Trial · NCT04211337
A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer
NCT04211337, Phase 3, Active not recruiting, Loxo Oncology, Inc.; results first posted 2024-06-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer. Trials, record NCT04211337. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04211337
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer
- NCT number
- NCT04211337
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 291 participants, actual
- Lead sponsor
- Loxo Oncology, Inc.
- Conditions
- Medullary Thyroid Cancer
- Interventions
- Selpercatinib, Cabozantinib, Vandetanib
- First posted
- 2019-12-26
- Last updated
- 2026-03-19
- Other registries' IDs
- eudract:2019-001978-28
- Results first posted
- 2024-06-13
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) | Baseline to Progressive Disease or Death from Any Cause, Whichever Occurs First, Up to 39 Months | 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. |
| Secondary | Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR) | Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months | 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). |
| Secondary | Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR | Baseline through Disease Progression or Death Up to 39 Months | 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. |
| Secondary | Duration of Response (DoR) by BICR | Date of CR or PR to Date of Disease Progression or Death Due to Any Cause Up to 39 Months | 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. |
| Secondary | Overall Survival (OS) | Baseline | 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. |
| Secondary | PFS2 by Investigator | Baseline | 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. |
| Secondary | 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) | Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months | 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. |
| Secondary | The Concordance of the Local Lab and the Central Lab RET Results: Percentage of Participants With RET-Positive Specimens as Called by the Central Lab, Which is Also RET-Positive as Called by a Local Lab (Positive Percent Agreement) | Baseline |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) | Baseline to Progressive Disease or Death from Any Cause, Whichever Occurs First, Up to 39 Months | 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 | Months | MEDIAN |
| SECONDARY | Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR) | Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months | 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 | Months | MEDIAN |
| SECONDARY | Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR | Baseline through Disease Progression or Death Up to 39 Months | 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. | Percentage of Participants | NUMBER |
| SECONDARY | Duration of Response (DoR) by BICR | Date of CR or PR to Date of Disease Progression or Death Due to Any Cause Up to 39 Months | 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 | Months | MEDIAN |
| SECONDARY | Overall Survival (OS) | Baseline | |||
| SECONDARY | PFS2 by Investigator | Baseline | |||
| SECONDARY | 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) | Baseline to Progressive Disease, Unacceptable Toxicity or Death from Any Cause Up to 39 Months | 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). | Weeks | MEAN |
| SECONDARY | The Concordance of the Local Lab and the Central Lab RET Results: Percentage of Participants With RET-Positive Specimens as Called by the Central Lab, Which is Also RET-Positive as Called by a Local Lab (Positive Percent Agreement) | Baseline |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) | Selpercatinib (TRT A) | NA | 95% Confidence Interval NA to NA | Months | |
| Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) | Cabozantinib or Vandetanib (TRT B) | 16.76 | 95% Confidence Interval 12.22 to 25.10 | Months | |
| Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR) | Selpercatinib (TRT A) | NA | 95% Confidence Interval NA to NA | Months | |
| Treatment Failure-Free Survival (TFFS) by Blinded Independent Committee Review (BICR) | Cabozantinib or Vandetanib (TRT B) | 13.93 | 95% Confidence Interval 11.27 to 25.10 | Months | |
| Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR | Selpercatinib (TRT A) | 69.4 | 95% Confidence Interval 62.4 to 75.8 | Percentage of Participants | |
| Overall Response Rate (ORR): Percentage of Participants With Complete Response (CR) or Partial Response (PR) by BICR | Cabozantinib or Vandetanib (TRT B) | 38.8 | 95% Confidence Interval 29.1 to 49.2 | Percentage of Participants | |
| Duration of Response (DoR) by BICR | Selpercatinib (TRT A) | NA | 95% Confidence Interval NA to NA | Months | |
| Duration of Response (DoR) by BICR | Cabozantinib or Vandetanib (TRT B) | 16.56 | 95% Confidence Interval 10.41 to NA | Months | |
| 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) | Selpercatinib (TRT A) | 0.08 | Standard Deviation 0.169 | Weeks | |
| 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) | Cabozantinib or Vandetanib (TRT B) | 0.24 | Standard Deviation 0.304 | Weeks |
Source ClinicalTrials.gov
Participant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Selpercatinib - Treatment A (TRT A) | 193 | |
| Overall Study | STARTED | Cabozantinib or Vandetanib - Treatment B (TRT B) | 98 | |
| Overall Study | Received at Least One Dose of Study Drug | Selpercatinib - Treatment A (TRT A) | 193 | |
| Overall Study | Received at Least One Dose of Study Drug | Cabozantinib or Vandetanib - Treatment B (TRT B) | 97 | |
| Overall Study | COMPLETED | Selpercatinib - Treatment A (TRT A) | 18 | |
| Overall Study | COMPLETED | Cabozantinib or Vandetanib - Treatment B (TRT B) | 56 | |
| Overall Study | NOT COMPLETED | Selpercatinib - Treatment A (TRT A) | 175 | |
| Overall Study | NOT COMPLETED | Cabozantinib or Vandetanib - Treatment B (TRT B) | 42 |
Source ClinicalTrials.gov
Adverse events342
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Selpercatinib - Treatment A (TRT A) | 11 | 193 | 12 |
| other | Anaemia | Blood and lymphatic system disorders | Cabozantinib or Vandetanib - Treatment B (TRT B) | 10 | 97 | 16 |
| other | Leukopenia | Blood and lymphatic system disorders | Selpercatinib - Treatment A (TRT A) | 10 | 193 | 13 |
| other | Leukopenia | Blood and lymphatic system disorders | Cabozantinib or Vandetanib - Treatment B (TRT B) | 6 | 97 | 7 |
| other | Lymphopenia | Blood and lymphatic system disorders | Selpercatinib - Treatment A (TRT A) | 10 | 193 | 16 |
| other | Lymphopenia | Blood and lymphatic system disorders | Cabozantinib or Vandetanib - Treatment B (TRT B) | 4 | 97 | 4 |
| other | Neutropenia | Blood and lymphatic system disorders | Selpercatinib - Treatment A (TRT A) | 3 | 193 | 6 |
| other | Neutropenia | Blood and lymphatic system disorders | Cabozantinib or Vandetanib - Treatment B (TRT B) | 8 | 97 | 17 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Selpercatinib - Treatment A (TRT A) | 4 | 193 | 5 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Cabozantinib or Vandetanib - Treatment B (TRT B) | 9 | 97 | 10 |
1 to 10 of 342Source ClinicalTrials.gov
See all 342 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Selpercatinib - Treatment A (TRT A) | 8 | 193 |
| Total, all-cause mortality | Cabozantinib or Vandetanib - Treatment B (TRT B) | 10 | 97 |
| Total, other adverse events | Selpercatinib - Treatment A (TRT A) | 184 | 193 |
| Total, other adverse events | Cabozantinib or Vandetanib - Treatment B (TRT B) | 96 | 97 |
| Total, serious adverse events | Selpercatinib - Treatment A (TRT A) | 43 | 193 |
| Total, serious adverse events | Cabozantinib or Vandetanib - Treatment B (TRT B) | 27 | 97 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Selpercatinib - Treatment A (TRT A) vs Cabozantinib or Vandetanib - Treatment B (TRT B): deaths from any cause | Trial comparisons |
| Selpercatinib - Treatment A (TRT A) vs Cabozantinib or Vandetanib - Treatment B (TRT B): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04211337
GET https://conductscience.org/api/trials/v1/NCT04211337/events?page=2