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

Completed

• Conditions: Giant Cell Tumor of Bone
• Interventions: Drug: Denosumab

• Registration Number: NCT05402865
• Lead Sponsor: Shanghai JMT-Bio Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2021-09
• Study Start: 2021-11-10
• Primary Completion: 2021-12-01
• Study Completion: 2022-02-18
• Study First Submitted: 2022-05-25
• Study First Submitted QC: 2022-05-30
• Last Update Submitted: 2022-05-30
• Study First Posted: 2022-06-02
• Last Update Posted: 2022-06-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 301

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Denosumab group	Denosumab	patients who had received denosumab

Primary Outcome Measures

Name	Time	Method
Tumor response rate	12 weeks (radiology), or through study completion, an average of 1 year (histopathology)	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

Secondary Outcome Measures

Name	Time	Method
Tumor response rate (Key secondary endpoint)	through study completion, an average of 1 year (radiology and histopathology)	Radiographic tumor response (CR/PR evaluated by ICDs or EORTC criteria), or at least 90% reduction of osteoclast like giant cells compared with baseline
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

Trial Locations

Locations (3)

Peking University Third Hospital; 🇨🇳 Beijing, Beijing, China

Beijing Jishuitan Hospital; 🇨🇳 Beijing, Beijing, China

West China Hospital of Sichuan University; 🇨🇳 Beijing, Beijing, China