Charcot-Marie-Tooth DiseaseNCT05827419
Hearing and Balance Disorders in Peripheral Neuropathy
An observational study of Charcot-Marie-Tooth Disease, run by Nemours Children's Clinic at 1 site in United States.
Trial summary
The long term goal of this proposal is to precisely characterize the auditory and vestibular abilities of children with Charcot-Marie-Tooth (CMT) and how these abilities evolve during the progression of the disorder. Read more about this study
- Who is it for?
- Ages 5 to 21 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2018to 10 Mar 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children and young adults between the ages of 5 and 21 years diagnosed with Charcot-Marie-Tooth will be included.
Who may be able to join
- Individuals diagnosed with Charcot-Marie Tooth
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
The long term goal of this proposal is to precisely characterize the auditory and vestibular abilities of children with Charcot-Marie-Tooth (CMT) and how these abilities evolve during the progression of the disorder. This information will be used to refine the management methods for hearing loss and vestibular disorders in these patients. Given that the phenotypic severity is variable within the CMT patient population, we predict that not all CMT patients will present with auditory and vestibular dysfunction. We will therefore collect specimens (i.e., buccal swabs and saliva) from study participants so that their DNA can be isolated and used to determine the genetic basis for auditory and vestibular dysfunction in peripheral neuropathies.
Read the detailed description
Aim #1: Evaluate the prevalence of auditory and vestibular disorders in children diagnosed with Charcot-Marie-Tooth.
We hypothesize that a significant number of children with Charcot-Marie-Tooth (CMT) will develop auditory and vestibular neuropathies, due to the progression of the disorder. We will examine the characteristics of auditory and vestibular abilities of children and how these relate to the type of CMT.
Aim #2: Examine the progression of hearing and vestibular abilities during the course of the disorder.
We hypothesize that the hearing and vestibular abilities of some children with CMT will deteriorate with time. We will repeat the auditory and vestibular testing on a yearly basis to precisely describe the progression of these abilities.
- Conditions
- Charcot-Marie-Tooth Disease
- Medical subject headings
- Charcot-Marie-Tooth Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of auditory disorders in children diagnosed with Charcot-Marie-Tooth Characteristics.
We will evaluate how many children with CMT have present hearing loss. The presence of hearing loss will be based on the results of the following tests (passed or failed): otoacoustic emissions, auditory brainstem responses, auditory event related potentials, audiometry and speech perception
Measured: 2 years
Prevalence of vestibular disorders in children diagnosed with Charcot-Marie-Tooth
We will evaluate how many children with CMT have present vestibular disorder. The presence of vestibular disorder will be based on the results of the following test (passed or failed): video head impulse test
Measured: 2 years
Progression of hearing loss
We will follow subjects longitudinally to evaluate the progression of hearing loss. The degree of hearing loss will be monitor over time to evaluate if changes are noticed.
All tests listed in Outcome 1 will be repeated and compared to initial values to define the presence or absence of changes in the degree of hearing loss.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2023
- Study start1 Jun 2018
- Primary completion10 Mar 2023
- Study completion10 Mar 2023
Why it stopped: Unable to recruit participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nemours Children's Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Thierry Morlet, PhD, Principal investigator, Nemours
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05827419
- Sponsor's protocol ID
- TM001
- Status last verified
- Oct 2018
- First submitted
- 25 Jun 2018
- Last updated
- 25 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hearing and Balance Disorders in Peripheral Neuropathy
- ClinicalTrials.gov ID
- NCT05827419
- Acronym / sponsor ID
- TM001
- Conditions
- Charcot-Marie-Tooth Disease
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Unable to recruit participants
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Nemours Children's ClinicUniversity, hospital or other
- Study start
- 1 Jun 2018 (actual)
- Primary completion
- 10 Mar 2023 (actual)
- Study completion
- 10 Mar 2023 (actual)
- First posted
- 25 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The long term goal of this proposal is to precisely characterize the auditory and vestibular abilities of children with Charcot-Marie-Tooth (CMT) and how these abilities evolve during the progression of the disorder. This information will be used to refine the management methods for hearing loss and vestibular disorders in these patients. Given that the phenotypic severity is variable within the CMT patient population, we predict that not all CMT patients will present with auditory and vestibular dysfunction. We will therefore collect specimens (i.e., buccal swabs and saliva) from study participants so that their DNA can be isolated and used to determine the genetic basis for auditory and vestibular dysfunction in peripheral neuropathies.
Detailed description
Aim #1: Evaluate the prevalence of auditory and vestibular disorders in children diagnosed with Charcot-Marie-Tooth.
We hypothesize that a significant number of children with Charcot-Marie-Tooth (CMT) will develop auditory and vestibular neuropathies, due to the progression of the disorder. We will examine the characteristics of auditory and vestibular abilities of children and how these relate to the type of CMT.
Aim #2: Examine the progression of hearing and vestibular abilities during the course of the disorder.
We hypothesize that the hearing and vestibular abilities of some children with CMT will deteriorate with time. We will repeat the auditory and vestibular testing on a yearly basis to precisely describe the progression of these abilities.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children and young adults between the ages of 5 and 21 years diagnosed with Charcot-Marie-Tooth will be included.
Key inclusion criteria (1 listed)
- Individuals diagnosed with Charcot-Marie Tooth
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Prevalence of auditory disorders in children diagnosed with Charcot-Marie-Tooth Characteristics.
Time frame: 2 years
We will evaluate how many children with CMT have present hearing loss. The presence of hearing loss will be based on the results of the following tests (passed or failed): otoacoustic emissions, auditory brainstem responses, auditory event related potentials, audiometry and speech perception
Prevalence of vestibular disorders in children diagnosed with Charcot-Marie-Tooth
Time frame: 2 years
We will evaluate how many children with CMT have present vestibular disorder. The presence of vestibular disorder will be based on the results of the following test (passed or failed): video head impulse test
Progression of hearing loss
Time frame: 2 years
We will follow subjects longitudinally to evaluate the progression of hearing loss. The degree of hearing loss will be monitor over time to evaluate if changes are noticed. All tests listed in Outcome 1 will be repeated and compared to initial values to define the presence or absence of changes in the degree of hearing loss.
Results and safety
The study was stopped early (reason given: Unable to recruit participants). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thierry Morlet, PhD principal investigator
Nemours
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Charcot-Marie-Tooth Disease, run by Nemours Children's Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Unable to recruit participants). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence of auditory disorders in children diagnosed with Charcot-Marie-Tooth Characteristics. (time frame: 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2023.