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Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital

NCT ID: NCT05029232

Last Updated: 2021-08-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-01

Study Completion Date

2023-08-01

Brief Summary

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Muscular dystrophies are a heterogenous group of inherited muscular disorders characterized by progressive muscle weakness. Historically, these disorders are difficult to treat. In the last three decades, there is a great progress in molecular and genetic basis of these disorders; early diagnosis is achievable with proper clinical recognition and advanced genetic testing .Duchenne Muscular Dystrophy (DMD) is a neuromuscular muscular X-linked recessive disorders that belong to a group of disorders known as dystrophinopathies. DMD characterized by a progressive degeneration of skeletal muscles, with symptoms that manifest early, at around 3 years, causing loss of ambulation within the 13 years of life, followed by cardiac complication (e.g., dilated cardiomyopathy and arrhythmia) and respiratory disorders, including chronic respiratory failure. The unique medical treatment available is steroid therapy, which appears to prolong walking capacity by at least two years. Thus, besides medical treatment, the physical therapy in multidisciplinary care is imperative for alleviating muscle atrophy, skeletal deformities, and motor function deterioration.

Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SCREENING

Blinding Strategy

NONE

Study Groups

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ambulant patient with DMD

patient that walk alone or with minor assist

Group Type ACTIVE_COMPARATOR

MLPA for duchenne

Intervention Type DIAGNOSTIC_TEST

MLPA test for genetic testing to detect gene affection in DMD , and other tests for confirmation and follow up

non ambulant patient with DMD

patient need wheel chair

Group Type ACTIVE_COMPARATOR

MLPA for duchenne

Intervention Type DIAGNOSTIC_TEST

MLPA test for genetic testing to detect gene affection in DMD , and other tests for confirmation and follow up

Interventions

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MLPA for duchenne

MLPA test for genetic testing to detect gene affection in DMD , and other tests for confirmation and follow up

Intervention Type DIAGNOSTIC_TEST

Other Intervention Names

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muscle enzymes thyroid function EMG , nerve conduction for limbs echocardiography , MRI brain , IQ test

Eligibility Criteria

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Inclusion Criteria

1. age of onset between 3- and 18-year-old
2. typical clinical manifestation of Duchenne muscular dystrophy
3. clinical manifestation confirmed by specific biochemical analysis or by genetic testing who presented to pediatric department and neurology outpatient clinic during the period of study.

Exclusion Criteria

1. children with another congenital muscular dystrophy
2. children with other types of myopathies
3. presence of CNS disorders such as brain insult \& spinal muscular atrophy
4. female gender
Minimum Eligible Age

3 Years

Maximum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Sohag University

OTHER

Sponsor Role lead

Responsible Party

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Nehal Samy Abdo

Assistant lecturer of pediatric. sohag university hospitals

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Sohag University Hospital

Sohag, , Egypt

Site Status

Countries

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Egypt

Central Contacts

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nehal s abdel magoud, assistant lecturer

Role: CONTACT

Phone: 01091666230

Email: [email protected]

abdel rahim A sadek, professor

Role: CONTACT

Phone: 01065067057

Email: [email protected]

Facility Contacts

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Osama R ElSherif, professor

Role: primary

References

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Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.

Reference Type BACKGROUND
PMID: 29395989 (View on PubMed)

Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. Epub 2018 Feb 3.

Reference Type BACKGROUND
PMID: 29395990 (View on PubMed)

Giliberto F, Radic CP, Luce L, Ferreiro V, de Brasi C, Szijan I. Symptomatic female carriers of Duchenne muscular dystrophy (DMD): genetic and clinical characterization. J Neurol Sci. 2014 Jan 15;336(1-2):36-41. doi: 10.1016/j.jns.2013.09.036. Epub 2013 Oct 5.

Reference Type BACKGROUND
PMID: 24135430 (View on PubMed)

Other Identifiers

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Soh-Med-21-07-21

Identifier Type: -

Identifier Source: org_study_id