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NCT04211337 · STUDY IN CONTEXT

A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

PFS is defined as the time from randomization until the occurrence of documented disease progression by the BICR, per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) criteria, or death from any cause in the absence of BICR-documented progressive disease. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.

All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 167; Cabozantinib or Vandetanib - 66

→

secondary · POSTED

TFFS by BICR is defined as the time from randomization to the first occurrence of: * documented radiographic disease progression per RECIST 1.1 as assessed by BICR; or * unacceptable toxicity leading to treatment discontinuation as assessed by the investigator. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum on study and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions. To qualify as an event, the toxicity must be from an intolerable AE (defined as any study drug-related AE that meets protocol guidance for treatment discontinuation, with the exception of alopecia); or death (due to any cause).

All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 166; Cabozantinib or Vandetanib - 61

→

secondary · POSTED

ORR is defined as the number of participants who achieved the best overall response (BOR) of CR or PR divided by the total number of participants randomized to each treatment arm. ORR per RECIST 1.1 as assessed by BICR.

All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol.

→

secondary · POSTED

DoR by BICR is defined as the time from the date that measurement criteria for complete response (CR) or partial response (PR) (whichever is first recorded) are first met by the BICR or investigator assessment, as applicable, until the first date that disease is recurrent or documented disease progression is observed, per RECIST 1.1 criteria, or the date of death from any cause in the absence of documented disease progression or recurrence. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.

All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 119, Cabozantinib or Vandetanib - 25

→

secondary · NOT_POSTED

Overall survival (OS) is defined as the time from randomization until death from any cause. If the participant is alive or lost to follow-up at the time of data analysis, OS data will be censored on the last date the participant is known to be alive.

Source null

→

secondary · NOT_POSTED

Progression-free survival 2 (PFS2) is defined as the time from randomization to disease progression (radiographic or symptomatic progression as determined by the investigator) on the next line of treatment or death from any cause in the absence of observed disease progression. If the participant is alive at the cutoff for analysis, and disease progression has not been observed, PFS2 data will be censored on the latest date of last progression-free assessment or start of the next line of treatment.

Source null

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

FACT · 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)

Score direction: Not stated by the study. Direction is never inferred from an instrument name.

release id
clinical-indications-draft-1
disease state id
ds-thyroid-cancer
nct id
NCT04211337
qol key
qol-0006
source section id
NCT04211337-om-0006
source outcome index
6
instrument
FACT
instrument basis
matched 'fact-' in the study's own wording
scale title
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)
measure definition
Comparative tolerability defined as a comparison of the proportion of time on treatment with high side effect bother as assessed by the FACT-GP5. The FACT-GP5 is a single question used to assess the overall bother of the treatment side effects. It is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bother from treatment side effects. Time with high side effect bother (i.e.) score of 3 or 4 is reported here and was derived as follows: cumulative amount of time, in weeks, during which a participant reports high side effect bother divided by the total duration of therapy (weeks), derived as (date of last study treatment dose - date of first study treatment dose + 1) divided by 7.
time frame
Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months
analysis role
secondary
reporting status
POSTED
higher is better
Source null
direction quote
Source null
population note
All participants who received the first dose of study treatment prior to the interim efficacy analysis and at least 6 months prior to the data cutoff date. Analysis of tolerability will be based on the actual treatment a participant received on the first study treatment administration regardless of which treatment they were randomized to receive ("as treated").
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
Open the exact result →
Full draft applicability and source-link record
trial
release id
clinical-indications-draft-1
disease state id
ds-thyroid-cancer
nct id
NCT04211337
trial label
A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer
applicability
narrower
applicability note
Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
provenance class
regulator-cited
discovery note
Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count
6
harms Count
2
qol Count
1
outcomes
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0000
    source section id
    NCT04211337-om-0000
    source outcome index
    0
    endpoint
    Progression Free Survival (PFS) by Blinded Independent Central Review (BICR)
    endpoint definition
    PFS is defined as the time from randomization until the occurrence of documented disease progression by the BICR, per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) criteria, or death from any cause in the absence of BICR-documented progressive disease. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.
    time origin
    Baseline to Progressive Disease or Death from Any Cause, Whichever Occurs First, Up to 39 Months
    analysis role
    primary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 167; Cabozantinib or Vandetanib - 66
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0001
    source section id
    NCT04211337-om-0001
    source outcome index
    1
    endpoint
    Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR)
    endpoint definition
    TFFS by BICR is defined as the time from randomization to the first occurrence of: * documented radiographic disease progression per RECIST 1.1 as assessed by BICR; or * unacceptable toxicity leading to treatment discontinuation as assessed by the investigator. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum on study and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions. To qualify as an event, the toxicity must be from an intolerable AE (defined as any study drug-related AE that meets protocol guidance for treatment discontinuation, with the exception of alopecia); or death (due to any cause).
    time origin
    Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 166; Cabozantinib or Vandetanib - 61
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0002
    source section id
    NCT04211337-om-0002
    source outcome index
    2
    endpoint
    Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR
    endpoint definition
    ORR is defined as the number of participants who achieved the best overall response (BOR) of CR or PR divided by the total number of participants randomized to each treatment arm. ORR per RECIST 1.1 as assessed by BICR.
    time origin
    Baseline through Disease Progression or Death Up to 39 Months
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0003
    source section id
    NCT04211337-om-0003
    source outcome index
    3
    endpoint
    Duration of Response (DoR) by BICR
    endpoint definition
    DoR by BICR is defined as the time from the date that measurement criteria for complete response (CR) or partial response (PR) (whichever is first recorded) are first met by the BICR or investigator assessment, as applicable, until the first date that disease is recurrent or documented disease progression is observed, per RECIST 1.1 criteria, or the date of death from any cause in the absence of documented disease progression or recurrence. Progressive Disease (PD) was at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.
    time origin
    Date of CR or PR to Date of Disease Progression or Death Due to Any Cause Up to 39 Months
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    All randomized participants, even if a participant did not take the assigned treatment, did not receive the correct treatment, or otherwise did not follow the protocol. Censored participants: Selpercatinib - 119, Cabozantinib or Vandetanib - 25
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0004
    source section id
    NCT04211337-om-0004
    source outcome index
    4
    endpoint
    Overall Survival (OS)
    endpoint definition
    Overall survival (OS) is defined as the time from randomization until death from any cause. If the participant is alive or lost to follow-up at the time of data analysis, OS data will be censored on the last date the participant is known to be alive.
    time origin
    Baseline
    analysis role
    secondary
    reporting status
    NOT_POSTED
    data cutoff
    Source null
    population note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0005
    source section id
    NCT04211337-om-0005
    source outcome index
    5
    endpoint
    PFS2 by Investigator
    endpoint definition
    Progression-free survival 2 (PFS2) is defined as the time from randomization to disease progression (radiographic or symptomatic progression as determined by the investigator) on the next line of treatment or death from any cause in the absence of observed disease progression. If the participant is alive at the cutoff for analysis, and disease progression has not been observed, PFS2 data will be censored on the latest date of last progression-free assessment or start of the next line of treatment.
    time origin
    Baseline
    analysis role
    secondary
    reporting status
    NOT_POSTED
    data cutoff
    Source null
    population note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
arms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0000
    arm key
    OG000
    arm label
    Selpercatinib (TRT A)
    regimen
    160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 mg/m2 BID (not to exceed 160 mg BID).
    analyzed count
    193
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0000#groups/OG000
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0000
    arm key
    OG001
    arm label
    Cabozantinib or Vandetanib (TRT B)
    regimen
    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
    analyzed count
    98
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0000#groups/OG001
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0001
    arm key
    OG000
    arm label
    Selpercatinib (TRT A)
    regimen
    160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 mg/m2 BID (not to exceed 160 mg BID).
    analyzed count
    193
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0001#groups/OG000
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0001
    arm key
    OG001
    arm label
    Cabozantinib or Vandetanib (TRT B)
    regimen
    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
    analyzed count
    98
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0001#groups/OG001
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0002
    arm key
    OG000
    arm label
    Selpercatinib (TRT A)
    regimen
    160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 mg/m2 BID (not to exceed 160 mg BID).
    analyzed count
    193
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0002#groups/OG000
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0002
    arm key
    OG001
    arm label
    Cabozantinib or Vandetanib (TRT B)
    regimen
    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
    analyzed count
    98
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0002#groups/OG001
  7. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0003
    arm key
    OG000
    arm label
    Selpercatinib (TRT A)
    regimen
    160 milligrams Selpercatinib administered orally (PO) twice daily (BID). Adolescent Dose: 92 mg/m2 BID (not to exceed 160 mg BID).
    analyzed count
    134
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0003#groups/OG000
  8. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    outcome key
    om-0003
    arm key
    OG001
    arm label
    Cabozantinib or Vandetanib (TRT B)
    regimen
    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
    analyzed count
    38
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT04211337-om-0003#groups/OG001
harms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    harm key
    EG000
    source section id
    NCT04211337-ae
    event group key
    EG000
    event group label
    Selpercatinib - Treatment A (TRT A)
    serious affected
    43
    serious at risk
    193
    other affected
    184
    other at risk
    193
    frequency threshold
    5
    collection period
    Baseline up to 39 months
    event definition
    All randomized participants who received at least 1 dose (including a partial dose) of study treatment. Analysis of safety data will be based on the actual treatment a participant received on the first study treatment administration regardless of which treatment they were randomized to receive ("as treated"). Gender specific events occurring only in male or female participants have had the number of participants At Risk adjusted accordingly.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    harm key
    EG001
    source section id
    NCT04211337-ae
    event group key
    EG001
    event group label
    Cabozantinib or Vandetanib - Treatment B (TRT B)
    serious affected
    27
    serious at risk
    97
    other affected
    96
    other at risk
    97
    frequency threshold
    5
    collection period
    Baseline up to 39 months
    event definition
    All randomized participants who received at least 1 dose (including a partial dose) of study treatment. Analysis of safety data will be based on the actual treatment a participant received on the first study treatment administration regardless of which treatment they were randomized to receive ("as treated"). Gender specific events occurring only in male or female participants have had the number of participants At Risk adjusted accordingly.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
quality of life
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-thyroid-cancer
    nct id
    NCT04211337
    qol key
    qol-0006
    source section id
    NCT04211337-om-0006
    source outcome index
    6
    instrument
    FACT
    instrument basis
    matched 'fact-' in the study's own wording
    scale title
    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)
    measure definition
    Comparative tolerability defined as a comparison of the proportion of time on treatment with high side effect bother as assessed by the FACT-GP5. The FACT-GP5 is a single question used to assess the overall bother of the treatment side effects. It is scored using a 5-point rating scale (0 = not at all; 1 = a little bit; 2 = somewhat; 3 = quite a bit; and 4 = very much), where lower scores reflect less bother from treatment side effects. Time with high side effect bother (i.e.) score of 3 or 4 is reported here and was derived as follows: cumulative amount of time, in weeks, during which a participant reports high side effect bother divided by the total duration of therapy (weeks), derived as (date of last study treatment dose - date of first study treatment dose + 1) divided by 7.
    time frame
    Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months
    analysis role
    secondary
    reporting status
    POSTED
    higher is better
    Source null
    direction quote
    Source null
    population note
    All participants who received the first dose of study treatment prior to the interim efficacy analysis and at least 6 months prior to the data cutoff date. Analysis of tolerability will be based on the actual treatment a participant received on the first study treatment administration regardless of which treatment they were randomized to receive ("as treated").
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
setting source links

    Preserved source evidence · Not medical advice