Eltrombopag vs Standard Treatment for ITP in Children

Recruiting in Palo Alto (17 mi)

+26 other locations

Overseen byAmanda Grimes, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Baylor College of Medicine

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Approved in 5 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests eltrombopag, a drug that helps make more platelets, in children with a specific blood condition. The goal is to see if it works better than standard treatments.

Research Team

Amanda Grimes, MD

Principal Investigator

Baylor College of Medicine - Texas Children's Hospital

Eligibility Criteria

This trial is for children aged 1 to less than 18 with newly diagnosed ITP, a condition causing low platelet count. They must not have received prior treatment or have failed standard treatments like IVIG, steroids, or Anti-D. Participants need pharmacologic treatment and should be able to return for lab studies.

Inclusion Criteria

I am seeking treatment for the first time or after a previous treatment didn't work.

I was diagnosed with ITP within the last 10 days and haven't been treated, or my initial ITP treatments didn't work.

My doctor says I need medication for my condition.

See 9 more

Exclusion Criteria

My heart does not function properly.

You have a history of using drugs or alcohol in a harmful way.

You have had a bad reaction to eltrombopag or any of its ingredients in the past.

See 18 more

Treatment Details

Interventions

Eltrombopag (Thrombopoietin Receptor Agonist)

Trial OverviewThe study compares Eltrombopag against three standard therapies (IVIG, Rho(D) Immune Globulin, Steroids) in treating ITP in children. It's an open label phase 3 trial where participants are randomly assigned to receive either the new intervention or one of the standard options.

Participant Groups

2Treatment groups

Experimental Treatment

Active Control

Group I: EltrombopagExperimental Treatment1 Intervention

Patients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response.

Group II: Standard first-line therapyActive Control3 Interventions

Subjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm. Standard therapy will be administered as commercially available drug. Investigator may choose amongst the following: * IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) * Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days * Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)

Eltrombopag is already approved in United States, European Union, Canada, Japan, China for the following indications:

🇺🇸 Approved in United States as Promacta for: