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Protocol No. UW17082

Principal Investigator Hofmann, Inga

Phase II

Age Group Children

Scope National

Sponsor Type Industry

Title A Phase II, Open-Label, Non-Controlled, Intra-Patient Dose-Escalation Study to Characterize the Pharmacokinetics after Oral Administration of Eltrombopag in Pediatric Patients with Refractory, Relapsed, or Treatment Naïve Severe Aplastic Anemia or Recurrent Aplastic Anemia

Objective This research study will find out if a drug called eltrombopag might help children with SAA. It will also help researchers understand how the body absorbs, distributes, breaks down, and eliminates eltrombopag

Treatment Cohort A:
  • hATG + CsA + Eltrombopag
  • CsA + Eltrombopag
    Cohort B:
  • hATG + CsA + Eltrombopag

  • Description Phase II study with Eltrombopag for Aplastic Anemia

    Key Eligibility For Cohort A patients:
  • History of prior diagnosis of SAA,
  • Diagnosis of relapsed/refractory SAA or recurrent AA following treatment for SAA, as per Section 5.1. Patients with recurrent AA (e.g., losing their response) are exempt from meeting the diagnostic criteria for SAA relapse at the time of study enrollment, but must have been previously diagnosed with SAA
  • Agree to concurrent eltrombopag treatment with appropriate, investigator-selected IST with either hATG + CsA or CsA
    For Cohort B patients:
  • Diagnosis of SAA at time of enrollment
  • Patients must not have been previously treated with IST, and must meet all criteria as described by the protocol
  • Patients must agree to treatment with hATG + CsA concurrent with eltrombopag.
  • Age 1 to 18 years
  • Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag
  • Normal karyotype with FISH for chromosomes 7 and 8
  • Karnofsky greater than or equal to 50 for patients 16 years of age and older or Lansky greater than or equal to 50 for patients below 16 years of age
  • Serum creatinine less than or equal to 2.5 × ULN and Total bilirubin less than or equal to 1.5 × ULN
  • Suitable candidate to receive a HSCT. (Candidacy for HSCT will be determined as per local practices or national guidelines)
  • Prior and/or active medical history of: Fanconi anemia (via chromosome breakage test or growth arrest by flow cytometry), Other known underlying inherited marrow failure syndrome (such as but not limited to Dyskeratosis Congenita, Congenital Amegakaryocytic Thrombocytopenia, or Shwachman-Diamond Syndrome), Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones greater than 50% of PMN or RBC at time of enrollment, Any cytogenetic abnormalities, including but not limited to chromosome 7 or myelodysplasia, in bone marrow within 4 weeks of study enrollment,MDS,
  • Other known or suspected underlying primary immunodeficiency, and any malignancy
  • Active infection not responding to appropriate therapy
  • Prior eltrombopag or other thrombopoietin receptor (TPO-R) agonist treatment for at least 2 months and a lack of response
  • Pregnant or breastfeeding
  • Male and female of childbearing potential must agree to use adequate birth control as defined by the protocol
  • Patients with known history of HIV positivity
  • Patients with known history of hepatitis B or C positivity
  • Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of study drug (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome
  • Impaired cardiac function: Patients with a history of congenital long QT syndrome or known family history of long QTc syndrome, corrected QTc >450 msec using Fridericia correction (QTcF) on the screening ECG (using triplicate ECGs)
  • Ventricular arrhythmias and or other cardiac arrhythmia not controlled with medication
  • Other clinically significant cardio-vascular disease (e.g., congestive heart failure, uncontrolled hypertension, history of labile hypertension)
  • History of known structural abnormalities (e.g., cardiomyopathy)

  • Applicable Disease Sites Hematologic cancers, other

    Status Open

    Participating Institutions UW Hospital and Clinics