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Clinical Trials/NCT05419960
NCT05419960
Recruiting
N/A

Audio-vestibular Evaluation of Children and Young Adults With Osteogenesis Imperfecta

Assistance Publique - Hôpitaux de Paris1 site in 1 country44 target enrollmentDecember 22, 2022

Overview

Phase
N/A
Intervention
Not specified
Conditions
Osteogenesis Imperfecta
Sponsor
Assistance Publique - Hôpitaux de Paris
Enrollment
44
Locations
1
Primary Endpoint
Ocular Vestibular Evoked Myogenic Potential (oVEMP)
Status
Recruiting
Last Updated
7 months ago

Overview

Brief Summary

The aim is to determine whether vestibular deficits are present in OI, then to establish whether a correlation exists between genetic type, severity of OI and audiovestibular phenotype. OI patients aged 12 to 20 years will undergo an audiometric, immittance, and vestibular assessment. When hearing loss is conductive or mixed or in cases where vestibular deficits are identified, a CT scan without injection will be performed. In case of sensorineural hearing loss or abnormal CT results, an MRI will be performed.

Detailed Description

Osteogenesis Imperfecta (OI), or Lobstein's disease, is a form of congenital osteoporosis, with a prevalence between 1/10,000 and 1/20,000 in France. As of 1979, four phenotypes have been described according to severity: moderate (type I), lethal (type II), severe (type III), and moderate-to-severe (type IV). OI exhibits phenotypic and genotypic variability, however, to date no correlations have been established between specific mutations and clinical presentation. There is a well-established association between OI and hearing loss, however, the reported prevalence of hearing loss varies between 2% to 94.1%. In patients with OI, Computed Tomography (CT) has revealed bone demineralization that progresses with age. The extent of the hypodense areas on the CT corresponds to the type of hearing loss: conductive hearing loss is associated with lesions of the fissula ante fenestram and round and oval windows while mixed hearing loss is associated with additional retrofenestral lesions. Severity of hearing loss is positively correlated with OI-related bone damage in the petrous bone. Magnetic Resonance Imaging (MRI) has also revealed in the pericochlear lesions with soft tissue hypersignal and enhancement on contrast medium injection in the otic capsule. Bone demineralization has also been linked to vestibular deficits, and some studies have reported correlations between Osteogenesis Imperfecta and vestibular deficits in adult patients, however, this relationship is less clear. The aim of the study is to determine whether vestibular deficits are also present in OI. Furthermore, the study will aim to establish whether a correlation exists between genetic type, severity of OI and audiovestibular phenotype. OI patients aged 12 to 20 years will be recruited and an audiometric, immittance, and vestibular assessment (videonystagmography, video Head Impulse Test (vHIT), vestibular evoked muscular potentials (cVEMP)) will be performed during their annual visit to the Centre de Référence des Maladies Rares des maladies osseuses constitutionnelles (henceforth CRMR OI) of Hôpital Necker-Enfants malades, Paris, France. When hearing loss is conductive, or mixed or in cases where vestibular deficits are identified, a CT scan without injection will be performed for the care. In case of sensorineural hearing loss, or abnormal CT results, an MRI will be proposed for the care. The investigation team will try to establish if there is a significant association between OI and vestibular deficit, and if so, whether the degree of vestibular impairment is correlated to radiological findings with respect to bone abnormalities, as well as the type and severity of the deafness.

Registry
clinicaltrials.gov
Start Date
December 22, 2022
End Date
December 22, 2027
Last Updated
7 months ago
Study Type
Observational
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Patients between the ages of 12-20 years at the time of inclusion
  • Diagnosis of Osteogenesis Imperfecta of any type
  • Currently followed by a physician at the CRMR OI
  • Information and non-opposition of major patients, holders of parental authority and minor patients to participate in the study

Exclusion Criteria

  • Patients with hearing loss of alternate origin e.g. Cochlear nerve deficiency, atresia, etc.
  • Neurological or developmental deficits limiting participation
  • Cervico-occipital instability e.g. Chiari's malformation
  • Limitations in mobility of the spine e.g. scoliosis, spinal fractural fusion
  • Ophthalmologic pathologies e.g. strabism or severe refraction disorder
  • Patients under AME (State Medical Aid)
  • Protected adult patients, adults unable to express their consent, pregnant or breastfeeding women

Outcomes

Primary Outcomes

Ocular Vestibular Evoked Myogenic Potential (oVEMP)

Time Frame: 24 months

Utricular Function Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta. The oVEMP assesses utricular function and reflects the function of the vestibular nuclei and the crossed vestibulo-ocular reflex (VOR) pathways, mostly contained in the medial longitudinal fasciculus (MLF). Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal.

Cervical Vestibular Evoked Myogenic Potential (cVEMP)

Time Frame: 24 months

Saccular Function Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta. The cVEMP assesses the saccular function via the saccular cervical reflex (sternocleidomastoid muscle's activation secondary to saccular auditory stimulation). Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal. The otolith organs are comprised of the vestibular saccule and utricule. The cVEMP assesses the saccular function via the saccular cervical reflex (sternocleidomastoid muscle's activation secondary to saccular auditory stimulation). Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal. Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta.

Central Vestibular Function

Time Frame: 24 months

Is evaluated by videonystagmography (VNG). The purpose of this test battery is to separate the vestibular disorders from disorders of the central neural system. Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal. Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta.

Semi-circular canal function

Time Frame: 24 months

Video Head Impulse Test (vHIT). The vHIT assesses the vestibular ocular reflex linked to the semi-circular canal function when stimulated at a high frequency. Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal. Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta.

Subjective Visual Vertical (SVV)

Time Frame: 24 months

Utricular Function Research of eventual vestibular disorder to determine if vestibular disorders can be linked to Osteogenesis Imperfecta. The patient is asked to orient a line on the vertical axis. An angle of 3 degrees from the vertical indicates an otolithic utricular disorder. Clinical norms will be applied to determine whether the exam is either within normal limits or abnormal.

Secondary Outcomes

  • Immittance testing(24 months)
  • Pure-tone audiometry(24 months)
  • Speech Audiometry(24 months)
  • Petrous bone Computed Tomography (CT)(24 months)
  • Severity of OI(24 months)
  • Magnetic Resonance Imaging (MRI)(24 months)

Study Sites (1)

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