Bone Cyst AneurysmalPhase 1/2Deno-ABCNCT03415477
Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts
A Phase 1/2 study testing Denosumab (Xgeva) in people with Bone Cyst Aneurysmal and Pathological Fracture, run by Peking University People's Hospital.
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. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Bone Cyst Aneurysmal, Pathological Fracture, Recurrent Disease
- What is being tested?
- Denosumab (Xgeva)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2014to 31 Dec 2017
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- metastases or multi-focal lesions
- malignancy
About this study
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.
Read the 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.
- Being tested
- Denosumab (Xgeva)
- Keywords
- aneurysmal bone cyst, denosumab, surgical treatment, complication
- Medical subject headings
- Bone Cysts, Aneurysmal, Fractures, Spontaneous, Recurrence, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Denosumab (Xgeva)Drug
Also called surgical treatment
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.
| Study group | Role | What they receive |
|---|---|---|
| denosumab (Xgeva) treatment | Experimental | Patients with aneurismal bone cysts received perioperative denosumab(Xgeva). Receives: Drug: Denosumab (Xgeva) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
the number of participats (complete response+ partial response according to RECIST 1.1)/ total participants number.
Measured: at least 1-year
local control rate
CT or MR proof that the tumor relapse in the primary site by at least two radiologist.
Measured: 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）
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.
Measured: at least 1-year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2018
- Study start1 Jan 2014
- Primary completion31 Dec 2016
- Study completion31 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Bone Cyst AneurysmalStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients with recurrent and/or refractory aneurysmal bone cysts treated with denosumab perioperatively.
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, GUO WEI, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03415477
- Sponsor's protocol ID
- Denosumab-ABC
- Acronym
- Deno-ABC
- Status last verified
- Jan 2018
- First submitted
- 10 Jan 2018
- Last updated
- 30 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 27649673Dubory 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.
PubMed 27244112Skubitz 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.
PubMed 26730528Pauli 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.
PubMed 24438319
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Denosumab: A Potential Treatment Option for Aneurysmal Bone Cysts
- ClinicalTrials.gov ID
- NCT03415477
- Acronym / sponsor ID
- Deno-ABC / Denosumab-ABC
- Keywords
- aneurysmal bone cyst, denosumab, surgical treatment, complication
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2014 (actual)
- Primary completion
- 31 Dec 2016 (actual)
- Study completion
- 31 Dec 2017 (actual)
- First posted
- 30 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients with recurrent and/or refractory aneurysmal bone cysts treated with denosumab perioperatively.
| Arm | Type | Receives |
|---|---|---|
| denosumab (Xgeva) treatment | Experimental | Drug: Denosumab (Xgeva)Patients with aneurismal bone cysts received perioperative denosumab(Xgeva). |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- metastases or multi-focal lesions
- malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Denosumab (Xgeva)Also: surgical treatmentDenosumab (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. | Drug | denosumab (Xgeva) treatment |
Primary outcome
Objective Response Rate (ORR)
Time frame: at least 1-year
the number of participats (complete response+ partial response according to RECIST 1.1)/ total participants number.
local control rate
Time frame: at least 1-year
CT or MR proof that the tumor relapse in the primary site by at least two radiologist.
1 secondary outcome
Number of participants with abnormal laboratory values and/or adverse events that are related to the use of Denosumab（Xgeva）
Time frame: at least 1-year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2018.
- Background: PubMed 27649673Ghermandi 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.
- Background: PubMed 27244112Dubory 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.
- Background: PubMed 26730528Skubitz 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.
- Background: PubMed 24438319Pauli 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.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Denosumab (Xgeva) in people with Bone Cyst Aneurysmal and Pathological Fracture, run by Peking University People's Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: at least 1-year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03415477, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2018.