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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-000192-20 | EudraCT Number |
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This study is designed to evaluate the discontinuation/re-treatment of pexidartinib therapy in previously treated participants with tenosynovial giant cell tumor (TGCT).
This multicenter study in previously pexidartinib-treated participants with TGCT will provide the Investigators and participants the option at Screening to either continue pexidartinib treatment (Treatment Continuation Cohort) or discontinue treatment with the possibility of re-initiating pexidartinib treatment (Treatment-Free/Re-Treatment Cohort).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Continuation Cohort | Experimental | Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment. |
|
| Treatment-Free/Re-Treatment Cohort | Experimental | Previously-treated participants with TGCT who discontinue pexidartinib treatment (Treatment-Free Period) and had the option to resume pexidartinib treatment at dose at completion of prior study (Re-Treatment Period). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pexidartinib | Drug | 200 mg capsules administered orally twice daily on an empty stomach (at least 1 hour before or at least 2 hours after a meal or snack) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment 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. | Baseline up to 12 months after last participant enrolled in Cohort |
| Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment 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. | Baseline up to 24 months after last participant enrolled in Cohort |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | 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. |
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Inclusion Criteria:
Note: A female is considered of reproductive potential following menarche and until becoming postmenopausal (no menstrual period for a minimum of 12 months) unless permanently sterile (undergone a hysterectomy, bilateral salpingectomy or bilateral oophorectomy) with a confirmed by follicle stimulating hormone (FSH) test level >40 mIU/mL.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Team Leader | Daiichi Sankyo | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Honor Health | Scottsdale | Arizona | 85258 | United States | ||
| USC Norris Comprehensive Cancer Center |
De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Studies for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.
As specified in the protocol, the study analysis was conducted based on 2 cohorts: Treatment Continuation Cohort and Treatment-Free/Re-Treatment Cohort.
A total of 32 participants who met all inclusion criteria and no exclusion criteria were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Continuation Cohort | Previously-treated participants with TGCT who continued their current dose of pexidartinib treatment. |
| FG001 | Treatment-Free/Re-Treatment Cohort |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 25, 2022 |
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|
| Baseline up to Month 24 |
| Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | 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. | Baseline up to Month 24 |
| Number of Participants With and Without Progressive Disease | 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. | Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort) |
| Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort | 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. | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months |
| Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort | 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. | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months |
| Number of Patients Reporting AEs (Treatment-free) | 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. | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months |
| Los Angeles |
| California |
| 90033 |
| United States |
| Sylvester Comprehensive Cancer Center | Miami | Florida | 33136 | United States |
| Dana-Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Chris O'Brien Lifehouse | Camperdown | New South Wales | 2050 | Australia |
| Peter MacCallum Cancer Centre | East Melbourne | Victoria | 3000 | Australia |
| Magyar Honvedseg Egeszsegugyi Kozpont | Budapest | H-1062 | Hungary |
| Rizzoli-Istituto Ortopedico Rizzoli | Bologna | 40136 | Italy |
| Fondazione IRCC Istituto Nazionale dei Tumori | Milan | 20133 | Italy |
| Leiden University Medical Center (LUMC) | Leiden | 2333 ZA | Netherlands |
| Hospital Sant Pau | Barcelona | 08041 | Spain |
| Hospital Virgen del Rocio | Seville | 41013 | Spain |
| National Taiwan University Hospital | Taipei | Taiwan |
Previously-treated participants with TGCT who discontinued pexidartinib treatment (Treatment-Free Period) and had the option to resume pexidartinib treatment at dose of completion of prior study (Re-Treatment Period).
| Resumed Pexidartinib Treatment |
|
| COMPLETED |
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| NOT COMPLETED |
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Demographic and baseline characteristics were assessed in the Full Analysis Set.
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment Continuation Cohort | Previously-treated participants with TGCT who continued their current dose of pexidartinib treatment. |
| BG001 | Treatment-Free/Re-Treatment Cohort | Previously-treated participants with TGCT who discontinued pexidartinib treatment (Treatment-Free Period) and had the option to resume pexidartinib treatment at dose of completion of prior study (Re-Treatment Period). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment 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. | The number of treatment-free participants was assessed in participants with available data in the Full Analysis Set. | Posted | Count of Participants | Participants | Baseline up to 12 months after last participant enrolled in Cohort |
|
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| ||||||||||||||||||||||||||
| Primary | Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment 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. | The number of treatment-free participants was assessed in participants with available data in the Full Analysis Set. | Posted | Count of Participants | Participants | Baseline up to 24 months after last participant enrolled in Cohort |
|
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | 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. | PROMIS Physical Function was assessed in participants with available data in the Evaluable Analysis Set. | Posted | Mean | Standard Deviation | score on a scale | Baseline up to Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts | 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. | EQ-5D-5L was assessed in participants with available data in the Evaluable Analysis Set. | Posted | Mean | Standard Deviation | score on a scale | Baseline up to Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants With and Without Progressive Disease | 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. | Tumor assessments were analyzed in participants with available data in the Full Analysis Set. | Posted | Count of Participants | Participants | Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort) |
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| Secondary | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort | 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. | Treatment-emergent adverse events were assessed in participants with available data in the Safety Analysis Set. | Posted | Count of Participants | Participants | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort | 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. | Treatment-emergent adverse events were assessed in participants with available data in the Safety Analysis Set . | Posted | Count of Participants | Participants | Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Patients Reporting AEs (Treatment-free) | 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. | 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. | Posted | Count of Participants | Participants | 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 collected from baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months.
TEAEs observed in the Treatment Continuation Cohort and the Re-Treatment Period of the Treatment-free/Re-Treatment Cohort are reported. AEs from the Treatment-free Period of the Treatment-free/Re-Treatment Cohort were captured and are reported as well.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment Continuation Cohort | Previously-treated participants with TGCT who continued their current dose of pexidartinib treatment. | 0 | 21 | 2 | 21 | 19 | 21 |
| EG001 | Re-Treatment Period of Treatment-Free/Re-Treatment Cohort | Previously-treated participants with TGCT who discontinued pexidartinib treatment (Treatment-Free Period) and had the option to resume pexidartinib treatment at dose of completion of prior study (Re-Treatment Period). | 0 | 3 | 0 | 3 | 3 | 3 |
| EG002 | Treatment-Free Period of Treatment-Free/Re-treatment Cohort | Previously-treated participants with TGCT who discontinued pexidartinib treatment (Treatment-Free Period) and reported adverse events. | 0 | 11 | 0 | 11 | 7 | 11 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Creutzfeldt-Jakob disease | Infections and infestations | MedDRA26 | Systematic Assessment |
| |
| Device dislocation | Product Issues | MedDRA26 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA26 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA26 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA26 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA26 | Systematic Assessment |
| |
| Blood creatinine phosphokinase increased | Investigations | MedDRA26 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA26 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA26 | Systematic Assessment |
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| Blood lactate dehydrogenase increased | Investigations | MedDRA26 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA26 | Systematic Assessment |
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| White blood cell count decreased | Investigations | MedDRA26 | Systematic Assessment |
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| Lymphocyte count decreased | Investigations | MedDRA26 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA26 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA26 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA26 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA26 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA26 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA26 | Systematic Assessment |
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| Generalised oedema | General disorders | MedDRA26 | Systematic Assessment |
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| Hair colour changes | Skin and subcutaneous tissue disorders | MedDRA26 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA26 | Systematic Assessment |
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| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA26 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA26 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA26 | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA26 | Systematic Assessment |
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| Leukopenia | Blood and lymphatic system disorders | MedDRA26 | Systematic Assessment |
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| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA26 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA26 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA26 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Intevertebral disc protusion | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA26 | Systematic Assessment |
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| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA26 | Systematic Assessment |
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| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA26 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA26 | Systematic Assessment |
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| Cellulitis | Infections and infestations | MedDRA26 | Systematic Assessment |
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| Blood uric acid increased | Investigations | MedDRA26 | Systematic Assessment |
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| Renal colic | Renal and urinary disorders | MedDRA26 | Systematic Assessment |
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| Urinary retention | Renal and urinary disorders | MedDRA26 | Systematic Assessment |
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| Animal bite | Injury, poisoning and procedural complications | MedDRA26 | Systematic Assessment |
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| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA26 | Systematic Assessment |
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| Sciatica | Nervous system disorders | MedDRA26 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA26 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Contact for Clinical Trial Information | Daiichi Sankyo | 908-992-6400 | CTRinfo@dsi.com |
| Jul 3, 2024 |
| Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000070779 | Giant Cell Tumor of Tendon Sheath |
| ID | Term |
|---|---|
| D005870 | Giant Cell Tumors |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D013585 | Synovitis |
| D007592 | Joint Diseases |
| D009140 | Musculoskeletal Diseases |
| D052256 | Tendinopathy |
| D009135 | Muscular Diseases |
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| ID | Term |
|---|---|
| C000600259 | pexidartinib |
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| 41 to 64 years |
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| 65 to 74 years |
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| 75 to 84 years |
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| ≥ 85 years |
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| Male |
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| White |
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| Not collected per local regulations |
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