Eltrombopag vs Standard Treatment for ITP in Children

Recruiting at 26 trial locations

Overseen By Amanda Grimes, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Baylor College of Medicine

Must be taking: IVIg, Steroids, Anti-D

Must not be taking: Anticoagulants, Anti-platelets

Disqualifiers: Severe bleeding, HIV, Hepatitis C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

122 Participants Needed

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either eltrombopag or standard therapy for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

40 weeks

Open-label extension

Participants may continue eltrombopag treatment for up to 1 year based on response

1 year

Treatment Details

Interventions

• Eltrombopag

Trial Overview The 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

Group I: Eltrombopag Experimental Treatment 1 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 therapy Active Control 3 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:

• Severe aplastic anemia
• Chronic immune thrombocytopenia
• Thrombocytopenia in patients with chronic hepatitis C

Approved in European Union as Revolade for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in Canada as Promacta for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in Japan as Revolade for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in China as Promacta for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials

1,044

Recruited

6,031,000+

Boston Children's Hospital

Collaborator

Trials

801

Recruited

5,584,000+

University of California, San Francisco

Collaborator

Trials

2,636

Recruited

19,080,000+