Eltrombopag for Aplastic Anemia

Recruiting in Palo Alto (17 mi)

+33 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Novartis Pharmaceuticals

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

This trial is for pediatric patients aged 1 to <18 with severe aplastic anemia (SAA) or recurrent aplastic anemia who haven't had a transplant option. They must not have certain bone marrow failure syndromes, prior immunosuppressive therapy, or active infections. Participants will receive eltrombopag alongside standard immunosuppressive treatments.

Inclusion Criteria

I agree to be treated with hATG, CsA, and eltrombopag together.

I have never been treated with IST.

I was previously diagnosed with severe aplastic anemia.

See 13 more

Exclusion Criteria

My tests show genetic abnormalities in my cells.

I have a known genetic condition affecting my bone marrow.

I have been diagnosed with Fanconi anemia.

See 11 more

Treatment Details

Interventions

CsA (Immunosuppressant)
Eltrombopag (Thrombopoietin Receptor Agonist)
hATG (Immunosuppressant)

Trial OverviewThe study tests how the body processes the drug eltrombopag when given with hATG and CsA in children with SAA or recurrent AA. It's an open-label, multi-center trial where doses of eltrombopag are increased within each patient to understand its pharmacokinetics.

Participant Groups

3Treatment groups

Experimental Treatment

Group I: Cohort BExperimental Treatment3 Interventions

previously untreated SAA, hATG (ATGAM®), CsA and eltrombopag begin on Day 1 and all patients will be treated with the same regimen

Group II: Cohort A (option 2)Experimental Treatment2 Interventions

CsA and eltrombopag begin on Day 1.

Group III: Cohort A (Option 1)Experimental Treatment3 Interventions

Regimen 1: hATG (ATGAM®), CsA and eltrombopag begin on Day 1.