Clinical Trial: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]
Official Title: A Disease Registry for Patients With Tenosynovial Giant Cell Tumors (TGCT), Also Known as Pigmented Villonodular Synovitis (PVNS) and Giant Cell Tumo
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, the burden of diffuse TGCT for patients (including pain, joint stiffness, swelling, reduced mobility and quality of life) or the economic impact of diffuse 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 Summary:
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) are planned to participate. The sites will be specialized sites that treat diffuse TGCT regularly.
Sponsor: Daiichi Sankyo Inc.
Current Primary Outcome:
- Percentage of participants with disease status reported as one of the categorical descriptions at Baseline [ Time Frame: at Baseline ]
Only patients with disease status reported as one of the five categorical descriptions below are included in this observation:
- fully active/able to carry on all pre-disease activities without restriction (0)
- restricted in physically strenuous activity, but ambulatory/able to carry out light or sedentary work (1)
- ambulatory (greater than [>] 50 percentage [%] of waking hours [h]), capable of all self care, but unable to carry out any work activities (2)
- capable of only limited self care, confined to bed/chair >50% of waking hours (3)
- completely disabled, cannot carry on any selfcare, totally confined to bed or chair (4)
- dead (5)
- Percentage of participants with disease status reported as one of the categorical descriptions at 12 Months [ Time Frame: at 12 Months ]
Only patients with disease status reported as one of the five categorical descriptions below are included in this observation:
- fully active/able to carry on all pre-disease activities without restriction (0)
- restricted in physically strenuous activity, but ambulatory/able to carry out light or sedentary work (1)
- ambulatory (greate
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Mean Number of Referrals or Specialist Visits to Assess the Health Resource Utilization in Patients with Diffuse TGCT through 24 Months [ Time Frame: at Baseline, 12 Months, 24 Months ]Categories: Baseline, 12 Months, 24 Months
- Mean Brief Pain Inventory (BPI) Average Interference Score From Baseline through 24 Months [ Time Frame: Baseline, 6, 12, 18 and 24 months ]
The BPI short form is a 5 minutes 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 change from baseline at each time point reflects the month X value minus the baseline value. The BPI average interference score ranges from 0 (pain does not interfere) to 10 (pain completely interferes).
Categories (scores on a scale): at Baseline, at Month 6, at Month 12, at Month 18 and at Month 24
- Mean Worst Stiffness Score from Baseline through 24 Months [ Time Frame: Baseline, 6, 12, 18 and 24 months ]
The Worst Stiffness numerical rating scale (NRS) is a one-item self-administered 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").
Categories (scores on a scale): at Baseline, at Month 6, at Month 12, at Month 18 and at Month 24
- Mean PROMIS Physical Function Score from Baseline through 24 Months [ Time Frame: Baseline, 6, 12, 18 and 24 months ]
PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures, developed by the US Department of Health and Human Services, that evaluates and monitors physical, mental, and social health. Physical function items relevant to the assessment of lower and upper limb function are to be selected from the PROMIS physical function item bank. Items assessing lower limb function will be administered to subjects with lower extremity tumors, and items assessing upper limb function will be administered to subjects with upper extremity tumors. The results from both sets of items will be combined and analyzed together.
Categories (scores on a scale): at Baseline, at Month 6, at Month 12, at Month 18 and at Month 24
- Mean Scores on the EuroQol Questionnaire (EQ) of 5 Dimensions (5D) on a Standardized 5-level (5L) Descriptive Health Status Scale (EQ-5D-5L), from Baseline through 24 Months [ Time Frame: Baseline, 6, 12, 18 and 24 months ]
The EQ-5D questionnaire is made up for two components; health state description and evaluation.
The EQ-5D-5L is the health state description for measuring health status. The descriptive system comprises the 5 dimensions of mobility, self care, usual activities, pain/discomfort, and anxiety/depression. The participants self-rate each dimension on a 5-point, categorical scale: no problems, slight problems, moderate problems, severe problems, and extreme problems.
Categories (scores on a scale): at Baseline, at Month 6, at Month 12, at Month 18 and at Month 24
- Mean Scores on the EQ-5D Visual Analogue Scale (EQ VAS) Evaluation, from Baseline through 24 Months [ Time Frame: Baseline, 6, 12, 18 and 24 months ]
The EQ-5D questionnaire is made up for two components; health state description and evaluation.
The EQ VAS is the evaluation that records the respondent's self-rated health on a 20 cm vertical, VAS with ends labeled "the best health you can imagine" and "the worst health you can imagine".
Categories (scores on a scale): at Baseline, at Month 6, at Month 12, at Month 18 and at Month 24
Original Secondary Outcome: Same as current
Information By: Daiichi Sankyo Inc.
Dates:
Date Received: October 24, 2016
Date Started: November 2016
Date Completion: January 2021
Last Updated: May 4, 2017
Last Verified: May 2017
- Mean Number of Referrals or Specialist Visits to Assess the Health Resource Utilization in Patients with Diffuse TGCT through 24 Months [ Time Frame: at Baseline, 12 Months, 24 Months ]
