Eltrombopag for Aplastic Anemia

Recruiting at 33 trial locations

Overseen By Novartis Pharmaceuticals

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must be taking: Eltrombopag, Immunosuppressive therapy

Must not be taking: Thrombopoietin receptor agonists

Disqualifiers: Fanconi anemia, Myelodysplastic syndrome, Malignancy, HIV, Hepatitis B or C, others

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

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

Compensation: Varies

Number of Visits: 1

Duration: 26 weeks

51 Participants Needed

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1 week

1 visit (in-person)

Treatment

Participants receive eltrombopag in combination with immunosuppressive therapy for 26 weeks

26 weeks

Regular visits for dose assessment and modification

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Periodic visits for monitoring

Long-term Follow-up

Participants are monitored annually for up to 3 years for long-term safety and effectiveness

3 years

Annual visits

Treatment Details

Interventions

• CsA
• Eltrombopag
• hATG

Trial Overview The 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

Group I: Cohort B Experimental Treatment 3 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 Treatment 2 Interventions

CsA and eltrombopag begin on Day 1.

Group III: Cohort A (Option 1) Experimental Treatment 3 Interventions

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

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD