Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts

NCT03415477 | Completed Phase 1 FDA Drug

• 1 other identifier: Denosumab-ABC
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Bone Cyst Aneurysmal; Pathological Fracture; Recurrent Disease; Refractory Tumor

Keywords

aneurysmal bone cyst, denosumab, surgical treatment, complication

Outcome Measures

Primary Outcomes (2)

• Objective Response Rate (ORR)

  the number of participats (complete response+ partial response according to RECIST 1.1)/ total participants number.

  at least 1-year

• 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 Outcomes (1)

• Number of participants with abnormal laboratory values and/or adverse events that are related to the use of Denosumab（Xgeva）

  at least 1-year

Study Arms (1)

denosumab (Xgeva) treatment

EXPERIMENTAL

Patients with aneurismal bone cysts received perioperative denosumab(Xgeva).

Drug: Denosumab (Xgeva)

Interventions

Denosumab (Xgeva) DRUG

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.

Also known as: surgical treatment

denosumab (Xgeva) treatment

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• pathologically diagnosed with primary aneurysmal bone cyst
• 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.
• pathological fracture
• recurrence cases

You may not qualify if:

• metastases or multi-focal lesions
• malignancy

Sponsors & Collaborators

• Peking University People's Hospital: lead

Related Publications (4)

• 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.

  PMID: 27649673 BACKGROUND

• 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.

  PMID: 27244112 BACKGROUND

• 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.

  PMID: 26730528 BACKGROUND

• 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.

  PMID: 24438319 BACKGROUND

MeSH Terms

Conditions

Bone Cysts, Aneurysmal; Fractures, Spontaneous; Recurrence; Neoplasms

Interventions

Denosumab; Surgical Procedures, Operative

Condition Hierarchy (Ancestors)

Bone Cysts; Cysts; Bone Diseases; Musculoskeletal Diseases; Fractures, Bone; Wounds and Injuries; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Head of Muscoloskeletal Tumor Center, Principal Investigator, Clinical Professor

Model Details: Patients with recurrent and/or refractory aneurysmal bone cysts treated with denosumab perioperatively.

Study Record Dates

• First Submitted: January 10, 2018
• First Posted: January 30, 2018
• Study Start: January 1, 2014
• Primary Completion: December 31, 2016
• Study Completion: December 31, 2017
• Last Updated: January 30, 2018

Record last verified: 2018-01

Data Sharing

• IPD Sharing: Will not share