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.

Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

Giant Cell Tumors

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.

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.

David Geffen School of Medicine at UCLA

University of California San Francisco

Memorial Sloan Kettering Cancer Center

University of Texas MD Anderson Cancer Center

Medizinische Universität Graz

CHU Nantes

Westdeutsches Tumorzentrum

Istituto Ortopedico Rizzoli

Istituto Nazionale Tumori-Foundazione IRCCS

Leiden University Medical Center

Radboud universitair medisch centrum

Hospital Sant Pau

Hospital Universitario Virgen del Rocio

The Royal Marsden Hospital

Nuffield Orthopaedic Centre

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.

All participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were enrolled in the study.

Overall

Tenosynovial Giant Cell Tumors

Age, Continuous

<40 years

≥40 and <60 years

≥60 years

Age, Customized

Sex: Female, Male

Race and Ethnicity were not collected from any participant.

Race and Ethnicity Not Collected

Austria

Netherlands

United States

Italy

United Kingdom

France

Germany

Spain

Region of Enrollment

Knee joint

Ankle joint

Shoulder joint

Foot

Elbow joint

Wrist joint

Hand

Temporomandibular

Tumor location/affected joint

Pain

Limited range of movement

Swelling

Stiffness

Patient unable to judge

Current symptoms

Not applicable

Missing

Most disturbing symptom

4 current symptoms

3 current symptoms

2 current symptoms

Unknown/missing

Number of participants with current symptoms

Demographic and baseline characteristics were assessed in the Full Analysis Set.

This was an observational study and the quality of data has not been verified 100%. Baseline data were checked during on-site visits for all patients enrolled in Europe. Due to the COVID-19 pandemic, the planned on-site monitoring visits in the US could not be performed. Instead, more frequent and detailed remote visits were performed for these sites. Follow-up data for all patients were regularly monitored remotely. A certain amount of incorrect/incomplete data has to be assumed for all sites.

Contact for Clinical Trial Information

Any prior or concomitant therapies for managing TGCT-related symptoms

Any changes to concomitant therapies for managing TGCT-related symptoms since the last visit

Any changes to TGCT treatment (including also planned treatments) since the last visit

All participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were enrolled at baseline in the study.

Baseline

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed within 1st year of the study.

Within 1st Year

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed within 2nd year of the study.

Within 2nd Year

Management plans were assessed in participants with available data in the Full Analysis Set. Participants may have be included in more than one category.

Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT)

Wait and see

Currently being treated or planned to be treated

Unknown/Missing

The current treatment plan (currently being treated or planned) was assessed in participants with no changes at any data collection point where the data of the previous reported data collection point is being reported in the Full Analysis Set.

Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT)

Radiotherapy

90Yttrium therapy

Systemic therapy

Surgery

Future surgery required

No current or planned treatment

Unchanged therapy

Type of treatment plans were assessed in participants with available data in the Full Analysis Set. Participants may have be included in more than one category.

Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)

Planned

Current

Status of treatment plan was assessed in participants currently being treated or planned to be treated in the Full Analysis Set. Participants with different reported treatment plans during the period (e.g. participants who may have switched treatment plans) are counted as 'currently being treated or planned to be treated' in the corresponding period.

Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)

Status of treatment plan was assessed in participants currently being treated in the Full Analysis Set. Participants may have been included in more than one category.

Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT)

Moderate diffuse

Severe diffuse

Not assessable

Tumor severity was assessed in participants in the Full Analysis Set.

Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT)

Complications were assessed among participants with documented surgeries.

Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT)

1 recurrence

2 recurrences

3 recurrences and more

All participants with diffuse tenosynovial giant cell tumors (d-TGCT) with tumor recurrence.

Participants With Tumor Recurrence

Tumor recurrence was assessed in participants with available baseline data in the Full Analysis Set.

Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT)

All participants with diffuse tenosynovial giant cell tumors (TGCT) who were enrolled in the study.

Time since last recent tumor recurrence until baseline was assessed in participants with available baseline data in the Full Analysis Set.

Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT)

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed for tumor recurrence within 1st year of the study.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed for tumor recurrence within 2nd year of the study.

All participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed for tumor recurrence within the whole observation period.

Time from baseline to first tumor recurrence was assessed in participants with available data in the Full Analysis Set.

Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT)

Brief Pain Inventory Pain Severity

Brief Pain Inventory Pain Interference

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who had BPI Pain Severity and Pain Interference assessments conducted at baseline.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who had BPI Pain Severity and Pain Interference assessments conducted at 6 months.

6 Months

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who had BPI Pain Severity and Pain Interference assessments conducted at 12 months.

12 Months

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who had BPI Pain Severity and Pain Interference assessments conducted at 18 months.

18 Months

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who had BPI Pain Severity and Pain Interference assessments conducted at 24 months.

24 Months

Brief Pain Inventory Pain Severity and Pain Interference were assessed in participants with available data in the Full Analysis Set.

Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed worst stiffness in last 24 hours (patient reported outcome) at baseline.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed worst stiffness in last 24 hours (patient reported outcome) at 6 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed worst stiffness in last 24 hours (patient reported outcome) at 18 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed worst stiffness in last 24 hours (patient reported outcome) at 24 months.

Worst stiffness was assessed in participants with available data in the Full Analysis Set.

Mean Worst Stiffness Score From Baseline Through 24 Months

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their physical function at baseline.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their physical function at 6 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their physical function at 12 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their physical function at 18 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their physical function at 24 months.

PROMIS physical function was assessed in participants with available data in the Full Analysis Set.

Mean PROMIS Physical Function Score From Baseline Through 24 Months

EQ-5D Index Score

EQ-5D VAS

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their quality of life at baseline.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their quality of life at 6 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their quality of life at 12 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their quality of life at 18 months.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who assessed their quality of life at 24 months.

Quality of life assessed by EQ-5D index score was assessed in participants with available data in the Full Analysis Set.

Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months

General practitioner visits

Specialists visits

Physical therapy sessions

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed and categorized by tumor status within 13-24 months before baseline.

13-24 Months Before Baseline

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed and categorized by tumor status within 1-12 months before baseline.

1-12 Months Before Baseline

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed and categorized by tumor status within 1st year of the study.

Participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed and categorized by tumor status within 2nd year of the study.

All participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were assessed and categorized by tumor status within the whole observation period.

Within Whole 2-year Period

Health resource utilization was assessed in participants with available data in the Full Analysis Set.

Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months

Days in rehabilitation

Work days missed

Number of days in rehabilitation was assessed in the Full Analysis Set. A participant could have documented days in rehabilitation for the 1st year visit as well as for the 2nd year visit which resulted in a sum greater than the single results per visit/year.

Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months

TCGT status at end of observational period: No evidence of disease (NED)

TCGT status at end of observational period: Radiological residual disease

TCGT status at end of observational period: Radiological recurrent disease

TCGT status at end of observational period: Missing

NED (only pts with NED): With complaints

NED (only pts with NED): Without complaints

NED (only pts with NED): Missing

Radiological residual disease (only pts with radiological residual disease): Watchful waiting

Radiological residual disease (only pts with radiological residual disease): Awaiting surgery

Radiological residual disease (only pts with radiological residual disease): Awaiting medication

Radiological residual disease (only pts with radiological residual disease):Awaiting other treatment

Radiological residual disease (only pts with radiological residual disease): Missing

TGCT outcome status was assessed in participants with available data in the Full Analysis Set.

Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

About this trial

Study ID

Status

Type

Placebo

Accepting

Trial Timing

What is this trial about?

What are the participation requirements?