Trial · NCT04526704
Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
NCT04526704, Phase 4, Completed, Daiichi Sankyo; results first posted 2024-12-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib. Trials, record NCT04526704. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04526704
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
- NCT number
- NCT04526704
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 32 participants, actual
- Lead sponsor
- Daiichi Sankyo
- Conditions
- Tenosynovial Giant Cell Tumor
- Interventions
- Pexidartinib
- First posted
- 2020-08-26
- Last updated
- 2024-12-30
- Other registries' IDs
- eudract:2020-000192-20
- Results first posted
- 2024-12-30
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort | Baseline up to 12 months after last participant enrolled in Cohort | Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first. The number of participants who remained treatment-free at Month 12 is reported. |
| Primary | Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort | Baseline up to 24 months after last participant enrolled in Cohort | Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first. The number of participants who remained treatment-free at Month 24 is reported. |
| Secondary | Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Baseline up to Month 24 | The PROMIS Physical Function Total Overall Score (includes 11 upper extremity questions and 13 lower extremity questions) ranges from 24 to 120, with each individual question being rated on a 5-point rating scale (where 1 is unable to do and 5 is without any difficulty). Higher PROMIS Physical Function Total Overall Scores indicate a better health state. The change from baseline in PROMIS Physical Function Total Overall Scores is being reported. |
| Secondary | Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Baseline up to Month 24 | The EQ-5D-5L questionnaire assessed a participant's mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The overall health is rated on a scale from 0 to 100, where 0 is worst health you can imagine and 100 is best health you can imagine. The change from baseline in EQ-5D-5L Scale Score is being reported. |
| Secondary | Number of Participants With and Without Progressive Disease | Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort) | Tumors were assessed based on the RECIST criteria. CR was defined as a disappearance of all target lesions, PR was defined as at least a 30% decrease in the sum of diameters of target lesions, and stable disease (SD) was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD; ≥30% increase in volume relative to lowest score during the study whether at baseline or some other visit. |
| Secondary | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Treatment-emergent adverse events (TEAEs) were defined as new adverse events (AEs) or pre-existing conditions that worsen in CTCAE grade after the first dose of study drug and up to 30 days after last dose of study drug. |
| Secondary | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Treatment-emergent adverse events (TEAEs) were defined as new adverse events (AEs) or pre-existing conditions that worsen in CTCAE grade after the first dose of study drug and up to 30 days after last dose of study drug. |
| Secondary | Number of Patients Reporting AEs (Treatment-free) | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Adverse events (AEs) were defined as any untoward medical occurrence in a participant who was administered a pharmaceutical product and that does not necessarily have to have a causal relationship with this treatment. Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first. |
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 | Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort | Baseline up to 12 months after last participant enrolled in Cohort | The number of treatment-free participants was assessed in participants with available data in the Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort | Baseline up to 24 months after last participant enrolled in Cohort | The number of treatment-free participants was assessed in participants with available data in the Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Baseline up to Month 24 | PROMIS Physical Function was assessed in participants with available data in the Evaluable Analysis Set. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Baseline up to Month 24 | EQ-5D-5L was assessed in participants with available data in the Evaluable Analysis Set. | score on a scale | MEAN |
| SECONDARY | Number of Participants With and Without Progressive Disease | Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort) | Tumor assessments were analyzed in participants with available data in the Full Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Treatment-emergent adverse events were assessed in participants with available data in the Safety Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Treatment-emergent adverse events were assessed in participants with available data in the Safety Analysis Set . | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients Reporting AEs (Treatment-free) | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months | Adverse events were assessed in the Safety Analysis Set in participants with available data in the Treatment-free Period of the Treatment-Free/Re-Treatment Cohort. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group45
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort | Treatment-Free/Re-Treatment Cohort | 8 | Participants | ||
| Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort | Treatment-Free/Re-Treatment Cohort | 8 | Participants | ||
| Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Treatment Continuation Cohort | -2.78 | Standard Deviation 6.42 | score on a scale | |
| Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Treatment-Free/Re-Treatment Cohort | -1.93 | Standard Deviation 4.80 | score on a scale | |
| Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Treatment Continuation Cohort | -2.4 | Standard Deviation 13.53 | score on a scale | |
| Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | Treatment-Free/Re-Treatment Cohort | 3.4 | Standard Deviation 7.59 | score on a scale | |
| Number of Participants With and Without Progressive Disease | Treatment Continuation Cohort | Not progressive | 16 | Participants | |
| Number of Participants With and Without Progressive Disease | Treatment-Free/Re-Treatment Cohort | Not progressive | 0 | Participants | |
| Number of Participants With and Without Progressive Disease | Treatment Continuation Cohort | Progressive disease | 0 | Participants | |
| Number of Participants With and Without Progressive Disease | Treatment-Free/Re-Treatment Cohort | Progressive disease | 1 | Participants |
1 to 10 of 45Source ClinicalTrials.gov
See all 45 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Treatment Continuation Cohort | 21 | |
| Overall Study | STARTED | Treatment-Free/Re-Treatment Cohort | 11 | |
| Overall Study | Resumed Pexidartinib Treatment | Treatment Continuation Cohort | 0 | |
| Overall Study | Resumed Pexidartinib Treatment | Treatment-Free/Re-Treatment Cohort | 3 | |
| Overall Study | COMPLETED | Treatment Continuation Cohort | 17 | |
| Overall Study | COMPLETED | Treatment-Free/Re-Treatment Cohort | 11 | |
| Overall Study | NOT COMPLETED | Treatment Continuation Cohort | 4 | |
| Overall Study | NOT COMPLETED | Treatment-Free/Re-Treatment Cohort | 0 |
Source ClinicalTrials.gov
Adverse events144
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 | COVID-19 | Infections and infestations | Treatment Continuation Cohort | 7 | 21 | |
| other | COVID-19 | Infections and infestations | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 2 | 3 | |
| other | COVID-19 | Infections and infestations | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 1 | 11 | |
| other | Upper respiratory tract infection | Infections and infestations | Treatment Continuation Cohort | 4 | 21 | |
| other | Upper respiratory tract infection | Infections and infestations | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 0 | 3 | |
| other | Upper respiratory tract infection | Infections and infestations | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 0 | 11 | |
| other | Tooth infection | Infections and infestations | Treatment Continuation Cohort | 2 | 21 | |
| other | Tooth infection | Infections and infestations | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 0 | 3 | |
| other | Tooth infection | Infections and infestations | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 0 | 11 | |
| other | Sinusitis | Infections and infestations | Treatment Continuation Cohort | 0 | 21 |
1 to 10 of 144Source ClinicalTrials.gov
See all 144 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Continuation Cohort | 0 | 21 |
| Total, all-cause mortality | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 0 | 3 |
| Total, all-cause mortality | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 0 | 11 |
| Total, other adverse events | Treatment Continuation Cohort | 19 | 21 |
| Total, other adverse events | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 3 | 3 |
| Total, other adverse events | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 7 | 11 |
| Total, serious adverse events | Treatment Continuation Cohort | 2 | 21 |
| Total, serious adverse events | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | 0 | 3 |
| Total, serious adverse events | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | 0 | 11 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT04526704
GET https://conductscience.org/api/trials/v1/NCT04526704/results?page=2