A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors

ENROLLING
Protocol # :
19-026
Conditions
Medullary Thyroid Cancer
Infantile Myofibromatosis
Infantile Fibrosarcoma
Papillary Thyroid Cancer
Soft Tissue Sarcoma
Phase
I/II
Disease Sites
Brain and Nervous System
Principal Investigator
Dubois, Steven
Site Research Nurses
Aspri, Tristan
Cavanaugh, Kerri, Lynn
Ezrre, Suzanne
Strachan, Marylynne

Trial Description

This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric
participants with an activating rearranged during transfection (RET) alteration and an
advanced solid or primary CNS tumor.

Eligibility Requirements

Inclusion Criteria:

- Advanced or metastatic solid or primary CNS tumor which has failed standard of care
therapies

- Evidence of an activating RET gene alteration in the tumor and/or blood

- Measurable or non-measurable disease

- Karnofsky (participants 16 years and older) or Lansky (participants younger than 16)
performance score of at least 50

- Participant with primary CNS tumors or cerebral metastases must be neurologically
stable for 7 days prior and must not have required increasing doses of steroids within
the last 7 days

- Adequate hematologic, hepatic and renal function.

- Ability to receive study drug therapy orally or via gastric access

- Willingness of men and women of reproductive potential to observe conventional and
effective birth control

Exclusion Criteria:

- Major surgery within two weeks prior to planned start of LOXO-292

- Clinically significant, uncontrolled cardiac, cardiovascular disease or history of
myocardial infarction within 6 months prior to planned start of LOXO-292

- Active uncontrolled systemic bacterial, viral, fungal or parasitic infection

- Clinically significant active malabsorption syndrome

- Pregnancy or lactation

- Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant
required a modification to current thyroid medication in the 7 days before start of
LOXO-292)

- Uncontrolled symptomatic hypercalcemia or hypocalcemia

- Known hypersensitivity to any of the components of the investigational agent, LOXO-292
or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension

- Prior treatment with a selective RET inhibitor(s) (including investigational selective
RET inhibitor[s])

19-026