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

This study has been withdrawn prior to enrollment.
(Stopped before any site initiation or any recruitment due to change in overall product development plan)
Sponsor:
Information provided by (Responsible Party):
Kuros Biosurgery AG
ClinicalTrials.gov Identifier:
NCT00459641
First received: April 11, 2007
Last updated: July 16, 2012
Last verified: July 2012
  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

Resource links provided by NLM:


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 ]

Enrollment: 0
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:

  1. Subjects 6 - 16 years of age
  2. 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
  3. 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
  4. Subjects with bone cyst volumes < 30 mL
  5. Subjects must be appropriately communicative to verbalise pain.
  6. Subjects must be able to understand and be willing to comply with the protocol procedures.
  7. Subjects who have provided written informed consent to participate in the study
  8. The legally authorised representative of the child must give written informed consent after the child has agreed to participate in the study.

Exclusion Criteria:

  1. A history of/or presence of active cancer
  2. Family history of retinoblastoma
  3. Indication of aneurysmal bone cyst on MRI, cyst aspiration or histology
  4. Possible presence of osteosarcoma or uncertain histology
  5. Systemic or localised infection at time of surgery
  6. Evidence of immune-suppression
  7. Evidence of hypercalcemia
  8. Cyst volume > 30 mL
  9. Fracture present in the cortical bone surrounding the cyst
  10. Evidence of rapid venous drainage from the cyst determined by rapid disappearance of X-ray contrast medium during the surgical process.
  11. Suspected or known evidence of allergic reactions towards any of the components of I-040302
  12. 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
  13. Pregnant or lactating females
  14. Participation in another clinical trial within 3 months prior to trial start
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

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: July 16, 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:
Cysts
Bone Cysts
Neoplasms
Pathological Conditions, Anatomical
Bone Diseases
Musculoskeletal Diseases

ClinicalTrials.gov processed this record on October 19, 2014