Neoplasms

Skin and Connective Tissue Diseases

All Conditions

Breast Neoplasms

Breast Diseases

Skin Diseases

Antineoplastic Agents

All Drugs and Chemicals

Fulvestrant

Exemestane

Letrozole

Anastrozole

Aromatase Inhibitors

Ribociclib + letrozole, or Ribociclib + anastrozole, or Ribociclib + exemestane, or Ribociclib + fulvestrant

Ribociclib + letrozole, or Ribociclib + anastrazole, or Ribociclib + exemestane, or Ribociclib + fulvestrant

First-line Ribociclib + endocrine therapy

First-line endocrine therapy

First-line chemtherapy

Novartis Pharmaceuticals

This is a non-interventional observational study conducted in Germany to evaluate the real-world effectiveness, tolerability, safety, and quality of life in patients with locally advanced/metastatic HR+/HER2- breast cancer treated with one of the following 1st line treatments: Ribociclib + AI/FUL, or endocrine monotherapy, or chemotherapy

This non-interventional study collects data from clinical practice capturing on effectiveness, safety and tolerability, duration of therapy, and quality of life of ribociclib in combination with an aromatase inhibitor/fulvestrant in daily routine and in line with the respective current German summary of product characteristics. In order to put these results into perspective, data is also collected on patients treated with endocrine therapy or chemotherapy for first line locally advanced or metastatic breast cancer. To gain insight into algorithms and outcome of sequential therapy, up to three lines of treatment are documented within this study. The information gathered and evaluated in this NIS is helping to answer open questions for the treatment of locally advanced/metastatic breast cancer and provides first insights into the treatment reality with ribociclib in this setting. This includes, but is not limited to, the questions on baseline demographics leading to a specific treatment decision, as well as efficacy and clinical routine related to treatment sequencing. In addition, data on the mutation status (including PIK3CA and BRCA1/2) is collected at different time points to generate insights into the clinical routine of mutation testing and to understand its impact on therapy sequencing.

A Non-interventional Study for Women With HR-positive, HER2-negative Locally Advanced or Metastatic Breast Cancer to Evaluate the Real-world Effectiveness of Treatment Algorithms Beginning With Ribociclib + AI/FUL, or With Endocrine Therapy or Chemotherapy as First Line Treatment

A Non-interventional Study for Women With HR-positive, HER2-negative Locally Advanced or Metastatic Breast Cancer to Evaluate the Real-world Effectiveness of Treatment Algorithms Beginning With Kisqali (Ribociclib) in Combination With an Aromatase Inhibitor/Fulvestrant, or With Endocrine Therapy or Chemotherapy as First Line Treatment

Progression-free survival was defined as first intake of study medication in current-line therapy up to first documented progress or death from any cause in this treatment line. If a patient had not had an event, progression-free survival was censored at the end of the respective therapy line.

Cox proportional hazard regression were calculated with the covariates treatment cohort, age group, previous therapy, ECOG performance status, grading status and metastasis status for the full analysis set excluding patients with missing data in covariates.

Propensity score matching (PSM) was performed to compare outcome between cohorts and performed separately for the Ribociclib+AI/FU vs. Chemotherapy cohort resp. the Ribociclib+AI/FU vs. Endocrine therapy cohort in a 5:1 ratio.

Sequential progression-free survival up to second-line therapy was defined as time between first intake of study medication in first-line therapy up to progress or death after second-line therapy. Sequential progression-free survival up to third-line therapy was defined as time between first intake of study medication in first-line therapy up to progress or death after third-line therapy. Sequential progression-free survival for all further therapy lines were analyzed according to the definitions above.

Conditional progression-free survival in first-line therapy was defined as PFS for patients with a period of up to 6, 12, or 24 months from start of study treatment (first intake) up to first progression.

This means that all patients with progression or death within the first 24 months of first-line therapy are classified into one of the three subgroups: Progression in month 0 to 6, Progression in month 7 to 12 and Progression in month 13 to 24. For these subgroups, the PFS is presented separately.

Time to treatment failure (TTF) from current-line therapy was defined as time between first intake of study medication in current-line therapy up to therapy discontinuation because of progress, adverse events leading to discontinuation or death.

Time to first chemotherapy was defined as time between first intake of study medication in first-line therapy up to first application of chemotherapy for patients enrolled in 1st line option ribociclib + AI/FU or endocrine therapy.

Time to next treatment / therapy (TTNT) was defined as time between first intake of study medication in first-line therapy up to first intake of medication in the next therapy line.

Overall survival (OS) was defined as time between first intake of study medication in first-line therapy up to death from any cause. If a patient was not known to have died, survival was censored at the date of last contact.

Patient adherence for ribociclib was assessed using the MMAS-8 questionnaire. This questionnaire was only send to patients enrolled in the first-line option ribociclib + AI/FU. The score was analyzed using descriptive statistics and adherence categories low, medium and high were displayed by visit. Questionnaires were only included if all questionnaire items were answered.

The total scale has a range of 0 to 8.0 where:

* Low Adherence (\< 6)
* Medium Adherence (6 to \<8)
* High Adherence (= 8)

Number of participants with mutations will be collected

Quality of life during documented therapy lines was assessed using the EORTC QLQ-C30. Scale and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.

Quality of life during documented therapy lines was assessed using the HADS-D. Scale ranges between 0 and 21, a high score means a high level of anxiety or depression.

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Symptoms and General Pathology

Immune System Diseases

Prostatic Neoplasms

Neoplasm Metastasis

Hypersensitivity

Genital Neoplasms, Male

Urogenital Neoplasms

Genital Diseases

Genital Diseases, Male

Urogenital Diseases

Prostatic Diseases

Male Urogenital Diseases

Reproductive Control Agents

Docetaxel

Leuprolide

Goserelin

Triptorelin Pamoate

Tubulin Modulators

Antimitotic Agents

Antineoplastic Agents, Hormonal

Contraceptive Agents

Contraceptive Agents, Female

Contraceptive Agents, Hormonal

abiraterone

enzalutamide

docetaxel

apalutamide

cabazitaxel

Radium-223

Lutetium-177

degarelix

goserelin

leuprorelin

triptorelin

Pfizer CT.gov Call Center

Comprehensive understanding of the epidemiology and disease burden of metastatic prostate cancer patients in Finland is lacking. This study will address the following questions:

* What are the demographic and clinical characteristics of metastatic prostate cancer patients?
* How are metastatic prostate cancer patients currently treated and how effective are these treatments?
* How does the development of castration-resistance affect patient outcomes?
* What is the economic burden of metastatic prostate cancer?

Male patients with cancer of the prostate.

Real World Evidence Study on Metastatic Prostate Cancer in the Pirkanmaa Hospital District in Finland

Real World Evidence Study on Metastatic Prostate Cancer Patient Characteristics, Treatment Patterns and Outcomes in the Pirkanmaa Hospital District in Finland

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Demographical and clinical variables of patients will be summarized

Pfizer

Respiratory Tract (Lung and Bronchial) Diseases

Carcinoma, Non-Small-Cell Lung

Lung Neoplasms

Respiratory Tract Neoplasms

Thoracic Neoplasms

Lung Diseases

Respiratory Tract Diseases

All eligible patients who meet all inclusion criteria and none of the exclusion criteria

This is a non interventional, observational, retrospective study including patients with EGFR-mutated advanced NSCLC, treated with 1st or 2nd generation EGFR TKI therapy in first line

Non Interventional

Konstantinos Syrigos, Prof.

Multinational, multi-center medical record review to describe the treatment patterns, clinical outcomes, and EGFR / T790M testing practices in EGFR-mutated advanced NSCLC patients receiving first-line EGFR TKI therapy in Europe.

The aim is to generate real-life data on treatment patterns of the EGFR-mutated advanced NSCLC patients receiving first- or second-generation EGFR TKI in first line of therapy as provided per routine practice in eight countries. An approximate number of 820 patients will take part in the REFLECT study.

It is anticipated that data, generated from the proposed large cohort across centers in Europe, will facilitate a better understanding of unmet medical needs in first line of treatment and will provide a platform for improving patients' management.

Treatment Patterns, Outcomes and Testing in EGFRm NSCLC Patients With EGFR TKI 1L Across Europe (REFLECT)

Real World Treatment Patterns, Clinical Outcomes and EGFR / T790M Testing Practices in EGFR-Mutated Advanced Non-Small Cell Lung Cancer Patients Receiving First-Line TKI Therapy

Frequency distribution of first-line EGFR TKI of first- or second generation prescribed: erlotinib, gefitinib, or afatinib

Proportion of patients with a progression event during first-line EGFR TKI therapy or other evidence recorded in the patient's medical records deemed by the clinician to be indicative of progression; Time to progression, defined as time from EGFR TKI therapy initiation in the first-line locally advanced or metastatic setting until the earliest of progression death, or end of available follow-up (i.e., progression-free survival); Proportion of patients discontinuing first-line EGFR therapy for reasons other than progression event or death

Proportion of patients prescribed second-line therapy, as well as time to second-line therapy initiation, as defined by time from first-line EGFR TKI discontinuation until the earliest of second-line initiation, death, or end of available follow-up; Frequency distribution of the second-line therapy regimen (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy) among patients initiating second-line therapy.

Patients' demographics, including: sex (male/female), and age at EGFR TKI initiation.

Baseline disease characteristics, including:

Smoking status at initial NSCLC diagnosis, Tumor histology and disease stage at initial NSCLC diagnosis, Eastern Cooperative Oncology Group (ECOG) performance status at initial NSCLC diagnosis and at EGFR TKI initiation, Treatment history: for patients initially diagnosed at earlier-stage disease, cancer-directed and palliative therapies received before first diagnosis of or progression to advanced/metastatic EGFR-mutated NSCLC.

Molecular testing patterns, including: type of sample/test used to assess the EGFR mutation, for example: primary/secondary tumor, tissue/cytology or blood sample; Molecular testing results: EGFR mutation type (exon 19 deletion, L858R mutation, other).

Incidence and time to development of BM from first diagnosis of locally advanced or metastatic NSCLC, including:

Proportion of patients with BM since diagnostic of metastatic disease and proportion of patients with BM developed during treatment, Type of test used to confirm BM diagnosis (e.g., tissue biopsy, imaging, spinal tap, neurologic exam), Proportion of patients who have BM at start of first-line EGFR TKI therapy. Proportion of patients with no development of BM, Patients' demographic and clinical characteristics associated with development of BM, Incidence of BM and time to development of BM from start of first-line EGFR TKI therapy, Proportion of patients prescribed treatments for BM and type of therapy Overall survival (in months) measured from first diagnosis of BM to the date of death from any cause, with patients last known to be alive censored at the date of last available follow-up.

Incidence and time to development of LM disease from first diagnosis of locally advanced or metastatic NSCLC, including:

Proportion of patients with LM at first diagnosis of metastatic disease, Type of test to confirm LM diagnosis (cerebrospinal fluid cytology, tissue, imaging, etc.), Proportion of patients who have LM at start of first-line EGFR TKI therapy, Proportion of patients who later develop (during treatment) LM, Proportion of patients with no development of LM, Incidence of LM and time to development of LM from start of first-line EGFR TKI therapy.

Overall survival (in months) measured from first diagnosis of LM disease to the date of death from any cause, with patients last known to be alive censored at the date of last available follow-up.

Proportion of patients prescribed other systemic treatments (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy) in combination with first-line EGFR TKI therapy.

Proportion of patients tested for T790M mutation and among those tested, the proportion who were positive for the mutation, including:

Binary distribution of T790M mutation testing and, among those tested, the proportion with a positive result, Type of test used to assess the T790M mutation (tissue/cytology or blood sample).

Among patients tested and positive for T790M mutation, what proportion receives treatment with osimertinib - applicable study measures include binary distribution of osimertinib treatment initiation, and requency distribution of the line of therapy (second- or later-line in the metastatic setting) in which osimertinib was initiated.

Among patients progressing on first-line EGFR TKI therapy and tested for T790M, proportion who receive (or not) second-line therapy and the second-line therapy selection (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy), including:

Proportion of patients prescribed second-line therapy, as well as time to second-line therapy initiation, as defined by time from first-line EGFR TKI discontinuation until the earliest of second-line initiation, death, or end of available follow-up, Among patients initiating second-line therapy, frequency distribution of the second-line therapy regimen observed (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy).

Among patients progressing on first-line EGFR TKI therapy and not tested for T790M, proportion who receive (or not) second-line therapy and the second-line therapy selection (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy), including:

Proportion of patients prescribed second-line therapy, as well as time to second-line therapy initiation, as defined by time from first-line EGFR TKI discontinuation until the earliest of second-line initiation, death, or end of available follow-up, Among patients initiating second-line therapy, frequency distribution of the second-line therapy regimen observed (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy).

Proportion of patients prescribed second-line therapy, as well as time to second-line therapy initiation, as defined by time from first-line EGFR TKI discontinuation until the earliest of second-line initiation, death, or end of available follow-up among patients tested and not tested for T790M mutation.

Proportion of patients prescribed third- or later lines of therapy and type of therapies selected (chemotherapy, osimertinib, other EGFR TKI, I/O therapy, or other therapy), and how these proportion varies by the second-line therapy category received.

OS (in months) measured overall from first diagnosis of or progression to locally advanced or metastatic disease, OS from start of first-line EGFR TKI therapy to the date of death from any cause, with patients last known to be alive censored at the date of last available follow-up.

Proportion of patients with other positive mutations, in case additional molecular testing were performed at the time of initial EGFR testing or at the time of T790M testing (e.g., KRAS, BRAF, ROS1 translocation, PD-L1 expression, MET amplification, ALK rearrangement, RET rearrangement, HER2 exon 20 insertion, TP53, others).

AstraZeneca

Carcinoma

Carcinoma, Bronchogenic

Bronchial Neoplasms

Amino Acids

Osimertinib

Mitogens

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

The recommended dose is 80 mg osimertinib once a day until disease progression or unacceptable toxicity according to the prescription information and clinical practice. It can be taken with or without food at the same time each day.

The results of phase III FLAURA study showed a significant PFS benefit for first-line Osimertinib versus standard EGFR-TKIs in patients with EGFR mutation-positive NSCLC, the median PFS was 18.9 months and 10.2 months, respectively. However, only 136 Chinese patients were enrolled in FLAURA study. The objectives of this study are to assess the efficacy and safety of Osimertinib in a real world setting in Chinese patients with locally advanced or metastatic, treatment naïve, epidermal growth factor receptor (EGFR) mutation-positive Non-Small Cell Lung Cancer (NSCLC).

Total of \~30 study sites are selected for conducting this observational study. Eligible patients will be prospectively and consecutively included. Therefore, the clinical practice in the selected patients group can represent the "real-world" situation in China, and the patient's medical record will be well documented and archived in those hospitals. All data defined in the protocol will be collected during the study and entered in the Electric Data Capture (EDC), being consistent to the patients' medical records.

The most important bias of the study is that patients' characteristics will affect the treatment duration, efficacy and safety, such as, higher proportion of patients with WHO PS 2\~3 enroll in the study will result in shorter TTD, poorer effectiveness and higher toxicities than expected. The \~30 sites are not randomly selected and potential selection bias exists. To minimize enrolment bias, the patients who are eligible and consent to participate in the current study will be enrolled consecutively as per protocol and without personal preference from investigators.

The self-selection bias may exit for the willingness and non-willingness participants. We'll try our best to discuss with the non-willingness participants to make sure the consistency/comparative between the willingness and non-willingness participants.

There could be a certain percentage of patients who would lost to follow up, it is unavoidable in clinical study, and is more common in real world study. We can minimize the bias by selecting the hospitals with normative and high-quality clinical practice, trying to collect the reason of lost to follow up and enhancing patient management during the follow up. The above bias is acceptable as this is a "real-world" study. Only descriptive analysis will be performed for the primary, secondary and exploratory objectives. No statistical comparisons between subgroups will be done.

Blood Sample Collection and Retention: about 10 mL Whole human blood samples will be collected using streck tube, sent to the NGS lab and plasma will be extracted for storage at -80℃. until test.

Tissue Sample Collection and Retention At baseline, fresh tissue samples (or cytological samples) will be collected or 6 slices of tissue sample olefin resections obtained from surgery or 10 slices of tissue samples olefin resection obtained from puncture biopsy will be provide to central laboratory for biomarker analysis. Before tissue sample olefin resection sent to central laboratory, a pathological quality should be conducted to ensure the percentage of tumor cells is greater than 10%. Tissue sample olefin resection will be labeled and put in a box and store at room temperature

First Line Osimertinib for EGFR Mutation-positive Non-Small Cell Lung Cancer in Real World Chinese Setting

A Prospective, National, Multi-centric, Non-interventional Study of First Line Osimertinib in Chinese Patients With Locally Advanced/Metastatic，EGFR Mutation-positive NSCLC in Real World Setting

Time to discontinuation (TTD), is defined as the time from the date of first dose of Osimertinib in this study until the date of Osimertinib discontinuation for any reason including disease progression, treatment toxicity, death or other reason as recorded in CRF. Subjects who are still on treatment at the time of analysis will be censored at the date of last dose received. Lost to follow-up patients will be censored at last documented contact with patient status "on treatment".

Progression-free survival (PFS), is defined as the time from the date of first dose of Osimertinib in this study until the date of disease progression as recorded in CRF or death (by any cause in the absence of progression) regardless of whether the subject withdraws from therapy or receives another anti-cancer therapy prior to progression, which usually refer to Response Evaluation Criteria In Solid Tumours (RECIST) in clinical practice. Subjects who have not progressed or died at the time of analysis will be censored at the time of the latest date of assessment. If the subject has no evaluable visits after the baseline visit, they will be censored at 0 days unless they die before the planned visit after the baseline visit. Lost to follow-up patients who have not progressed will be censored at last documented contact with patient status "non-progression".

Progression-free survival rate (PFS rate), is defined as the percentage of patients who do not progress on Osimertinib treatment

Objective Response Rate (ORR), is defined as the percentage of patients with complete response or partial response by investigator assessment as recorded in the CRF, which usually refer to Response Evaluation Criteria In Solid Tumours (RECIST) in clinical practice.

Disease Control Rate (DCR), is defined as the percentage of patients with non-progression by investigator assessment as recorded in the CRF, which usually refer to Response Evaluation Criteria In Solid Tumours (RECIST) in clinical practice.

Defined as the proportion of patients who are still alive at a particular time in the study (eg, 1 year or 2 years). The patient should be contacted 1 week after the termination of the corresponding OS analysis data to determine survival status. Lost to follow-up patients who have not progressed will be censored at last documented contact with patient status "survival".

The mutation rate of de novo T790M test by high sensitive technique (analyzed by Next Generation Sequencing platform).

de novo T790M

Incidence of Adverse Events (AEs): Nature, incidence, severity and seriousness of adverse events, Incidence of Serious Adverse Events (SAEs), which usually be graded by CTCAE v4.03 based on current clinical practice.

First Affiliated Hospital of Zhejiang University

Behaviors and Mental Disorders

Quality of Life

QOL assessments using the ESAS, CPC, EQ5D every 4-8 weeks +/- 2 weeks

CT radiographic imaging every 12 weeks +/- 2 weeks

CT imaging

Samantha Pollard, PhD

Cheryl Ho, MD

This pilot clinical trial aims to assess the real world quality of life and survival of patients treated with targeted therapy that has preliminary evidence of efficacy in subjects with advanced rare cancers or cancer harbouring rare molecular aberrations. The treatment has been granted conditional or full approved by Health Canada (HC) as effective and safe. Due to the rarity of the cancer or molecular aberration the uncertainty level of the health technology assessment (HTA) by the pan Canadian Oncology Review (pCODR) was too high for consideration of funding or it was not submitted for consideration. Consequently, the goal of this study is to generate real world evidence to support HTA decision making throughout the life cycle of the product.

In Canada, adoption of new systemic therapies for cancer treatment is a multi-step process. After completion of clinical trials, pharmaceutical companies submit a request for a new drug approval to Health Canada. Therapies undergo rigorous review at Health Canada to confirm safety and efficacy before a Notice of Compliance (NOC) or NOC conditional (NOC/c) is issued that allows sale of the new medication in Canada. An additional layer of evaluation, a health technology assessment (HTA), is undertaken through the pan Canadian Oncology Drug Review (pCODR). A positive recommendation from pCODR is required for national price negotiations through the pan Canadian Pharmaceutical Alliance (pCPA). Once this step is completed, provincial pharmacies establish local contracts to permit local implementation of the new therapy.

As our understanding of pathogenesis evolves and next generation sequencing becomes more affordable, rare molecular entities amenable to treatment with targeted or selected therapies, like immunotherapy, are being increasingly identified. These molecular aberrations can occur with frequencies of 1-2% making it unfeasible to conduct large randomized trials to establish benefit. Determination of the added value of these new therapies in terms of outcomes and quality of life is challenging in the absence of comparators in trials resulting in increased uncertainty in terms of outcomes, quality of life (QOL) and cost-effectiveness. To address the uncertainty, pharmaceutical companies and the pCODR economic guidance panel create economic models with sensitivity analyses to try to estimate the cost-effectiveness of therapy. These models however, are dependent on the nature and quality of available data.

This study proposes to use real world data to generate evidence to assist in evaluate new therapies in rare molecular entities. The key components include regular interval radiographic imaging and collection of patient reported outcomes using standardized QOL questionnaires. The aim is to provide high quality real world evidence for assessment and economic modelling to reduce uncertainty and facilitate HTA decision-making.

PRecision Oncology Evidence Development in Cancer Treatment - Clinical: PREDiCTc

British Columbia Cancer Agency

Her2 positive mBC patients who have received at least 3 lines of treatment in the metastatic setting.

No intervention

Gerald Batist

This retrospective observational study will collect Canadian real-world data (RWD) from HER2+ metastatic breast cancer (mBC) patients to describe treatment sequences of all therapies received in the metastatic setting, to measure overall survival (OS), progression free survival (PFS), time to next active anti-cancer therapy, and to estimate the health resources utilization (HRU) during third-line therapy.

The proportion of treatment received during all lines of therapy in the metastatic setting will be described. The 2-year survival rate and median OS with interquartile range (IQR) for third-line therapy will be calculated. HRU data and costs during third-line therapy will be collected.

RWD Study in HER2+ mBC Patients in Third-Line Therapy

Real-World Data Study in HER2+ Metastatic Breast Cancer Patients in Third-Line Therapy

The results of this retrospective observational study will not be formally documented in a Clinical Study Report. Coded patient-level data will be shared with the sponsor.

The proportion of each type of treatment received for all lines of therapy in the metastatic setting will be determined.

The proportion of HER2+ mBC having brain metastasis at start of third lineas well as number of patients developing brain metastasis during third line will be quantified.

The proportion of each type of radiation therapy received during third line, the time between brain metastasis detection and whole brain radiation (WBR), and the proportion of patients receiving a second WBR before death will be quantified.

The 2-year survival rate and median OS in third-line HER2+ mBC patients will be quantified.

The median PFS in third-line HER2+ mBC patients will be quantified.

The median time to next active anticancer treatment in third-line HER2+ mBC patients will be quantified .

Information regarding HRU from HER2+ mBC patients in Canada during third-line therapy will be analyzed.

Knight Therapeutics

Exactis Innovation

Musculoskeletal Diseases

Wounds and Injuries

Nervous System Diseases

Rare Diseases

Synovitis

Giant Cell Tumors

Giant Cell Tumor of Tendon Sheath

Synovitis, Pigmented Villonodular

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Joint Diseases

Tendinopathy

Muscular Diseases

Pigmented Villonodular Synovitis

Global Clinical Leader

TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with diffuse TGCT (d-TGCT), the burden of d-TGCT for patients (including pain, joint stiffness, swelling, reduced mobility and quality of life) or the economic impact of d-TGCT.

This study aims to collect data by an observational disease registry involving no intervention to the patient or changes to investigators treatment decisions.

GCT is a rare, benign, but potentially locally aggressive and recurrent disease. Treatment pattern and treatment initiation, continuation or changes are solely at the discretion of the physician and the patient.

There will be no attempt to influence the prescribing patterns of any individual treating physician. All medication will be prescribed in the usual standard of care and will not be provided by the study sponsor.

Participation in the study will in no way influence payment or reimbursement for any treatment received by patients during the study.

It is the responsibility of the investigator and his study staff to enter all relevant patient data required for this registry in the electronic Case Report Form (eCRF) and in the patients' medical records.

Approximately 15 sites from up to 6 European Countries (France, Germany, Italy, The Netherlands, Spain and United Kingdom) and 4 sites from the US are planned to participate. The sites will be specialized sites that treat d-TGCT regularly.

Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

A Disease Registry for Patients With Tenosynovial Giant Cell Tumors (TGCT), Also Known as Pigmented Villonodular Synovitis (PVNS) and Giant Cell Tumor of the Tendon Sheath (GCT-TS)

The management plan received among the patients was collected from information routinely recorded in the patient files / medical records.

The current TGCT treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.

Type of treatment plan received among the patients was collected from information routinely recorded in the patient files / medical records.

The status of treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.

Type of current TGCT treatment among the patients was collected from information routinely recorded in the patient files / medical records.

Tumor severity classification was collected from information routinely recorded in the patient files / medical records. ). Tumor severity classification was based on MRI. In this classification scheme, moderate diffuse TGCT is characterized by intra- and/or extra-articular disease, without or with involvement of muscle/tendinous tissue/ligaments. Severe diffuse TGCT is characterized by intra- and extra-articular involvement and involvement of at least one of the 3 structures (muscle/tendinous tissue/ligaments).

Complications due to surgery were collected from information routinely recorded in the patient files / medical records.

Tumor recurrence was collected from information routinely recorded in the patient files / medical records.

Time since most recent tumor recurrence was collected from information routinely recorded in the patient files / medical records.

Time from baseline to first tumor recurrence was collected from information routinely recorded in the patient files / medical records.

The BPI short form is a self administered questionnaire that assesses severity of pain, impact of pain on daily function, location of pain, pain medications and amount of pain relief in the past 24 hours or the past week. The BPI pain severity and interference score ranges from 0 (pain does not interfere) to 10 (pain completely interferes), where lower scores indicate better clinical outcome.

The Worst Stiffness numerical rating scale (NRS) is a one-item self-administered, patient-reported outcome questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranges from 0 ("no stiffness") to 10 ("stiffness as bad as you can imagine"), where lower scores indicate better clinical outcome.

PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health. Physical function scores range from 0 (worst physical function) to 100 (best physical function, where higher scores indicate better clinical outcome.

The EQ-5D-5L questionnaire measures the patient's quality of life (QoL) based on the 5 dimensions mobility, self care, usual activities, pain/discomfort, and anxiety/depression. The EQ-5D index score ranges from 0 (worst QoL) to 1 (best QoL), where lower scores indicate worse clinical outcome. The EQ-5D VAS reports patient's self rated health ranging from 0 (worst health) to 100 (best health you can imagine), where lower scores indicate worse clinical outcome.

Health resource utilization was assessed by mean number of general practitioner visits, specialist visits, and physical therapy sessions. In addition, the mean number of days in rehabilitation and the number of work days missed were also reported.

The median number of days in rehabilitation and the number of work days missed were also reported.

TGCT outcome status was collected from information routinely recorded in the patient files / medical records.

Optimapharm

Daiichi Sankyo

Treatment-emergent adverse events and serious adverse events were not assessed in this non-interventional study. Adverse drug reactions suspected to be related to pexidartinib were only collated for patients from the United States.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Non-interventional Study for Women With HR-positive, HER2-negative Locally Advanced or Metastatic Breast Cancer to Evaluate the Real-world Effectiveness of Treatment Algorithms Beginning With Ribociclib + AI/FUL, or With Endocrine Therapy or Chemotherapy as First Line Treatment	NCT06311383	Breast Cancer	First-line Ribo... First-line endo... First-line chem...	18 Years -	Novartis
Real World Evidence Study on Metastatic Prostate Cancer in the Pirkanmaa Hospital District in Finland	NCT05701007	Metastatic Cast... Metastatic Cast...	abiraterone enzalutamide docetaxel apalutamide cabazitaxel Radium-223 Lutetium-177 degarelix goserelin leuprorelin triptorelin	18 Years -	Pfizer
Treatment Patterns, Outcomes and Testing in EGFRm NSCLC Patients With EGFR TKI 1L Across Europe (REFLECT)	NCT04031898	Lung Cancer	Non Interventio...	-	AstraZeneca
First Line Osimertinib for EGFR Mutation-positive Non-Small Cell Lung Cancer in Real World Chinese Setting	NCT04391283	Carcinoma, Non-...	Osimertinib	18 Years -	First Affiliated Hospital of Zhejiang University
PRecision Oncology Evidence Development in Cancer Treatment - Clinical: PREDiCTc	NCT04814095	Cancer	QOL CT imaging	18 Years -	British Columbia Cancer Agency
Treatment Patterns, Outcomes and Testing in EGFRm NSCLC Patients With EGFR TKI 1L Across Europe (REFLECT)	NCT04031898	Lung Cancer	Non Interventio...	-	AstraZeneca
RWD Study in HER2+ mBC Patients in Third-Line Therapy	NCT04566458	Breast Cancer S...	No intervention	18 Years -	Exactis Innovation
First Line Osimertinib for EGFR Mutation-positive Non-Small Cell Lung Cancer in Real World Chinese Setting	NCT04391283	Carcinoma, Non-...	Osimertinib	18 Years -	First Affiliated Hospital of Zhejiang University
Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project	NCT02948088	Giant Cell Tumo...		18 Years -	Daiichi Sankyo
RWD Study in HER2+ mBC Patients in Third-Line Therapy	NCT04566458	Breast Cancer S...	No intervention	18 Years -	Exactis Innovation
A Study of Participants With Squamous Cell Oesophageal Carcinoma Receiving Nivolumab as Part of an Early Access to Medicines Scheme (EAMS) Program in the United Kingdom	NCT04936399	Esophageal Squa...	Nivolumab	18 Years -	Bristol-Myers Squibb
PRecision Oncology Evidence Development in Cancer Treatment - Clinical: PREDiCTc	NCT04814095	Cancer	QOL CT imaging	18 Years -	British Columbia Cancer Agency

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