A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients With Sickle Cell Disease.
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
Sponsors
Source
Eli Lilly and Company
Oversight Info
Has Dmc
Yes
Brief Summary
The main purpose of the study is to evaluate the efficacy and safety of the study drug known
as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with
sickle cell disease. The study will also investigate reduction in daily pain in children who
have sickle cell disease.
Detailed Description
The submission database was validated for data reported through the data cutoff date for the
submission database lock (SDBL). The SDBL data cutoff was 17 July 2015 for all participants
except for 2 in the youngest age group, for whom the SDBL data cutoff occurred on 08 August
2015. The data cutoff date for SDBL corresponds to the primary completion date for the study.
The SDBL occurred on 31 August 2015.
The study was stopped following SDBL and review of the topline information indicated that the
primary and secondary efficacy endpoints were not met. Subsequently, the Sponsor requested
that participants discontinue study drug immediately and that discontinuation visits for all
active study participants be conducted as soon as feasible.
After the data cutoff date for SDBL, the Sponsor continued to collect safety data through the
final participants contact; some additional efficacy data were collected through the final
visit. The last patient visit (LPV) occurred on 17 December 2015, which corresponds to the
study completion date and led to the planned supplemental database lock (PSDBL) on 22 January
2016. This supplemental data base was originally designed to capture additional blinded and
randomized information to enhance safety data for labeling should the study have been
positive.
The safety information contained in this record reflects the entire safety information and
reflects the information from the supplemental data base lock in January of 2016. The
efficacy information contained in this record reflects the information collected through
primary completion date in the submission database. Primary analyses of the major efficacy
objectives were repeated using the entire double-blind period data from the PSDBL and did not
change the original conclusions and were consistent with the results from the original
efficacy analyses included in the SDBL.
Overall Status
Terminated
Start Date
2013-04-01
Completion Date
2015-12-01
Primary Completion Date
2015-07-01
Phase
Phase 3
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
Number of Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC) |
Randomization through 24 Months |
Secondary Outcome
Measure |
Time Frame |
Monthly Rate of Days With Pain |
Randomization through 9 Months |
Monthly Mean in Faces Pain Scale-Revised Score |
Randomization through 9 Months |
Number of Painful Crisis Events Per Participant Per Year (Rate of Painful Crisis) |
Randomization through 24 Months |
Number of Hospitalizations for VOC Per Participant Per Year (Rate of Hospitalizations) |
Randomization through 24 Months |
Number of Acute Chest Syndrome Per Participant Per Year (Rate of Acute Chest Syndrome) |
Randomization through 24 Months |
Number of Red Blood Cell (RBC) Transfusions Due to Sickle Cell Disease (SCD) Per Participant Per Year (Rate of RBC Transfusions) |
Randomization through 24 Months |
Monthly Rate of Days of Analgesic Use |
Randomization through 9 Months |
Quarterly Rate of School Absence Due to Sickle Cell Pain |
Randomization through 9 Months |
Time to First Transient Ischemic Attack (TIA)/Ischemic Stroke |
Randomization through 24 Months |
Number of Days Hospitalized for VOC |
Randomization through 24 Months |
Time From Randomization to First and Second VOC |
Randomization to First VOC and Second VOC respectively (up to 24 Months) |
Percentage of Participants With Hemorrhagic Events Requiring Medical Intervention |
First Dose through 24 Months |
Enrollment
341
Condition
Intervention
Eligibility
Criteria
Inclusion Criteria:
- Have SCD [homozygous sickle cell (HbSS) or hemoglobin (HbS) Beta^0 thalassemia]
- Are participants with SCD who have had ≥2 episodes of vaso-occlusive crisis (VOC) in
the past year
- Have a body weight ≥19 kilograms (kg) and are ≥2 and <18 years of age, inclusive at
the time of screening
- If participants are ≥2 and ≤16 years of age, must have had a transcranial Doppler
within the last year
Exclusion Criteria:
- History of: transient ischemic attack (TIA)/ ischemic or hemorrhagic stroke, severe
head trauma, intracranial hemorrhage, intracranial neoplasm, arteriovenous
malformation, or aneurysm
- History of abnormal or conditional [velocity in middle or anterior cerebral, or
internal carotid artery ≥170 centimeter per second (cm/sec)] transcranial Doppler
within the last year
- History of, or are undergoing treatment with, chronic red blood cell (RBC) transfusion
therapy
- Are at an increased risk for bleeding complications
- Are receiving chronic treatment with nonsteroidal anti-inflammatory drug (NSAID)s and
cannot be switched to another analgesic
Gender
All
Minimum Age
2 Years
Maximum Age
17 Years
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon-Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) |
Study Director |
Eli Lilly and Company |
Location
Facility |
Children's Hospital of Oakland Oakland California 94609 United States |
Stanford Univ Medical Center Palo Alto California 94304 United States |
Connecticut Children's Medical Center Hartford Connecticut 06106 United States |
Howard University Hospital Washington District of Columbia 20060 United States |
Emory University Atlanta Georgia 30322 United States |
Memorial Health University Medical Center Savannah Georgia 31404 United States |
Ann & Robert H Lurie Children's Hospital of Chicago Chicago Illinois 60611 United States |
Boston Children's Hospital Boston Massachusetts 02115 United States |
Childrens Hospital of Michigan Detroit Michigan 48201 United States |
Children's Mercy Hospital Kansas City Missouri 64108 United States |
Albert Einstein College of Medicine Bronx New York 10467 United States |
University of NC at Chapel Hill School of Medicine Chapel Hill North Carolina 27599 United States |
Childrens Hospital Medical Center Cincinnati Ohio 45229 United States |
Rainbow Babies and Children's Hospital Cleveland Ohio 44106 United States |
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19134 United States |
St Christophers Hospital For Children Philadelphia Pennsylvania 19134 United States |
Childrens Hospital of Pittsburgh Pittsburgh Pennsylvania 15224 United States |
Medical University of South Carolina Charleston South Carolina 29425 United States |
St Jude Childrens Research Hospital Memphis Tennessee 38105 United States |
Mary Bridge Children's Hospital and Health Center Tacoma Washington 98405 United States |
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician. Brussel 1200 Belgium |
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For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician. Jeddah 21859 Saudi Arabia |
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For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician. Abu Dhabi United Arab Emirates |
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician. Tooting London SW17 0QT United Kingdom |
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician. London SE1 7EH United Kingdom |
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Location Countries
Country
Belgium
Brazil
Canada
Egypt
Ghana
Italy
Kenya
Lebanon
Oman
Saudi Arabia
Turkey
United Arab Emirates
United Kingdom
United States
Verification Date
2019-09-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Has Expanded Access
No
Condition Browse
Secondary Id
H7T-MC-TADO
2012-003837-41
Number Of Arms
2
Intervention Browse
Mesh Term
Prasugrel Hydrochloride
Arm Group
Arm Group Label
Prasugrel
Arm Group Type
Experimental
Description
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.
Arm Group Label
Placebo
Arm Group Type
Placebo Comparator
Description
Participants in this treatment group will receive daily orally administered placebo and will follow visit schedule identical to that in the active treatment group.
Firstreceived Results Date
N/A
Why Stopped
The study is being terminated for lack of efficacy.
Removed Countries
Country
France
Netherlands
Patient Data
Sharing Ipd
Yes
Ipd Description
Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
Ipd Time Frame
Data are available 6 months after the primary publication and approval of the indication studied in the US and EU, whichever is later. Data will be indefinitely available for requesting.
Ipd Access Criteria
A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.
Ipd Url
https://vivli.org/
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)
Study First Submitted
February 14, 2013
Study First Submitted Qc
February 14, 2013
Study First Posted
February 18, 2013
Last Update Submitted
September 10, 2019
Last Update Submitted Qc
September 10, 2019
Last Update Posted
September 25, 2019
Results First Submitted
June 15, 2016
Results First Submitted Qc
June 15, 2016
Results First Posted
July 27, 2016
ClinicalTrials.gov processed this data on December 11, 2019
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.