Clinical trial NCT02948088: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Giant Cell Tumors
Keywords: Tenosynovial Giant Cell Tumors
Disease registry
Routine clinical practice
Real World Evidence
Lead sponsor: Daiichi Sankyo
Sponsor class: INDUSTRY
Collaborators: name: Optimapharm
First posted: 2016-10-28
Enrollment: 183
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study enrolled patients with d-TGCT.

Brief 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT)
timeFrame: at Baseline
measure: Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT)
timeFrame: at Baseline (prior to any d-TGCT therapy)
measure: Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT)
timeFrame: at Baseline
measure: Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT)
timeFrame: at Baseline
measure: Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT)
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Mean Worst Stiffness Score From Baseline Through 24 Months
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Mean PROMIS Physical Function Score From Baseline Through 24 Months
timeFrame: Baseline up to end of observation period (approximately 2 years)
Secondary outcomes:
measure: Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months
timeFrame: Baseline up to end of observation period (approximately 2 years)
measure: Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months
timeFrame: 13-24 months before baseline up to end of observation period (approximately 2 years)
measure: Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months
timeFrame: 13-24 months before baseline up to end of observation period (approximately 2 years)
measure: Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT)
timeFrame: At end of observation period (approximately 2 years post-baseline)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: David Geffen School of Medicine at UCLA
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Houston
country: United States
facility: University of Texas MD Anderson Cancer Center
state: Texas
city: Graz
country: Austria
facility: Medizinische Universität Graz
city: Nantes
country: France
facility: CHU Nantes
city: Essen
country: Germany
facility: Westdeutsches Tumorzentrum
city: Bologna
country: Italy
facility: Istituto Ortopedico Rizzoli
city: Milano
country: Italy
facility: Istituto Nazionale Tumori-Foundazione IRCCS
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
city: Nijmegen
country: Netherlands
facility: Radboud universitair medisch centrum
city: Barcelona
country: Spain
facility: Hospital Sant Pau
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: London
country: United Kingdom
facility: The Royal Marsden Hospital
city: Oxford
country: United Kingdom
facility: Nuffield Orthopaedic Centre
Study officials:
affiliation: Daiichi Sankyo
name: Global Clinical Leader
role: STUDY_DIRECTOR
References:
citation: 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.