Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project (TOP)

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)

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.

Study Overview

• Status: Completed
• Conditions: Giant Cell Tumors

Detailed 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.

• Study Type: Observational
• Enrollment (Actual): 183

Contacts and Locations

Study Locations

• Austria
  • Graz, Austria, 8036
    • Medizinische Universität Graz
• France
  • Nantes, France, 44093
    • CHU Nantes
• Germany
  • Essen, Germany, 45122
    • Westdeutsches Tumorzentrum
• Italy
  • Bologna, Italy, 40136
    • Istituto Ortopedico Rizzoli
  • Milano, Italy, 20133
    • Istituto Nazionale Tumori-Foundazione IRCCS
• Netherlands
  • Leiden, Netherlands, 2333
    • Leiden University Medical Center
  • Nijmegen, Netherlands, 6525 GA
    • Radboud Universitair Medisch Centrum
• Spain
  • Barcelona, Spain, 08041
    • Hospital Sant Pau
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen Del Rocio
• United Kingdom
  • London, United Kingdom, SW3 6JJ
    • The Royal Marsden Hospital
  • Oxford, United Kingdom, OX3 7LD
    • Nuffield Orthopaedic Centre
• United States
  • California
    • Los Angeles, California, United States, 90095
      • David Geffen School of Medicine at UCLA
    • San Francisco, California, United States, 94158
      • University of California San Francisco
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This study enrolled patients with d-TGCT.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)
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)
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)
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)
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)
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
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)
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
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
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)
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)
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)
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)
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)
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)
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)

Collaborators and Investigators

• Sponsor: Daiichi Sankyo, Inc.
• Collaborators: Optimapharm
• Investigators: Study Director: Global Clinical Leader, Daiichi Sankyo, Inc.

Publications and helpful links

General Publications

• Bernthal NM, Spierenburg G, Healey JH, Palmerini E, Bauer S; TOPP Study Group; Gelderblom H, Staals EL, Lopez-Bastida J, Fronk EM, Ye X, Laeis P, van de Sande MAJ. The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study. Orphanet J Rare Dis. 2021 Apr 29;16(1):191. doi: 10.1186/s13023-021-01820-6.

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2016
• Primary Completion (Actual): March 11, 2021
• Study Completion (Actual): March 11, 2021

Study Registration Dates

• First Submitted: October 24, 2016
• First Submitted That Met QC Criteria: October 26, 2016
• First Posted (Estimated): October 28, 2016

Study Record Updates

• Last Update Posted (Actual): October 31, 2023
• Last Update Submitted That Met QC Criteria: January 12, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: Real World Evidence; Tenosynovial Giant Cell Tumors; Disease registry; Routine clinical practice
• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Neoplasms, Connective Tissue; Tendinopathy; Synovitis; Giant Cell Tumors; Giant Cell Tumor of Tendon Sheath
• Other Study ID Numbers: DS-ONC-01-15-EU

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: 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/
• IPD Sharing Time Frame: 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.
• IPD Sharing Access Criteria: 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.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR)