A Real-world Study on Patients of Unresectable Giant Cell Tumor of Bone

A Real-world Study on Patients of Surgically Unsalvageable or Severe Post-surgery Morbidity Associated Giant Cell Tumor of Bone

This is a retrospective observational real-world study, which evaluates the efficacy and safety of denosumab and non-denosumab therapies in the treatment of Chinese populations of surgically unsalvageable or severe post-surgery morbidity associated giant cell tumor of bone (GCTB), collectively referred to as unresectable GCTB, during 2013-2021 in three medical centers, serving as the external control for a single arm phase Ib/II trial on JMT103 treatment of GCTB. 301 patients were enrolled and divided into 2 groups according to their actual previous exposures. Group 1 (n=135) was denosumab group. Group 2 (n=166) included two types of exposures other than denosumab: other anti-GCTB drug therapies, or no therapy on GCTB patients. The dosage, route, frequency and other administration methods was collected according to the actual previous treatment records. The primary outcome measure was the tumor response rate [radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria) within 12 weeks, or at least 90% reduction of osteoclast like giant cells compared with baseline]. The key secondary endpoint was the tumor response rate [radiographic tumor response (CR/PR evaluated by ICDS or EORTC criteria), or at least 90% reduction of osteoclast like giant cells compared with baseline]. Other secondary Outcome Measures include: proportion of patients whose tumors was surgically resectable; median duration of tumor response (DOR), disease control rate (DCR), and time to disease progression (TTP); and types and proportion of key adverse reactions.

Study Overview

• Status: Completed
• Conditions: Giant Cell Tumor of Bone
• Intervention / Treatment: Drug: Denosumab

• Study Type: Observational
• Enrollment (Actual): 301

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100191
      • Peking University Third Hospital
    • Beijing, Beijing, China, 100035
      • BeiJing JiShuiTan Hospital
    • Beijing, Beijing, China, 610044
      • West China Hospital of Sichuan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Chinese

Description

Inclusion Criteria:

• 1. Both genders, aged 18 years or above;
• 2. Patients pathologically diagnosed of giant cell tumor of bone (GCTB), and are surgically unsalvageable, or salvageable but surgery was associated with severe morbidity based on physician's judgement;
• 3. For patients received denosumab or other anti-GCTB medications, one of the two is required: imaging data (including X-ray, CT, MRI or PET-CT) prior to and at least once followed to administration, or pathological data on surgically resected GCTB; for patients with no medication, imaging data or pathological data on surgically resected GCTB is required.

Exclusion Criteria:

• 1. No access to definite treatment information;
• 2. Bone metabolic diseases diagnosed before treatment: hypo-/ hyperparathyroidism, hypo-/hyperthyroidism, hypopituitarism, hyperprolactinemia, Cushing syndrome, acromegaly, Paget disease, etc;
• 3. Complication of malignant tumor receiving anti-tumor therapy.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Denosumab group; patients who had received denosumab	Drug: Denosumab; Denosumab, subcutaneous injection, specific usage to see the medical records. Non-denosumab, specific usage to see the medical records.; Other Names: Non-denosumab
Non-denosumab group; patients who had received anti-GCTB drug therapy other than denosumab, or who did not receive any anti-GCTB medication

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tumor response rate	Radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria) within 12 weeks, or at least 90% reduction of osteoclast like giant cells compared with baseline	12 weeks (radiology), or through study completion, an average of 1 year (histopathology)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tumor response rate (Key secondary endpoint)	Radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria), or at least 90% reduction of osteoclast like giant cells compared with baseline	through study completion, an average of 1 year (radiology and histopathology)
Proportion of patients that is surgically resectable		through study completion, an average of 1 year
Median duration of tumor response (DOR)		through study completion, an average of 1 year
Disease control rate (DCR)		through study completion, an average of 1 year
Time to disease progression (TTP)		through study completion, an average of 1 year
Types and proportion of key adverse reactions		through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Shanghai JMT-Bio Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 10, 2021
• Primary Completion (ACTUAL): December 1, 2021
• Study Completion (ACTUAL): February 18, 2022

Study Registration Dates

• First Submitted: May 25, 2022
• First Submitted That Met QC Criteria: May 30, 2022
• First Posted (ACTUAL): June 2, 2022

Study Record Updates

• Last Update Posted (ACTUAL): June 2, 2022
• Last Update Submitted That Met QC Criteria: May 30, 2022
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Musculoskeletal Diseases; Bone Diseases; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Bone Neoplasms; Giant Cell Tumors; Giant Cell Tumor of Bone; Physiological Effects of Drugs; Bone Density Conservation Agents; Denosumab
• Other Study ID Numbers: JMT103CN03-1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No