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

NCT00459641 | Withdrawn Phase 2 DMC

• 1 other identifier: CS I-040302/01
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Bone Cysts

Keywords

Osteogenic Gel; Solitary Bone Cysts

Outcome Measures

Primary Outcomes (1)

• Radiographic images

  At 6 months and throughout the study

Secondary Outcomes (4)

• Blood parameters

  At 6 months and throughout the study

• Vital signs

  At 6 months and throughout the study

• Questionnaires of life questionnaire

  At 6 months and throughout the study

• Pharmacoeconomic evaluation

  At 6 months and throughout the study

Study Arms (2)

1

EXPERIMENTAL

I-040302 doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34

Drug: I-040302

2

ACTIVE COMPARATOR

Standard of care (bone marrow aspirate or steroids)

Other: Standard of Care

Interventions

I-040302 DRUG

doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34

1

Standard of Care OTHER

Bone marrow aspirate or steroids

Also known as: Bone marrow aspirate or steroids

2

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Kuros Biosurgery AG: lead

MeSH Terms

Conditions

Bone Cysts

Interventions

Standard of Care; Steroids

Condition Hierarchy (Ancestors)

Cysts; Neoplasms; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Virginia Jamieson, MD

  Kuros Biosurgery AG, Technoparkstrasse 1, CH-8005 Zurich

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2007
• First Posted: April 12, 2007
• Study Start: December 1, 2012
• Primary Completion: June 1, 2014
• Study Completion: July 1, 2019
• Last Updated: July 17, 2012

Record last verified: 2012-07