostat)
Sponsor:
Information provided by (Responsible Party):
Italfarmaco
ClinicalTrials.gov Identifier:
NCT01261624
First received: December 15, 2010
Last updated: March 11, 2014
Last verified: March 2014
Results First Received: January 14, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Polyarticular Course Juvenile Idiopathic Arthritis
Intervention: Drug: Givinostat

  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
Recruitment period: October 2010 - December 2011. The study was conducted by nine Investigators in five countries across Europe; three Investigators in Italy, two Investigators each in Romania and Serbia, and one Investigator each in Czech Republic and Slovenia

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Low Dose Treatment Cohort (LDTC): 0.50 mg/kg Twice Daily (BID) Patients (pts) received the dose of 0.50 mg/kg BID for 12 weeks in fed condition
High Dose Treatment Cohort (HDTC): 0.75 mg/kg BID Patients received the dose of 0.75 mg/kg BID for 12 weeks in fed conditions

Participant Flow for 2 periods

Period 1:   Week 0 to Week 12
    Low Dose Treatment Cohort (LDTC): 0.50 mg/kg Twice Daily (BID)     High Dose Treatment Cohort (HDTC): 0.75 mg/kg BID  
STARTED     10     6  
COMPLETED     9     3  
NOT COMPLETED     1     3  
Disease Progression                 1                 1  
Insufficient Response                 0                 2  

Period 2:   Week 13 to Week 24
    Low Dose Treatment Cohort (LDTC): 0.50 mg/kg Twice Daily (BID)     High Dose Treatment Cohort (HDTC): 0.75 mg/kg BID  
STARTED     1 [1]   11 [2]
COMPLETED     1     10  
NOT COMPLETED     0     1  
Insufficient Response                 0                 1  
[1] Of 9 pts from LDTC: 5 non-responders switched to HDTC; 4 pts responded, but 3 switched to HDTC
[2] Of 6 pts of HDTC: 3 non-responders went out of study, 3 continued. 8 swithched from LDTC to HDTC



  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.
No text entered.

Reporting Groups
  Description
Low Dose Treatment Cohort (LDTC): 0.50 mg/kg BID Patient received the dose of 0.50 mg/kg for 12 weeks in fed condition
High Dose Treatment Cohort (HDTC): 0.75 mg/kg BID Patient received the dose of 0.75 mg/kg for 12 weeks in fed condition
Total Total of all reporting groups

Baseline Measures
    Low Dose Treatment Cohort (LDTC): 0.50 mg/kg BID     High Dose Treatment Cohort (HDTC): 0.75 mg/kg BID     Total  
Number of Participants  
[units: participants]
  10     6     16  
Age  
[units: years]
Mean ± Standard Deviation
  11.7  ± 5.5     9.7  ± 5.7     10.9  ± 5.4  
Gender  
[units: participants]
     
Female     8     6     14  
Male     2     0     2  
Race (NIH/OMB)  
[units: participants]
     
American Indian or Alaska Native     0     0     0  
Asian     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0  
Black or African American     0     0     0  
White     10     6     16  
More than one race     0     0     0  
Unknown or Not Reported     0     0     0  
Region of Enrollment  
[units: participants]
     
Serbia     5     1     6  
Czech Republic     1     1     2  
Slovenia     1     1     2  
Romania     1     0     1  
Italy     2     3     5  
Body Weight  
[units: kg]
Mean ± Standard Deviation
  37.36  ± 15.40     37.92  ± 18.59     37.57  ± 16.05  
Height  
[units: m]
Mean ± Standard Deviation
  1.388  ± 0.204     1.365  ± 0.277     1.379  ± 0.225  



  Outcome Measures
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1.  Primary:   ACR Pediatric Response Level (ACRPRL) 30 After 12 Weeks of Treatment   [ Time Frame: 12 weeks of treatment ]

2.  Secondary:   ACR Pediatric Response Level (ACR 50, 70, 90 and 100) at Week 12   [ Time Frame: at week12 ]


  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
No text entered.


  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 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.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Clinical R&D Director
Organization: Italfarmaco S.p.A.
phone: 026443 ext 2584
e-mail: p.bettica@italfarmaco.com


No publications provided


Responsible Party: Italfarmaco
ClinicalTrials.gov Identifier: NCT01261624     History of Changes
Other Study ID Numbers: DSC/08/2357/36
Study First Received: December 15, 2010
Results First Received: January 14, 2014
Last Updated: March 11, 2014
Health Authority: Czech Republic: State Institute for Drug Control
Slovenia: Javna agencija Republike Slovenije za zdravila in medicinske pripomočke
Sweden: Medical Products Agency
Spain: Ministerio de Sanidad y Consumo-Agencia Española de Medicamentos y Productos Sanitarios Republic of Serbia: Medicines and Medical Devices Agency of Serbia
Romania: National Medicine and Medical Devices Agency
Belgium: Agence Fédérale des Médicaments et des Produits de Santé (AFMPS)