Clinical trial NCT03415477: Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts
Official title: Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Bone Cyst Aneurysmal
Pathological Fracture
Recurrent Disease
Refractory Tumor
Keywords: aneurysmal bone cyst, denosumab, surgical treatment, complication
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2018-01-30
Enrollment: 6
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Patients with recurrent and/or refractory aneurysmal bone cysts treated with denosumab perioperatively.
Primary purpose: TREATMENT

Brief summary: The efficacy of traditional therapeutic approaches for aneurysmal bone cysts (ABC), such as surgery, embolization, sclerotherapy and radiotherapy, are often compromised for lesions in axial skeletons and adolescents complicated with pathological fracture. Therefore, denosumab, a new drug that has been successfully used in giant cell tumor of bone but has seldom used in ABC, was used to treat ABC in this trial.

Detailed description: Patients with primary ABC treated with perioperative denosumab in the Musculoskeletal Tumor Center of Peking University People's Hospital between January 2014 and December 2016 will be reported in this trial.Approved by the Ethics Committee of Peking University People's Hospital and with the full informed consent from the patients or their families, all patients received surgery and treatment with denosumab perioperatively. Denosumab 120 mg was given subcutaneously (abdomen wall or upper arms) every 4 weeks (Q4W), with a loading dose on both Day 8 and Day 15 of the first cycle. Meanwhile, all patients were suggested to take 800mg oral calcium supplement every day and avoid any dental or oral surgery (tooth extraction, tooth filling, etc.). Patients were followed once a month since the date of surgery. On follow-up visits, patient-reported adverse reactions, X-ray image of jawbone, serum calcium, alkaline phosphatase, renal and hepatic function, hemoglobin, white blood cell, and platelet count were collected, and histological examinations, radiological examinations (such as X-ray, computed tomography (CT), magnetic resonance image (MRI) and positron emission tomography-computed tomography (PET-CT) and postoperative functional status evaluations by MSTS（Musculoskeletal tumor society） score were conducted.

Eligibility criteria: Inclusion Criteria:

1. pathologically diagnosed with primary aneurysmal bone cyst
2. lesions in axial skeletons (i.e. spine, pelvis) which are difficult to reach a satisfactory surgical margin, and surgery can lead to very high incidence of complications.
3. pathological fracture
4. recurrence cases

Exclusion Criteria:

1. metastases or multi-focal lesions
2. malignancy

Primary outcomes:
measure: Objective Response Rate (ORR)
timeFrame: at least 1-year
measure: local control rate
timeFrame: at least 1-year
Secondary outcomes:
measure: Number of participants with abnormal laboratory values and/or adverse events that are related to the use of Denosumab（Xgeva）
timeFrame: at least 1-year
Other outcomes:
Locations:
Study officials:
References:
citation: Ghermandi R, Terzi S, Gasbarrini A, Boriani S. Denosumab: non-surgical treatment option for selective arterial embolization resistant aneurysmal bone cyst of the spine and sacrum. Case report. Eur Rev Med Pharmacol Sci. 2016 Sep;20(17):3692-5.
citation: Dubory A, Missenard G, Domont J, Court C. Interest of Denosumab for the Treatment of Giant-cells Tumors and Aneurysmal Bone Cysts of the Spine. About Nine Cases. Spine (Phila Pa 1976). 2016 Jun;41(11):E654-E660. doi: 10.1097/BRS.0000000000001350.
citation: Skubitz KM, Peltola JC, Santos ER, Cheng EY. Response of Aneurysmal Bone Cyst to Denosumab. Spine (Phila Pa 1976). 2015 Nov;40(22):E1201-4. doi: 10.1097/BRS.0000000000001027.
citation: Pauli C, Fuchs B, Pfirrmann C, Bridge JA, Hofer S, Bode B. Response of an aggressive periosteal aneurysmal bone cyst (ABC) of the radius to denosumab therapy. World J Surg Oncol. 2014 Jan 20;12:17. doi: 10.1186/1477-7819-12-17.