Trial Outcomes & Findings for Audiovestibular Function in Infratentorial Superficial Siderosis (NCT NCT04200664)

NCT ID: NCT04200664

Last Updated: 2025-06-10

Results Overview

Clinical audiological (hearing) tests were performed to assess hearing function. The hearing tests included psychophysical, physiological and electrophysiological measures, (pure-tone audiometry, tympanometry, auditory brainstem responses and acoustic reflexes). The tests were perfomed in line with the procedure guidelines by the British Society of Audiology (www.thebsa.org.uk). The mean value of pure-tone audiometric thresholds for both ears measured in decibel hearing level (as described in the guidelines by the British Society of Audiology) was calculated and reported. Other parameters were reported as normal or abnormal (in at least one ear) or inconclusive.

Recruitment status

COMPLETED

Target enrollment

11 participants

Primary outcome timeframe

Baseline visit

Results posted on

2025-06-10

Participant Flow

Of the 90 anticipated participants, only 11 participants were recruited to the study - into the Siderosis group. Participants were not recruited into the non siderosis groups (ie Age-Related Hearing Loss group and Control group) due COVID19 restrictions at the time of the study, which impacted significantly on recruitment opportunities and dramatically reduced the participant number in the study.

There were limited opportunities to recruit participants into study, (we anticipated 30 participants in each group). The apparent discrepancy between the anticipated recruitment number and the actual number of participants across all groups was due to COVID-19 pandemic and social restrictions placed. Subsequent easing off of the restrictions allowed to recruit 11 participants (from November 2019 to July 2021) but study-related time constraints did not allow to proceed with further recruitment.

Participant milestones

Participant milestones
Measure	Siderosis (iSS) Group participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust	Age-related Hearing Loss (ARHL) Group Age-Related Hearing Loss group not recruited due to the impact of COVID19 on recruitment opportunities.	Control Group Control group not recruited due to the impact of COVID19 on recruitment opportunities.
Overall Study STARTED	11	0	0
Overall Study COMPLETED	10	0	0
Overall Study NOT COMPLETED	1	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Siderosis (iSS) Group participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust	Age-related Hearing Loss (ARHL) Group Age-Related Hearing Loss group not recruited due to the impact of COVID19 on recruitment opportunities.	Control Group Control group not recruited due to the impact of COVID19 on recruitment opportunities.
Overall Study Withdrawal by Subject	1	0	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Siderosis (iSS) Group n=10 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Age, Customized	52.5 years STANDARD_DEVIATION 14.5 • n=10 Participants
Sex: Female, Male Female	2 Participants n=10 Participants
Sex: Female, Male Male	8 Participants n=10 Participants

PRIMARY outcome

Timeframe: Baseline visit

Population: Participants with known diagnosis of infratentorial superficial siderosis.

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=11 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Hearing Evaluation - Pure Tone Audiometry	57.3 decibel hearing level Standard Deviation 39.9

PRIMARY outcome

Timeframe: Baseline visit

Population: Vestibular tests included video head impulse test, oculomotor and rotatory chair testing using video or electronystagmography; bithermal calorics, ocular and cervical vestibular evoked myogenic potentials. The results are presented as normal if bilateral or unilateral normal findings;

Clinical vestibular and balance tests were performed to assess vestibular/balance function.

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=10 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Vestibular/Balance Evaluation Bithermal calorics · Normal	2 Participants
Vestibular/Balance Evaluation Bithermal calorics · Not tested	7 Participants
Vestibular/Balance Evaluation Video head impulse test · Abnormal	6 Participants
Vestibular/Balance Evaluation Video head impulse test · Normal	1 Participants
Vestibular/Balance Evaluation Video head impulse test · Not tested	3 Participants
Vestibular/Balance Evaluation Video- electro-nystagmography (oculomotor; rotatory chair) - central vestibular assessment · Abnormal	9 Participants
Vestibular/Balance Evaluation Video- electro-nystagmography (oculomotor; rotatory chair) - central vestibular assessment · Normal	1 Participants
Vestibular/Balance Evaluation Video- electro-nystagmography (oculomotor; rotatory chair) - central vestibular assessment · Not tested	0 Participants
Vestibular/Balance Evaluation Video- electronystagmography (oculomotor; rotatory chair) - peripheral vestibular assessment · Abnormal	7 Participants
Vestibular/Balance Evaluation Video- electronystagmography (oculomotor; rotatory chair) - peripheral vestibular assessment · Normal	3 Participants
Vestibular/Balance Evaluation Video- electronystagmography (oculomotor; rotatory chair) - peripheral vestibular assessment · Not tested	0 Participants
Vestibular/Balance Evaluation Bithermal calorics · Abnormal	1 Participants
Vestibular/Balance Evaluation ocular Vestibular Evoked Myogenic Potentials · Abnormal	0 Participants
Vestibular/Balance Evaluation ocular Vestibular Evoked Myogenic Potentials · Normal	9 Participants
Vestibular/Balance Evaluation ocular Vestibular Evoked Myogenic Potentials · Not tested	1 Participants
Vestibular/Balance Evaluation cervical Vestibular Evoked Myogenic Potentials · Abnormal	3 Participants
Vestibular/Balance Evaluation cervical Vestibular Evoked Myogenic Potentials · Normal	7 Participants
Vestibular/Balance Evaluation cervical Vestibular Evoked Myogenic Potentials · Not tested	0 Participants

PRIMARY outcome

Timeframe: Baseline visit

Population: Results for psychophysical, physiological/electrophysiological measures (units: participants); normal if bilateral or unilateral normal findings; Auditory brainstem responses were recorded as normal, abnormal or inconclusive

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=11 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Hearing Evaluation - Auditory Brainstem Responses/Acoustic Reflexes Abnormal	5 Participants
Hearing Evaluation - Auditory Brainstem Responses/Acoustic Reflexes Inconclusive	2 Participants
Hearing Evaluation - Auditory Brainstem Responses/Acoustic Reflexes Not performed	4 Participants

PRIMARY outcome

Timeframe: Baseline

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=11 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Hearing Evaluation - Tympanogram Abnormal	0 Participants
Hearing Evaluation - Tympanogram Normal	11 Participants

PRIMARY outcome

Timeframe: Baseline

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=11 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Hearing Evaluation - Otoacoustic Emissions Present	3 Participants
Hearing Evaluation - Otoacoustic Emissions Absent	3 Participants
Hearing Evaluation - Otoacoustic Emissions Not performed	5 Participants

PRIMARY outcome

Timeframe: Baseline

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=11 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Hearing Evaluation - Listening in Spatialised Sentences Noise Test Abnormal	4 Participants
Hearing Evaluation - Listening in Spatialised Sentences Noise Test Normal	1 Participants
Hearing Evaluation - Listening in Spatialised Sentences Noise Test Not tested	6 Participants

SECONDARY outcome

Timeframe: Baseline visit

Siderosis group participants underwent formal imaging procedures as part of their clinical care pathway. The imaging results were reviewed with the results obtained from hearing and vestibular/balance assessments. All participants had MRI Brain, as part of their clinical care pathway; susceptibility-weighted images were reviewed to identify the appearance of superficial siderosis, in keeping with the radiological diagnostic criteria (Reference: Wilson et al, 2017).

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=10 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Superior cerebellar vermis · Hypointense signal consistent with superficial siderosis observed	10 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Superior cerebellar vermis · Hypointense signal consistent with superficial siderosis not observed	0 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Cerebellar folia · Hypointense signal consistent with superficial siderosis observed	10 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Cerebellar folia · Hypointense signal consistent with superficial siderosis not observed	0 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Midbrain · Hypointense signal consistent with superficial siderosis observed	10 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Midbrain · Hypointense signal consistent with superficial siderosis not observed	0 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Pons · Hypointense signal consistent with superficial siderosis observed	9 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Pons · Hypointense signal consistent with superficial siderosis not observed	1 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Medulla · Hypointense signal consistent with superficial siderosis observed	9 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Medulla · Hypointense signal consistent with superficial siderosis not observed	1 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results 8th cranial nerve (unilateral or bilateral) · Hypointense signal consistent with superficial siderosis observed	9 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results 8th cranial nerve (unilateral or bilateral) · Hypointense signal consistent with superficial siderosis not observed	1 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Supratentorial extension · Hypointense signal consistent with superficial siderosis observed	10 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Supratentorial extension · Hypointense signal consistent with superficial siderosis not observed	0 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Sylvian fissures · Hypointense signal consistent with superficial siderosis observed	9 Participants
Correlation Between Clinically Obtained Imaging (Siderosis Group Participants) and the Hearing and Balance Assessment Results Sylvian fissures · Hypointense signal consistent with superficial siderosis not observed	1 Participants

SECONDARY outcome

Timeframe: Baseline visit

Siderosis group participants underwent a formal neuro-cognitive assessment as part of their clinical care pathway. The results of this clinical assessment were reviewed to determine if an association exists between the cognitive assessment results and the results obtained from hearing and vestibular/balance assessments performed during the study. A formal neuro-cognitive assessment was performed using the Wechsler Adult Intelligence Scale test. Cognitive impairment in a domain was defined as scoring at or below the fifth percentile on any one test in that domain.

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=7 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Correlation Between Clinically Obtained Results of Cognitive Function Assessment (Siderosis Group Participants) and the Hearing and Balance Assessments Participants who underwent neurocognitive assessment	7 Participants
Correlation Between Clinically Obtained Results of Cognitive Function Assessment (Siderosis Group Participants) and the Hearing and Balance Assessments Executive function abnormal	5 Participants
Correlation Between Clinically Obtained Results of Cognitive Function Assessment (Siderosis Group Participants) and the Hearing and Balance Assessments Visual memory abnormal	5 Participants

SECONDARY outcome

Timeframe: Baseline visit

University of Pennsylvania Smell Identification Test (UPSIT, British version) was performed (siderosis group participants) to assess their olfactory function. UPSIT is a forced-choice 40-item self-administered test. The scores are calculated using manufacturer's answer key as the sum of correct answers (maximum best = 40, minimum score 6; anosmia \<19(males, females); normosmia \>33 (males) and \>34 (females)), and compared to the age- and gender-matched norms. The scores are also grouped into categories (ranging from normosmia to anosmia), with higher scores indicating better olfactory function.

Outcome measures

Outcome measures
Measure	Siderosis (iSS) Group n=10 Participants Participants with a known diagnosis of infratentorial superficial siderosis (defined using standardised radiological criteria) confirmed by a consultant neurologist with expertise in this condition at University College London Hospitals National Health Service (NHS) Foundation Trust
Smell Identification Test	25.5 UPSIT score Standard Deviation 7.8

Adverse Events

Siderosis (iSS) Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr N Kharytaniuk (Clinical Research Fellow)

Ear Institute, University College London

Phone: 02076792000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place