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NCT02948088: TOP

Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project

Completed Results posted Last updated 31 October 2023
What this trial tests

trial in Giant Cell Tumors in 183 participants. Completed in 11 March 2021.

Timeline
18 November 2016
Primary endpoint
11 March 2021
11 March 2021

Quick facts

Lead sponsorDaiichi Sankyo
StatusCompleted
Study typeOBSERVATIONAL
Enrollment183
Start date18 November 2016
Primary completion11 March 2021
Estimated completion11 March 2021
Sites15 locations across France, Italy, Netherlands, Austria, United Kingdom, Germany, United States, Spain

Conditions studied

Sponsor

Daiichi Sankyo — full company profile →

Who can join

18 and older, any sex, with Giant Cell Tumors. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

The management plan received among the patients was collected from information routinely recorded in the patient files / medical records.

Any prior or concomitant therapies for managing TGCT-related symptoms
GroupValue95% CI
Baseline94
Within 1st YearNA
Within 2nd YearNA
Any changes to concomitant therapies for managing TGCT-related symptoms since the last visit
GroupValue95% CI
BaselineNA
Within 1st Year43
Within 2nd Year19
Any changes to TGCT treatment (including also planned treatments) since the last visit
GroupValue95% CI
BaselineNA
Within 1st Year74
Within 2nd Year61
Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

The current TGCT treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.

Wait and see
GroupValue95% CI
Baseline79
Within 1st Year77
Within 2nd Year105
Currently being treated or planned to be treated
GroupValue95% CI
Baseline97
Within 1st Year85
Within 2nd Year65
Unknown/Missing
GroupValue95% CI
Baseline0
Within 1st Year11
Within 2nd Year3
Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

Type of treatment plan received among the patients was collected from information routinely recorded in the patient files / medical records.

Radiotherapy
GroupValue95% CI
Baseline5
Within 1st Year1
Within 2nd Year0
90Yttrium therapy
GroupValue95% CI
Baseline1
Within 1st Year1
Within 2nd Year0
Systemic therapy
GroupValue95% CI
Baseline48
Within 1st Year16
Within 2nd Year8
Surgery
GroupValue95% CI
Baseline42
Within 1st Year40
Within 2nd Year25
Future surgery required
GroupValue95% CI
Baseline5
Within 1st Year2
Within 2nd Year0
No current or planned treatment
GroupValue95% CI
Baseline79
Within 1st Year77
Within 2nd Year105
Unchanged therapy
GroupValue95% CI
Baseline0
Within 1st Year36
Within 2nd Year35
Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

The status of treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.

Planned
GroupValue95% CI
Baseline50
Within 1st Year9
Within 2nd Year9
Current
GroupValue95% CI
Baseline44
Within 1st Year50
Within 2nd Year23
Missing
GroupValue95% CI
Baseline5
Within 1st Year2
Within 2nd Year0
Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

Type of current TGCT treatment among the patients was collected from information routinely recorded in the patient files / medical records.

90Yttrium therapy
GroupValue95% CI
Baseline0
Within 1st Year1
Within 2nd Year0
Systemic therapy
GroupValue95% CI
Baseline37
Within 1st Year12
Within 2nd Year8
Surgery
GroupValue95% CI
Baseline7
Within 1st Year37
Within 2nd Year17
Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT) Primary · at Baseline

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).

Moderate diffuse
GroupValue95% CI
Tenosynovial Giant Cell Tumors66
Severe diffuse
GroupValue95% CI
Tenosynovial Giant Cell Tumors90
Not assessable
GroupValue95% CI
Tenosynovial Giant Cell Tumors20
Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT) Primary · at Baseline (prior to any d-TGCT therapy)

Complications due to surgery were collected from information routinely recorded in the patient files / medical records.

GroupValue95% CI
Tenosynovial Giant Cell Tumors9
Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) Primary · at Baseline

Tumor recurrence was collected from information routinely recorded in the patient files / medical records.

1 recurrence
GroupValue95% CI
Participants With Tumor Recurrence40
2 recurrences
GroupValue95% CI
Participants With Tumor Recurrence18
3 recurrences and more
GroupValue95% CI
Participants With Tumor Recurrence19
Missing
GroupValue95% CI
Participants With Tumor Recurrence1
Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT) Primary · at Baseline

Time since most recent tumor recurrence was collected from information routinely recorded in the patient files / medical records.

GroupValue95% CI
Tenosynovial Giant Cell Tumors12.550.03 – 137.49
Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT) Primary · Baseline up to end of observation period (approximately 2 years)

Time from baseline to first tumor recurrence was collected from information routinely recorded in the patient files / medical records.

GroupValue95% CI
Within 1st Year8.42 – 15
Within 2nd Year19.012 – 24
Tenosynovial Giant Cell Tumors14.82 – 24
Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months Primary · Baseline up to end of observation period (approximately 2 years)

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.

Brief Pain Inventory Pain Severity
GroupValue95% CI
Baseline3.35± 2.36
6 Months3.12± 2.26
12 Months3.15± 2.22
18 Months3.05± 2.30
24 Months2.60± 2.19
Brief Pain Inventory Pain Interference
GroupValue95% CI
Baseline2.97± 2.65
6 Months2.94± 2.56
12 Months2.80± 2.44
18 Months2.80± 2.47
24 Months2.53± 2.57
Mean Worst Stiffness Score From Baseline Through 24 Months Primary · Baseline up to end of observation period (approximately 2 years)

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.

GroupValue95% CI
Baseline4.4± 2.8
6 Months4.2± 2.6
12 Months4.2± 2.8
18 Months4.2± 2.7
24 Months3.9± 2.7

Sponsor's own description

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.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Pexidartinib: First Approval.
    Lamb YN. · · 2019 · cited 155× · PMID 31602563 · DOI 10.1007/s40265-019-01210-0
  2. The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study.
    Bernthal NM, Spierenburg G, Healey JH, Palmerini E, et al · · 2021 · cited 44× · PMID 33926503 · DOI 10.1186/s13023-021-01820-6
  3. A prospective real-world study of the diffuse-type tenosynovial giant cell tumor patient journey: A 2-year observational analysis.
    Bernthal NM, Healey JH, Palmerini E, Bauer S, et al · · 2022 · cited 9× · PMID 36006054 · DOI 10.1002/jso.27067
  4. Tenosynovial Giant Cell Tumor Observational Platform Project (TOPP) Registry: A 2-Year Analysis of Patient-Reported Outcomes and Treatment Strategies.
    Palmerini E, Healey JH, Bernthal NM, Bauer S, et al · · 2023 · cited 8× · PMID 36869793 · DOI 10.1093/oncolo/oyad011
  5. Case Report: Giant Cell Tumor of Tendon Sheath After Breast Augmentation.
    Zhang Y, Fan Y, Zhang H, Bu H, et al · · 2022 · PMID 35814458 · DOI 10.3389/fonc.2022.878635

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