Protocol

TADO: A Phase 3 Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease

Diseases

Hematologic Disorders : Sickle cell disease

Description

This is a Phase III study. Prasugrel is considered investigational for sickle cell disease. Investigational means that this drug is not approved by the United States Food and Drug Administration (FDA) for use in sickle cell disease however the FDA allows us to use this drug for research. Prasugrel stops platelets from becoming sticky and helps prevent blood clots. Studies in adults have shown prasugrel lessens the level of pain during pain crises and this study is being done to see if prasugrel can do the same in children.

The current standard of care is clinical management with or without Hydroxyurea.

Participants will be assigned into a treatment group through the randomization process. The probability of receiving any one of the treatments is 1 in 2, the same as flipping a coin.

Participants will be randomized into one of two groups. One group will receive the study drug prasugrel and one group will receive a placebo (sugar pill). Neither the participant nor the study team will know what drug is given (double-blind) (although, if the doctor needs to find out he/she can do so). Whether a participant receives prasugrel or placebo (sugar pill) will be determined by chance.


Objectives

The primary objective of this study is to assess the efficacy of prasugrel compared to placebo in pediatric patients with sickle-cell disease (SCD) as measured by reduction in the rate of vaso-occlusive crisis (VOC), which is a composite endpoint of painful crisis or acute chest syndrome. A painful crisis is defined as an onset of moderate to severe pain that lasts at least 2 hours for which there is no explanation other than vaso-occlusion and which requires therapy with oral or parenteral opioids, ketorolac, or other analgesics prescribed by a health care provider in a medical setting such as a hospital, clinic, emergency room visit, or telephone management. Acute chest syndrome is defined as an acute illness characterized by fever and/or respiratory symptoms, accompanied by a new pulmonary infiltrate on a chest X-ray.


Eligibility criteria, among others, include:

For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE (1-866-278-5833).


Contact

Jeremie Estepp, MD
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105 USA
Voice: 1-888-226-4343 or 901-595-4055
FAX: 901-595-4011

Referring or consulting physicians only: protocolinfo@stjude.org

For all other inquiries about St. Jude Children's Research Hospital studies: info@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.