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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02948088
Recruitment Status : Active, not recruiting
First Posted : October 28, 2016
Last Update Posted : January 18, 2020
Sponsor:
Collaborator:
SSS International Clinical Research GmbH
Information provided by (Responsible Party):
Daiichi Sankyo, Inc.

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.


Condition or disease
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 diffuse TGCT regularly.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 24 Months
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 Start Date : November 2016
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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)

  2. 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 (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)

  3. Percentage of participants with disease status reported as one of the categorical descriptions at 24 Months [ Time Frame: at 24 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 (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)


Secondary Outcome Measures :
  1. 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

  2. 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


  3. 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


  4. 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


  5. 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


  6. 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




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with diffuse TGCT
Criteria

Inclusion Criteria:

  • Written informed consent for participation in the study (ICF)
  • Patients with diffuse TGCT (diagnosed histologically) confirmed naïve or recurrent case

Exclusion Criteria:

  • None

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02948088


Locations
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United States, California
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
University of California San Francisco
San Francisco, California, United States, 94158
United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Austria
Medizinische Universität Graz
Graz, Austria, 8036
France
CHU Nantes
Nantes, France, 44093
Germany
Westdeutsches Tumorzentrum
Essen, Germany, 45122
Italy
Istituto Ortopedico Rizzoli
Bologna, Italy, 40136
Istituto Nazionale Tumori-Foundazione IRCCS
Milano, Italy, 20133
Netherlands
Leiden University Medical Center
Leiden, Netherlands, 2333
Radboud universitair medisch centrum
Nijmegen, Netherlands, 6525 GA
Spain
Hospital Sant Pau
Barcelona, Spain, 08041
Hospital Universitario Virgen del Rocio
Sevilla, Spain, 41013
United Kingdom
The Royal Marsden Hospital
London, United Kingdom, SW3 6JJ
Nuffield Orthopaedic Centre
Oxford, United Kingdom, OX3 7LD
Sponsors and Collaborators
Daiichi Sankyo, Inc.
SSS International Clinical Research GmbH
Investigators
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Study Director: Global Clinical Leader Daiichi Sankyo, Inc.
Additional Information:

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Responsible Party: Daiichi Sankyo, Inc.
ClinicalTrials.gov Identifier: NCT02948088    
Other Study ID Numbers: DS-ONC-01-15-EU
First Posted: October 28, 2016    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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/
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
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.
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.
URL: https://vivli.org/ourmember/daiichi-sankyo/
Keywords provided by Daiichi Sankyo, Inc.:
Tenosynovial Giant Cell Tumors
Disease registry
Routine clinical practice
Real World Evidence
Additional relevant MeSH terms:
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Giant Cell Tumors
Giant Cell Tumor of Tendon Sheath
Neoplasms
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Synovitis
Joint Diseases
Musculoskeletal Diseases
Tendinopathy
Muscular Diseases