Clinical trial NCT01476696: A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
Official title: An Open-Label, Dose-Ranging Study of Prasugrel in Pediatric Patients With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease
pediatrics
SCD
children
child
kids
pain crisis
sickle cell anemia
sickle cell pain
Lead sponsor: Eli Lilly and Company
Sponsor class: INDUSTRY
Collaborators: name: Daiichi Sankyo
First posted: 2011-11-22
Enrollment: 33
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).


Eligibility criteria: Inclusion Criteria:

* Are male or female with SCD \[(homozygous sickle cell (HbSS) and hemoglobin S beta \^0 thalassemia (HbS β\^0 thalassemia)\]
* Have a body weight ≥12 kilograms (kg) and are ≥2 to \<18 years of age at the time of screening
* If participants are ≥2 and ≤16 years of age, have had a transcranial Doppler within the last year
* Participants on hydroxyurea must be on a stable dose for the 60 days prior to enrollment without signs of hematologic toxicity at screening
* Have a legal representative that is in competent mental condition to provide written informed consent on behalf of the study participant before entering the study. The child may be required to give documented assent, if required by local regulations.
* If sexually active, has a negative pregnancy test at screening (if female) and agrees to use a reliable method of birth control during the study (for both males and females)

Exclusion Criteria:

* Known to have hemoglobin C sickle cell (HbSC) or hemoglobin S beta \^plus thalassemia (HbS β\^+ thalassemia) genotypes
* Vaso-occlusive crisis (VOC) requiring medical attention within 15 days prior to screening
* Have a concomitant medical illness (for example, terminal malignancy) that in the opinion of the investigator is associated with reduced survival
* Hepatic dysfunction characterized by alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN)
* Renal dysfunction requiring chronic dialysis or creatinine ≥ 1.5 milligrams per deciliter (mg/dL)
* Contraindication for antiplatelet therapy
* History of intolerance or allergy to approved thienopyridines (clopidogrel, ticlopidine, or prasugrel)
* Participants with a hematocrit \<18%
* History of abnormal or conditional transcranial Doppler \[velocity in middle cerebral or carotid artery ≥170 centimeters per second (cm/sec)\] within the last year
* Any history of bleeding diathesis
* Any history of renal papillary necrosis
* Active internal bleeding
* History of spontaneous gastrointestinal bleeding
* Gross hematuria or \> 300 red blood cells (RBC)/high-powered field (HPF) on urinalysis at the time of screening
* Any history of vitreous hemorrhage
* Prior history of hemorrhagic or ischemic stroke, a transient ischemic attack (TIA), or other intracranial hemorrhage
* Have clinical findings, in the judgment of the investigator, associated with an increased risk of bleeding
* Platelet count \<100,000 per microliter (μl) of blood
* Have had recent surgery (within 30 days prior to screening) or are scheduled to undergo surgery within the next 60 days
* History of dysfunctional uteral bleeding, in the judgment of the investigator
* Treatment with packed RBC or whole blood transfusion therapy within 30 days prior to dosing
* Any nonsteroidal anti-inflammatory drug (NSAID) use within 5 days prior to screening
* Any aspirin, warfarin, thienopyridine, or other antiplatelet medication use within 10 days prior to dosing
* Anticipated use of aspirin, warfarin, thienopyridine, or other antiplatelet medication during the study period

Primary outcomes:
measure: Pharmacokinetics: Area Under the Concentration-Time Curve (AUC) of Prasugrel Active Metabolite (Pras-AM)
timeFrame: Parts A and B: 0.5, 1, 1.5, 2, 4 hours postdose
measure: Percentage of Platelet Inhibition as Measured by VerifyNow™P2Y12 (VN)
timeFrame: Part A: 4 hours postdose and Part B: at steady state (14 ± 4 days after the start of each new dosage)
Secondary outcomes:
measure: Pharmacokinetics: Area Under the Concentration-Time Curve (AUC) of Prasugrel Inactive Metabolite
timeFrame: Part A: 0.5, 1, 1.5, 2, 4 hours postdose
measure: Number of Participants With Pain
timeFrame: Part B: Baseline and Day14 ± 4 days postdose in each dosing period
measure: Number of Participants With Hemorrhagic Events Requiring Medical Intervention
timeFrame: Part B: Baseline up to Day 36
Other outcomes:
Locations:
city: Oakland
country: United States
facility: 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.
state: California
city: Washington
country: United States
facility: 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.
state: District of Columbia
city: Chicago
country: United States
facility: 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.
state: Illinois
city: New Orleans
country: United States
facility: 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.
state: Louisiana
city: Boston
country: United States
facility: 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.
state: Massachusetts
city: St Louis
country: United States
facility: 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.
state: Missouri
city: Chapel Hill
country: United States
facility: 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.
state: North Carolina
city: Cincinnati
country: United States
facility: 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.
state: Ohio
city: Pittsburgh
country: United States
facility: 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.
state: Pennsylvania
Study officials:
affiliation: Eli Lilly and Company
name: 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)
role: STUDY_DIRECTOR
References: