Safety and Tolerability of I-040302 in Children and Young Adults With Solitary Bone Cysts - Full Text View

Sponsor:	Kuros Biosurgery AG
Information provided by (Responsible Party):	Kuros Biosurgery AG
ClinicalTrials.gov Identifier:	NCT00459641

Purpose

This is a randomised, controlled, open-label study of a single intralesional administration of I-040302 or a single bone marrow aspirate or a single steroid injection (methylprednisolone). Subjects will undergo screening with X-ray and magnetic resonance imaging (MRI) in the diagnosis of a solitary bone cyst.

Condition	Intervention	Phase
Bone Cysts	Drug: I-040302 Other: Standard of Care	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase 2 Randomised, Open-label, Multi-centre Ascending Dose Study of the Efficacy, Safety and Tolerability of I-040302 Versus Control Injection (Bone Marrow Aspirate or Steroids) in Children and Young Adults With Solitary Bone Cysts

Further study details as provided by Kuros Biosurgery AG:

Primary Outcome Measures:

Radiographic images [ Time Frame: At 6 months and throughout the study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Blood parameters [ Time Frame: At 6 months and throughout the study ] [ Designated as safety issue: Yes ]
Vital signs [ Time Frame: At 6 months and throughout the study ] [ Designated as safety issue: Yes ]
Questionnaires of life questionnaire [ Time Frame: At 6 months and throughout the study ] [ Designated as safety issue: Yes ]
Pharmacoeconomic evaluation [ Time Frame: At 6 months and throughout the study ] [ Designated as safety issue: No ]

Estimated Enrollment:	51
Study Start Date:	December 2012
Estimated Study Completion Date:	July 2019
Estimated Primary Completion Date:	June 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 1 I-040302 doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34	Drug: I-040302 doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34
Active Comparator: 2 Standard of care (bone marrow aspirate or steroids)	Other: Standard of Care Bone marrow aspirate or steroids Other Name: Bone marrow aspirate or steroids

Eligibility

Ages Eligible for Study:	6 Years to 16 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Subjects 6 - 16 years of age
Diagnostic evidence of unicameral bone cysts based on one or more of a combination of any of the following methods:
- Cyst fluid examination
- Plain radiographs
- MRI
Subjects with the following types of cysts:
- Intact cysts that are either growing or at risk of fracture, requiring surgical intervention
- Persistence of a cyst cavity following cyst fracture that has received conservative treatment/s to repair the fracture
- Persistence of a cyst cavity despite repeated interventions
Subjects with bone cyst volumes < 30 mL
Subjects must be appropriately communicative to verbalise pain.
Subjects must be able to understand and be willing to comply with the protocol procedures.
Subjects who have provided written informed consent to participate in the study
The legally authorised representative of the child must give written informed consent after the child has agreed to participate in the study.

Exclusion Criteria:

A history of/or presence of active cancer
Family history of retinoblastoma
Indication of aneurysmal bone cyst on MRI, cyst aspiration or histology
Possible presence of osteosarcoma or uncertain histology
Systemic or localised infection at time of surgery
Evidence of immune-suppression
Evidence of hypercalcemia
Cyst volume > 30 mL
Fracture present in the cortical bone surrounding the cyst
Evidence of rapid venous drainage from the cyst determined by rapid disappearance of X-ray contrast medium during the surgical process.
Suspected or known evidence of allergic reactions towards any of the components of I-040302
Known clinically significant organ or systemic diseases such that, in the opinion of the investigator, the significance of the disease will compromise the subject's participation in the study
Pregnant or lactating females
Participation in another clinical trial within 3 months prior to trial start

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00459641

Contacts

Contact: Virginia Jamieson	0041 44 200 5600 ext 14	virginia.jamieson@kuros.ch
Contact: Mitra Safari	0041 44 200 5600 ext 82	mitra.safari@kuros.ch

Locations

Italy
Clinica Ortopedica	Not yet recruiting
Bologna, Italy, 40136
Contact: Mario Mercuri, MD +39 51 6366222 mario.mercuri@ior.it
Principal Investigator: Mario Mercuri, MD
U.O. Ortopedia e Traumatologia	Not yet recruiting
Genova, Italy, 16147
Contact: Silvio Boero, MD +39 010 5636240 silvioboero@ospedale-gaslini.ge.it
Principal Investigator: Silvio Boero, MD
Switzerland
Service d'Orthopedie Pediatrique	Not yet recruiting
Geneve, Switzerland, 1211
Contact: Dimitri Ceroni, MD +41 223824786 Dimitri.Ceroni@hcuge.ch
Principal Investigator: Dimitri Ceroni, MD

Sponsors and Collaborators

Kuros Biosurgery AG

Investigators

Study Director:

Virginia Jamieson, MD

Kuros Biosurgery AG, Technoparkstrasse 1, CH-8005 Zurich

More Information

No publications provided

Responsible Party:	Kuros Biosurgery AG
ClinicalTrials.gov Identifier:	NCT00459641 History of Changes
Other Study ID Numbers:	CS I-040302/01
Study First Received:	April 11, 2007
Last Updated:	March 15, 2012
Health Authority:	Switzerland: Ethikkommission Switzerland: Swissmedic Italy: National Institute of Health Italy: The Italian Medicines Agency United Kingdom: Medicines and Healthcare Products Regulatory Agency United Kingdom: National Health Service

Keywords provided by Kuros Biosurgery AG:

Osteogenic Gel
Solitary Bone Cysts

Additional relevant MeSH terms:

Bone Cysts
Cysts
Neoplasms

Bone Diseases
Musculoskeletal Diseases
Pathological Conditions, Anatomical

ClinicalTrials.gov processed this record on May 24, 2012