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Tyrosine Kinase Inhibitor

Selpercatinib for Medullary Thyroid Cancer (LIBRETTO-531 Trial)

Phase 3

Waitlist Available

Research Sponsored by Loxo Oncology, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Tumor tissue in sufficient quantity to allow for retrospective central analysis of RET mutation status

A defined/acceptable RET gene alteration identified in a tumor, germline deoxyribonucleic acid (DNA) or blood sample

Must not have

Active hemorrhage or at significant risk for hemorrhage

Active uncontrolled systemic bacterial, viral, or fungal infection or serious ongoing uncontrolled intercurrent illness

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to date of death from any cause up to 36 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a new drug to see if it's more effective and safe than the current standard treatment for people with a certain type of thyroid cancer.

Who is the study for?

This trial is for adults and some minors (12+ if allowed) with advanced, inoperable or metastatic Medullary Thyroid Cancer that has a specific RET gene change. They shouldn't have had kinase inhibitor treatment before. Participants need to show disease progression, be reasonably healthy overall, able to swallow capsules, and willing to use effective contraception.

What is being tested?

The study tests if Selpercatinib is safer and more effective than standard treatments (Cabozantinib or Vandetanib) for RET-mutant MTC. If the standard treatment fails, participants may switch to Selpercatinib. The goal is to compare outcomes between the new drug and existing options.

What are the potential side effects?

Selpercatinib might cause liver issues, high blood pressure, bleeding problems, dry mouth or altered taste sensation; Cabozantinib can lead to diarrhea, mouth sores or hand-foot syndrome; Vandetanib may result in rash or acneiform dermatitis.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My tumor sample is large enough for further genetic testing.

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My cancer has a specific change in the RET gene.

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My medullary thyroid cancer is advanced and I haven't had kinase inhibitor treatments.

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I can take care of myself and am up and about more than half of my waking hours.

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I can swallow pills.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently experiencing significant bleeding or at high risk for serious bleeding.

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I do not have any uncontrolled infections or serious ongoing illnesses.

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My cancer has a gene change that might resist selpercatinib treatment.

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I have symptoms from cancer spread to my brain or spinal cord.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to date of death from any cause up to 36 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to date of death from any cause up to 36 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to date of death from any cause up to 36 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Progression Free Survival (PFS) by Blinded Independent Central Review (BICR)

Secondary study objectives

Comparative Tolerability: Number of Weeks With High Side Effect Bother Based Score of 3 or 4 on the Functional Assessment of Cancer Therapy Item GP5 (FACT-GP5)

Duration of Response (DoR) by BICR

Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Selpercatinib - Treatment A (TRT A)Experimental Treatment1 Intervention

160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 milligrams per square meter (mg/m2) BID (not to exceed 160 mg BID).

Group II: Cabozantinib or Vandetanib - Treatment B (TRT B)Active Control2 Interventions

140 mg Cabozantinib administered orally daily (QD) or 300 mg Vandetanib administered orally QD per physician choice. Cabozantinib Adolescent Dose: 40 mg/m2. Vandetanib Adolescent Dose: * 0.7 - \<0.9 - 100 mg every other day (QOD) * 0.9 - \<1.2 - 100 mg QD * 1.2 - \<1.6 - 7-day schedule 100 mg - 200 mg - 100 mg - 200 mg - 100 mg - 200 mg - 100 mg * ≥1.6 - 200 QD

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Selpercatinib

2021

Completed Phase 1

~600

0 / 9Eligibility criteria met