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A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)

This study has been terminated.
(The study is being terminated for lack of efficacy.)
Sponsor:
Collaborator:
Daiichi Sankyo Inc.
Information provided by (Responsible Party):
Eli Lilly and Company
ClinicalTrials.gov Identifier:
NCT01794000
First received: February 14, 2013
Last updated: June 15, 2016
Last verified: June 2016
Results First Received: June 15, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Sickle Cell Disease
Interventions: Drug: Prasugrel
Drug: Placebo

  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
No text entered.

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
Prasugrel Participants (Pts.) will be titrated from initial daily dose of 0.08 milligram per kilogram (mg/kg) of orally administered prasugrel monotherapy at randomization to a dose that will achieve a P2Y12 reaction units (PRU) level of 231 to 136, as measured by VerifyNow instrument. This corresponds to a range of platelet inhibition of approximately 30% to 60%. The maximum possible dose allowed is 0.12 mg/kg daily, not to exceed 10 mg daily.
Placebo Participants in this treatment group will receive daily orally administered placebo and will follow visit schedule identical to that in the active treatment group.

Participant Flow for 2 periods

Period 1:   Double-Blind Phase (DBP)
    Prasugrel   Placebo
STARTED   171   170 
Received at Least One Dose of Drug   170   170 
Discontinued During Double Blind Phase   169   166 
COMPLETED   2   4 
NOT COMPLETED   169   166 
Adverse Event                5                2 
Death                1                2 
Entry Criteria Not Met                2                0 
Parent/Caregiver Decision                5                2 
Physician Decision                2                0 
Sponsor Decision                148                149 
Withdrawal by Subject                6                11 

Period 2:   Open-Label Extension Phase (OLE)
    Prasugrel   Placebo
STARTED   3 [1]   0 [2] 
COMPLETED   0   0 
NOT COMPLETED   3   0 
Sponsor Decision                3                0 
[1] Pts. who had not permanently discontinued study drug and concluded DBP were eligible to enter OLE
[2] Placebo arm was not applicable to Open-Label Extension Phase



  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.
All randomized participants.

Reporting Groups
  Description
Prasugrel Participants will be titrated from initial daily dose of 0.08 milligram per kilogram (mg/kg) of orally administered prasugrel monotherapy at randomization to a dose that will achieve a P2Y12 reaction units (PRU) level of 231 to 136, as measured by VerifyNow instrument. This corresponds to a range of platelet inhibition of approximately 30% to 60%. The maximum possible dose allowed is 0.12 mg/kg daily, not to exceed 10 mg daily.
Placebo Participants in this treatment group will receive daily orally administered placebo and will follow visit schedule identical to that in the active treatment group.
Total Total of all reporting groups

Baseline Measures
   Prasugrel   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 171   170   341 
Age 
[Units: Years]
Mean (Standard Deviation)
 10.606  (4.334)   10.580  (4.349)   10.593  (4.335) 
Gender 
[Units: Participants]
     
Female   87   86   173 
Male   84   84   168 
Ethnicity (NIH/OMB) 
[Units: Participants]
     
Hispanic or Latino   2   1   3 
Not Hispanic or Latino   94   98   192 
Unknown or Not Reported   75   71   146 
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   113   109   222 
White   58   58   116 
More than one race   0   2   2 
Unknown or Not Reported   0   1   1 
Region of Enrollment 
[Units: Participants]
     
United States   23   25   48 
Egypt   23   22   45 
United Kingdom   5   4   9 
Ghana   29   28   57 
Kenya   47   44   91 
Oman   2   4   6 
Lebanon   8   8   16 
Saudi Arabia   1   0   1 
Canada   4   4   8 
Turkey   20   22   42 
Belgium   1   2   3 
Brazil   1   0   1 
Italy   7   7   14 
Hydroxyurea Use at Baseline 
[Units: Participants]
     
Yes   77   76   153 
No   94   94   188 


  Outcome Measures
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1.  Primary:   Number of Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC)   [ Time Frame: Randomization through 24 Months ]

2.  Secondary:   Monthly Rate of Days With Pain   [ Time Frame: Randomization through 9 Months ]

3.  Secondary:   Monthly Mean in Faces Pain Scale-Revised Score   [ Time Frame: Randomization through 9 Months ]

4.  Secondary:   Number of Painful Crisis Events Per Participant Per Year (Rate of Painful Crisis)   [ Time Frame: Randomization through 24 Months ]

5.  Secondary:   Number of Hospitalizations for VOC Per Participant Per Year (Rate of Hospitalizations)   [ Time Frame: Randomization through 24 Months ]

6.  Secondary:   Number of Acute Chest Syndrome Per Participant Per Year (Rate of Acute Chest Syndrome)   [ Time Frame: Randomization through 24 Months ]

7.  Secondary:   Number of Red Blood Cell (RBC) Transfusions Due to Sickle Cell Disease (SCD) Per Participant Per Year (Rate of RBC Transfusions)   [ Time Frame: Randomization through 24 Months ]

8.  Secondary:   Monthly Rate of Days of Analgesic Use   [ Time Frame: Randomization through 9 Months ]

9.  Secondary:   Quarterly Rate of School Absence Due to Sickle Cell Pain   [ Time Frame: Randomization through 9 Months ]

10.  Secondary:   Time to First Transient Ischemic Attack (TIA)/Ischemic Stroke   [ Time Frame: Randomization through 24 Months ]

11.  Secondary:   Number of Days Hospitalized for VOC   [ Time Frame: Randomization through 24 Months ]

12.  Secondary:   Time From Randomization to First and Second VOC   [ Time Frame: Randomization to First VOC and Second VOC respectively (up to 24 Months) ]

13.  Secondary:   Percentage of Participants With Hemorrhagic Events Requiring Medical Intervention   [ Time Frame: First Dose through 24 Months ]


  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 stopped following Submission Database Lock, the topline information indicated that the primary and secondary efficacy endpoints were not met.


  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 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: Chief Medical Officer
Organization: Eli Lilly and Company
phone: 800-545-5979


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT01794000     History of Changes
Other Study ID Numbers: 13038
H7T-MC-TADO ( Other Identifier: Eli Lilly and Company )
2012-003837-41 ( EudraCT Number )
Study First Received: February 14, 2013
Results First Received: June 15, 2016
Last Updated: June 15, 2016
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
Brazil: National Health Surveillance Agency
Canada: Health Canada
Egypt: Ministry of Health and Population
Ghana : Food and Drugs Board
Italy: Ethics Committee
Kenya: Pharmacy and Poisons Board
Lebanon: Ministry of Public Health
Oman: Ministry of Health, Sultanate of Oman
Saudi Arabia: Ministry of Health
Turkey: Ministry of Health
United Arab Emirates: Drug Control Department - Medicines and Pharmacy Control - Ministry of Health
United Arab Emirates: General Authority for Health Services for Abu Dhabi
United Kingdom: Medicines and Healthcare Products Regulatory Agency