Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

Giant Cell Tumors Clinical Trial

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Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02948088
• Other study ID #: DS-ONC-01-15-EU
• Status: Completed
• Start date: November 18, 2016
• Est. completion date: March 11, 2021

Study information

• Verified date: January 2023
• Source: Daiichi Sankyo, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 183
• Est. completion date: March 11, 2021
• Est. primary completion date: March 11, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Written informed consent for participation in the study (ICF)

• Patients with d-TGCT (diagnosed histologically) confirmed naïve or recurrent case

Exclusion Criteria:

• As this is a non interventional study, no explicit exclusion criteria have been defined

Related Conditions & MeSH terms

• Giant Cell Tumor of Tendon Sheath
• Giant Cell Tumors

Locations

Country	Name	City	State
Austria	Medizinische Universität Graz	Graz
France	CHU Nantes	Nantes
Germany	Westdeutsches Tumorzentrum	Essen
Italy	Istituto Ortopedico Rizzoli	Bologna
Italy	Istituto Nazionale Tumori-Foundazione IRCCS	Milano
Netherlands	Leiden University Medical Center	Leiden
Netherlands	Radboud universitair medisch centrum	Nijmegen
Spain	Hospital Sant Pau	Barcelona
Spain	Hospital Universitario Virgen del Rocio	Sevilla
United Kingdom	The Royal Marsden Hospital	London
United Kingdom	Nuffield Orthopaedic Centre	Oxford
United States	University of Texas MD Anderson Cancer Center	Houston	Texas
United States	David Geffen School of Medicine at UCLA	Los Angeles	California
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	University of California San Francisco	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
Daiichi Sankyo, Inc.	Optimapharm

Countries where clinical trial is conducted

United States,  Austria,  France,  Germany,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT)	The management plan received among the patients was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT)	The current TGCT treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)	Type of treatment plan received among the patients was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)	The status of treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT)	Type of current TGCT treatment among the patients was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT)	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).	at Baseline
Primary	Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT)	Complications due to surgery were collected from information routinely recorded in the patient files / medical records.	at Baseline (prior to any d-TGCT therapy)
Primary	Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT)	Tumor recurrence was collected from information routinely recorded in the patient files / medical records.	at Baseline
Primary	Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT)	Time since most recent tumor recurrence was collected from information routinely recorded in the patient files / medical records.	at Baseline
Primary	Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT)	Time from baseline to first tumor recurrence was collected from information routinely recorded in the patient files / medical records.	Baseline up to end of observation period (approximately 2 years)
Primary	Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months	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.	Baseline up to end of observation period (approximately 2 years)
Primary	Mean Worst Stiffness Score From Baseline Through 24 Months	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.	Baseline up to end of observation period (approximately 2 years)
Primary	Mean PROMIS Physical Function Score From Baseline Through 24 Months	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.	Baseline up to end of observation period (approximately 2 years)
Secondary	Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months	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.	Baseline up to end of observation period (approximately 2 years)
Secondary	Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months	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.	13-24 months before baseline up to end of observation period (approximately 2 years)
Secondary	Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months	The median number of days in rehabilitation and the number of work days missed were also reported.	13-24 months before baseline up to end of observation period (approximately 2 years)
Secondary	Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT)	TGCT outcome status was collected from information routinely recorded in the patient files / medical records.	At end of observation period (approximately 2 years post-baseline)