Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts

Recurrent Disease Clinical Trial

— Deno-ABC  

Official title:

Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03415477
• Other study ID #: Denosumab-ABC
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: January 10, 2018
• Last updated: January 29, 2018
• Start date: January 1, 2014
• Est. completion date: December 31, 2017

Study information

• Verified date: January 2018
• Source: Peking University People's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: December 31, 2017
• Est. primary completion date: December 31, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Bone Cyst Aneurysmal
• Bone Cysts
• Cysts
• Pathological Fracture
• Recurrent Disease
• Refractory Tumor

Intervention

Drug:
• Denosumab (Xgeva)
Denosumab (Xgeva) 120 mg was given subcutaneously (abdomen wall or upper arms) every 4 weeks (Q4W), with a loading dose on both Day 8 and Day15 of the first cycle, perioperatively.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

References & Publications (4)

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):E654-60. doi: 10.1097/BRS.0000000000001350. — View 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. — View 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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Objective Response Rate (ORR)	the number of participats (complete response+ partial response according to RECIST 1.1)/ total participants number.	at least 1-year
Primary	local control rate	CT or MR proof that the tumor relapse in the primary site by at least two radiologist.	at least 1-year
Secondary	Number of participants with abnormal laboratory values and/or adverse events that are related to the use of Denosumab(Xgeva)	include 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 in follow-up every 3-month.	at least 1-year