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

NCT ID: NCT01794000

Last Updated: 2019-09-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 341 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-04-30
• Study Completion Date: 2015-12-31

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.

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

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.

Group Type: EXPERIMENTAL

Prasugrel

Intervention Type: DRUG

Administered orally

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Administered orally

Interventions

Prasugrel

Administered orally

Intervention Type: DRUG

Placebo

Administered orally

Intervention Type: DRUG

Other Intervention Names

LY640315; Effient; Efient

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Daiichi Sankyo

INDUSTRY

Sponsor Role: collaborator

Eli Lilly and Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

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

Eli Lilly and Company

Locations

Children's Hospital of Oakland

Oakland, California, United States

Stanford Univ Medical Center

Palo Alto, California, United States

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Howard University Hospital

Washington D.C., District of Columbia, United States

Emory University

Atlanta, Georgia, United States

Memorial Health University Medical Center

Savannah, Georgia, United States

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Childrens Hospital of Michigan

Detroit, Michigan, United States

Children's Mercy Hospital

Kansas City, Missouri, United States

Albert Einstein College of Medicine

The Bronx, New York, United States

University of NC at Chapel Hill School of Medicine

Chapel Hill, North Carolina, United States

Childrens Hospital Medical Center

Cincinnati, Ohio, United States

Rainbow Babies and Children's Hospital

Cleveland, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

St Christophers Hospital For Children

Philadelphia, Pennsylvania, United States

Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

St Jude Childrens Research Hospital

Memphis, Tennessee, United States

Mary Bridge Children's Hospital and Health Center

Tacoma, Washington, 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.

Brussels, , Belgium

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Montegnée, , Belgium

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Rio de Janeiro, , Brazil

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Montreal, Quebec, Canada

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Al Fayyum, , Egypt

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Al Mansurah, , Egypt

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Alexandria, , Egypt

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Cairo, , Egypt

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Ismailia, , Egypt

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Zagazig, , Egypt

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Agogo, , Ghana

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Korle Bu, , Ghana

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Genova, , Italy

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Modena, , Italy

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Monza, , Italy

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Padua, , Italy

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Verona, , Italy

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Busia, , Kenya

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Kisumu, , Kenya

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Kombewa, , Kenya

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Nairobi, , Kenya

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Beirut, , Lebanon

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Muscat, , Oman

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Jeddah, , Saudi Arabia

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Balcali Adana, , Turkey (Türkiye)

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Mersin, , Turkey (Türkiye)

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Abu Dhabi, , United Arab Emirates

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Tooting, London, United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

Countries

United States; Belgium; Brazil; Canada; Egypt; Ghana; Italy; Kenya; Lebanon; Oman; Saudi Arabia; Turkey (Türkiye); United Arab Emirates; United Kingdom

References

Heeney MM, Hoppe CC, Abboud MR, Inusa B, Kanter J, Ogutu B, Brown PB, Heath LE, Jakubowski JA, Zhou C, Zamoryakhin D, Agbenyega T, Colombatti R, Hassab HM, Nduba VN, Oyieko JN, Robitaille N, Segbefia CI, Rees DC; DOVE Investigators. A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 Feb 18;374(7):625-35. doi: 10.1056/NEJMoa1512021. Epub 2015 Dec 8.

Reference Type: DERIVED

PMID: 26644172 (View on PubMed)

Related Links

http://www.nejm.org/doi/full/10.1056/NEJMoa1512021#t=articleTop

A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events

Other Identifiers

H7T-MC-TADO

Identifier Type: OTHER

Identifier Source: secondary_id

2012-003837-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

13038

Identifier Type: -

Identifier Source: org_study_id