Phase 2  | 

LIBRETTO-121; NCT03899792

Pediatric Cancer

RET Inhibitor

A Phase 1/2 Study of the Oral RET Inhibitor Selpercatinib in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors*

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Key Inclusion Criteria
  • Pediatric patients aged 6 months to 21 years with advanced or metastatic solid or primary central nervous system (CNS) tumors and have failed standard-of-care therapies
  • Evidence of an activating RET gene alteration in the tumor and/or blood
  • Measurable or evaluable disease
  • Karnofsky (patients ≥16 years of age) or Lansky (patients <16 years of age) performance score of at least 50
  • Patients with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior to start of treatment and must not have required increasing doses of steroids within the last 7 days
  • Adequate hematologic, hepatic, and renal function
  • Able to receive study drug therapy orally or via gastric access
  • Males and females of reproductive potential must be willing to use conventional and effective birth control
Key Exclusion Criteria
  • Major surgery within 14 days prior to planned start of selpercatinib
  • Clinically significant, uncontrolled cardiac or cardiovascular disease, or history of myocardial infarction within 6 months prior to planned start of selpercatinib
  • Active uncontrolled systemic bacterial, viral, fungal, or parasitic infection
  • Clinically significant active malabsorption syndrome
  • Pregnant or breastfeeding
  • Uncontrolled symptomatic hyperthyroidism or hypothyroidism
  • Uncontrolled symptomatic hypercalcemia or hypocalcemia
  • For patients who will be receiving selpercatinib suspension: Known hypersensitivity to any of the components of the investigational agent or Ora-Sweet® and Ora-Plus®   
  • Prior treatment with a selective RET inhibitor(s), including investigational
This clinical trial is being conducted globally.
Selpercatinib is administered PO.
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The safety and efficacy of the agents under investigation have not been established. There is no guarantee that the agents will receive regulatory approval and become commercially available for the uses being investigated.