Selpercatinib in Solid or CNS tumors with RET-Alterations



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The purpose of this study is to test the safety and efficacy of the investigational drug called Selpercatinib in children whose cancers have a specific genetic change (RET-fusion or RET-mutation). Selpercatinib has been FDA-approved for adults and pediatric patients 12 years of age or older for certain types of lung and thyroid cancers. Selpercatinib is administered in capsule form, liquid suspension, or may also be administered by a feeding tube for subjects who are unable to swallow.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at CancerTrials [at] or 267-425-5544.

Eligibility & Criteria

IRB #:
Official Title:
A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 (Selpercatinib) in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors
Study Phase:
Phase I
Phase II
Eligible Age Range:
6 Months - 21 Years
Study Categories:

Visit Criteria

As a participant in the research, you will:

  • Be given selpercatinib to take in the clinic and at home
  • Complete frequent clinic visits at CHOP
  • Have frequent blood tests, including research blood tests to measure selpercatinib levels (pharmacokinetic studies), hormone levels, and tests to measure genetic material in your blood
  • Have periodic EKGs
  • Have periodic imaging performed to evaluate your response to treatment, evaluate your growth plate (if you are not at full adult height)
  • Have a dental evaluation (if you are ≥ 5 years old)
  • Have a puberty maturation evaluation (if you are ≥ 7 years old)
  • Be asked to complete questionnaires and medication diaries
  • Be asked if we can submit previously obtained samples of your tumor to the sponsor; this will not require new biopsy
  • Be asked if we can obtain a small sample of cerebrospinal fluid (CSF) if collected as part of your regular cancer care