Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

NCT02948088 | Completed Results

• 1 other identifier: DS-ONC-01-15-EU
• Study Type: observational
• Target: 183
• Locations: 8 countries
• Sites: 15

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.

Conditions

Giant Cell Tumors

Keywords

Tenosynovial Giant Cell Tumors; Disease registry; Routine clinical practice; Real World Evidence

Outcome Measures

Primary Outcomes (13)

• 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 Outcomes (4)

• Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months

  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

  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

  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)

  At end of observation period (approximately 2 years post-baseline)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study enrolled patients with d-TGCT.

You may qualify if:

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

Sponsors & Collaborators

• Daiichi Sankyo: lead
• Optimapharm: collaborator

Study Sites (15)

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

University of California San Francisco

San Francisco, California, 94158, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Medizinische Universität Graz

Graz, 8036, Austria

Location

CHU Nantes

Nantes, 44093, France

Location

Westdeutsches Tumorzentrum

Essen, 45122, Germany

Location

Istituto Ortopedico Rizzoli

Bologna, 40136, Italy

Location

Istituto Nazionale Tumori-Foundazione IRCCS

Milan, 20133, Italy

Location

Leiden University Medical Center

Leiden, 2333, Netherlands

Location

Radboud universitair medisch centrum

Nijmegen, 6525 GA, Netherlands

Location

Hospital Sant Pau

Barcelona, 08041, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

The Royal Marsden Hospital

London, SW3 6JJ, United Kingdom

Location

Nuffield Orthopaedic Centre

Oxford, OX3 7LD, United Kingdom

Location

Related Publications (2)

• Palmerini E, Healey JH, Bernthal NM, Bauer S, Schreuder H, Leithner A, Martin-Broto J, Gouin F, Lopez-Bastida J, Gelderblom H, Staals EL, Mercier F, Laeis P, Ye X, van de Sande M. Tenosynovial Giant Cell Tumor Observational Platform Project (TOPP) Registry: A 2-Year Analysis of Patient-Reported Outcomes and Treatment Strategies. Oncologist. 2023 Jun 2;28(6):e425-e435. doi: 10.1093/oncolo/oyad011.

  PMID: 36869793 DERIVED

• 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.

  PMID: 33926503 DERIVED

MeSH Terms

Conditions

Giant Cell Tumors; Synovitis, Pigmented Villonodular

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Giant Cell Tumor of Tendon Sheath; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Limitations and Caveats

This was an observational study and the quality of data has not been verified 100%. Baseline data were checked during on-site visits for all patients enrolled in Europe. Due to the COVID-19 pandemic, the planned on-site monitoring visits in the US could not be performed. Instead, more frequent and detailed remote visits were performed for these sites. Follow-up data for all patients were regularly monitored remotely. A certain amount of incorrect/incomplete data has to be assumed for all sites.

Results Point of Contact

• Title: Contact for Clinical Trial Information
• Organization: Daiichi Sankyo

CTRinfo@dsi.com

908-992-6400

Study Officials

• Global Clinical Leader

  Daiichi Sankyo

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 24 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2016
• First Posted: October 28, 2016
• Study Start: November 18, 2016
• Primary Completion: March 11, 2021
• Study Completion: March 11, 2021
• Last Updated: October 31, 2023
• Results First Posted: October 31, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); FR (1); AU (1); NL (2); GB (2); ES (2); IT (2); DE (1)