Clinical trial NCT05402865: A Real-world Study on Patients of Unresectable Giant Cell Tumor of Bone
Official title: A Real-world Study on Patients of Surgically Unsalvageable or Severe Post-surgery Morbidity Associated Giant Cell Tumor of Bone
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Giant Cell Tumor of Bone
Lead sponsor: Shanghai JMT-Bio Inc.
Sponsor class: INDUSTRY
First posted: 2022-06-02
Enrollment: 301
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Chinese

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Tumor response rate
timeFrame: 12 weeks (radiology), or through study completion, an average of 1 year (histopathology)
Secondary outcomes:
measure: Tumor response rate (Key secondary endpoint)
timeFrame: through study completion, an average of 1 year (radiology and histopathology)
measure: Proportion of patients that is surgically resectable
timeFrame: through study completion, an average of 1 year
measure: Median duration of tumor response (DOR)
timeFrame: through study completion, an average of 1 year
measure: Disease control rate (DCR)
timeFrame: through study completion, an average of 1 year
measure: Time to disease progression (TTP)
timeFrame: through study completion, an average of 1 year
measure: Types and proportion of key adverse reactions
timeFrame: through study completion, an average of 1 year
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Jishuitan Hospital
state: Beijing
city: Beijing
country: China
facility: Peking University Third Hospital
state: Beijing
city: Beijing
country: China
facility: West China Hospital of Sichuan University
state: Beijing
Study officials:
References: