Limb-girdle Muscular DystrophyNCT06399770
The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy
An observational study of Limb-girdle Muscular Dystrophy, run by Assiut University.
Trial summary
1. to detect the characteristic patterns of muscle involvement in suspected cases of LGMD using muscle ultrasound 2. to use the muscle ultrasound findings clinically categorized the different types of LGMD 3. to correlate the muscle ultrasound findings with the findings of the other assissed scales Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Limb-girdle Muscular Dystrophy
- What is being tested?
- Muscle ultrasound
- Type of treatment
- Medical device
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2024to 1 Jul 2026
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: * Patients presented with clinical manifestations suggestive of LGMD who presented with gradual progressive limb-girdle weakness ,myopathic changes on electromyography and elevated creatine phosphokinase serum level * age and sex matched healthy control group as a normal referencce
Who may be able to join
- gender : both sex are included Willingness to participate in the study and to be subjected to disease related examination and assessments Willing and able to provide informd consent
Who cannot join
- patients unable to give informed consent Patients with acute or subacute onset of symptoms of muscle involvement including inflammatory myopathies Patients with systemic diseases causing secondary myopathy
Trial locations and contacts
No sites are listed yet.
About this study
- to detect the characteristic patterns of muscle involvement in suspected cases of LGMD using muscle ultrasound
- to use the muscle ultrasound findings clinically categorized the different types of LGMD
- to correlate the muscle ultrasound findings with the findings of the other assissed scales
Read the detailed description
Limb-girdle muscular dystrophies (LGMD) are muscular dystrophies that affect skeletal muscles, mostly proximal (hips and shoulder muscles). They are caused by a mutation in a gene encoding a protein, which is specific to each subtype. LGMD inheritance is either autosomal dominant or recessive. Several types of LGMD were described depending on the causative mutations (1). LGMD is one of inherited myopathies that characterized by selective involvement of muscles. Selectivity is a sign of muscle diseases and helps direct the diagnosis; however, it cannot always be identified clinically; consequently, the importance of using muscle MRI and ultrasound are to detect this selectivity and aid in the diagnosis (2). Ultrasound with a high resolution enables viewing of the muscle, nerve, and nearby structures and can provide real-time information in neuromuscular illnesses (3). Muscle Ultrasound (MUS) can detect the same patterns of muscle affection as MRI (4). In addition, it is safe, accessible, low-cost, and free of ionizing radiation, with no known contraindications and no difficulties associated with MRI, such as claustrophobia, metallic implants, and the requirement for sedation in children; hence, it can be used as a supplemental technique to electro- diagnosis (5). Ultrasound can provide real-time information related to muscle activation and movement patterns; so, selective substitution of muscle ultrasound for MRI can result in significant cost-saving for the health care system (6). However, its primary drawback is that it is operator-dependent and has a limited ability to image deeper structures (7). According to studies, MUS can provide a key provisional probable diagnosis, especially when genetic diagnosis is not accessible or expensive, or to provide guide to the proper genetic testing when it is available (2, 8). In limb girdle pattern of weakness patients, MUS assisted diagnose quadriceps sparing myopathy, selective affection of one head of gastrocnemius as medial head and soleus more than lateral head in FKRP (LGMD2I) and calpain-3 deficiency (LGMD2A), and selective affection of lateral head as in LGMD2B.
(dysferlin) (9). MUS can also help diagnose the treatable types of limb girdle myopathies like POPME by recognizing specific muscle involvement patterns (2). Few research (10), utilized the MUS to detect suspected LGMD, and none compared the MUS findings with those of the different psychometric measures. The present study aims to we aimed to evaluate the role of MUS in the assessment of patients with suspected LGMD to identify characteristic patterns these of muscle involvement in suspected cases of LGMD and to correlate changes with findings of the different psychometric scales.
- Conditions
- Limb-girdle Muscular Dystrophy
- Being tested
- Muscle ultrasound
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Muscle ultrasoundDevice
Role of muscle ultrasound in LGMD
What is being measured
The primary outcome is the main question the study is designed to answer.
The role of muscle ultrasound in assessment of a sample of patients with limb-girdle muscular dystrophy
The role of muscle ultrasound in assessment of sample of patients with LGMD
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2024
- Study start1 Jun 2024 (estimated)
- Primary completion1 Jun 2026 (estimated)
- Study completion1 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb-girdle Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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-control
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Abanoub Bassem Fikry Ramzy, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06399770
- Sponsor's protocol ID
- Limb-girdle muscular dystrophy
- Status last verified
- May 2024
- First submitted
- 1 May 2024
- Last updated
- 6 May 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06399770
- Acronym / sponsor ID
- Limb-girdle muscular dystrophy
- Conditions
- Limb-girdle Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Jun 2024 (estimated)
- Primary completion
- 1 Jun 2026 (estimated)
- Study completion
- 1 Jul 2026 (estimated)
- First posted
- 6 May 2024 (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.
- to detect the characteristic patterns of muscle involvement in suspected cases of LGMD using muscle ultrasound
- to use the muscle ultrasound findings clinically categorized the different types of LGMD
- to correlate the muscle ultrasound findings with the findings of the other assissed scales
Detailed description
Limb-girdle muscular dystrophies (LGMD) are muscular dystrophies that affect skeletal muscles, mostly proximal (hips and shoulder muscles). They are caused by a mutation in a gene encoding a protein, which is specific to each subtype. LGMD inheritance is either autosomal dominant or recessive. Several types of LGMD were described depending on the causative mutations (1). LGMD is one of inherited myopathies that characterized by selective involvement of muscles. Selectivity is a sign of muscle diseases and helps direct the diagnosis; however, it cannot always be identified clinically; consequently, the importance of using muscle MRI and ultrasound are to detect this selectivity and aid in the diagnosis (2). Ultrasound with a high resolution enables viewing of the muscle, nerve, and nearby structures and can provide real-time information in neuromuscular illnesses (3). Muscle Ultrasound (MUS) can detect the same patterns of muscle affection as MRI (4). In addition, it is safe, accessible, low-cost, and free of ionizing radiation, with no known contraindications and no difficulties associated with MRI, such as claustrophobia, metallic implants, and the requirement for sedation in children; hence, it can be used as a supplemental technique to electro- diagnosis (5). Ultrasound can provide real-time information related to muscle activation and movement patterns; so, selective substitution of muscle ultrasound for MRI can result in significant cost-saving for the health care system (6). However, its primary drawback is that it is operator-dependent and has a limited ability to image deeper structures (7). According to studies, MUS can provide a key provisional probable diagnosis, especially when genetic diagnosis is not accessible or expensive, or to provide guide to the proper genetic testing when it is available (2, 8). In limb girdle pattern of weakness patients, MUS assisted diagnose quadriceps sparing myopathy, selective affection of one head of gastrocnemius as medial head and soleus more than lateral head in FKRP (LGMD2I) and calpain-3 deficiency (LGMD2A), and selective affection of lateral head as in LGMD2B.
(dysferlin) (9). MUS can also help diagnose the treatable types of limb girdle myopathies like POPME by recognizing specific muscle involvement patterns (2). Few research (10), utilized the MUS to detect suspected LGMD, and none compared the MUS findings with those of the different psychometric measures. The present study aims to we aimed to evaluate the role of MUS in the assessment of patients with suspected LGMD to identify characteristic patterns these of muscle involvement in suspected cases of LGMD and to correlate changes with findings of the different psychometric scales.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- * Patients presented with clinical manifestations suggestive of LGMD who presented with gradual progressive limb-girdle weakness ,myopathic changes on electromyography and elevated creatine phosphokinase serum level * age and sex matched healthy control group as a normal referencce
Key inclusion criteria (1 listed)
- gender : both sex are included Willingness to participate in the study and to be subjected to disease related examination and assessments Willing and able to provide informd consent
Key exclusion criteria (1 listed)
- patients unable to give informed consent Patients with acute or subacute onset of symptoms of muscle involvement including inflammatory myopathies Patients with systemic diseases causing secondary myopathy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Muscle ultrasoundRole of muscle ultrasound in LGMD | Device | – |
Primary outcome
The role of muscle ultrasound in assessment of a sample of patients with limb-girdle muscular dystrophy
Time frame: Baseline
The role of muscle ultrasound in assessment of sample of patients with LGMD
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2024.
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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.
Questions about this trial
What is this clinical trial studying?
An observational study of Limb-girdle Muscular Dystrophy, run by Assiut University.
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: status not confirmed, as of the sponsor's last update on 6 May 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Abanoub Bassem Fikry (+201210927033, Abanoubbassem2@icloud.Com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The role of muscle ultrasound in assessment of a sample of patients with limb-girdle muscular dystrophy (time frame: Baseline). 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 6 May 2024.