Spinal Muscular AtrophyNCT02124616
National Registry for Egyptian Pediatric Neuromuscular Diseases
An observational study of Spinal Muscular Atrophy and Muscular Dystrophy, run by Ain Shams University at 1 site in Egypt.
Trial summary
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases
- How many people
- 200 participants
- Where
- 1 siteEgypt
- Study dates
- Apr 2014to Dec 2020
- How long will I take part?
- 5 Years
- 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 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.
Who may be able to join
- Weakness, hypotonia.
- Nerve conduction study and electromyographic confirmation of lower motor neuron affection.
Who cannot join
- Chromosomal diseases.
- Malformations and deformations.
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
Cairo, Abassia, 11381
Mohsen S Elalfy, MD, PhD, elalfym@hotmail.com
Sahar MA Hassanein, MD, PhD
About this study
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
Read the detailed description
Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis).
Participants: Eligible infants and children with inherited and acquired neuromuscular diseases.
DESIGN: This study is a prospective cohort study.
Outcome measures: Motor development assessment, respiratory and cardiac examination.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes, Polyneuropathies
- Keywords
- spinal muscular atrophy, Muscular dystrophy, Muscle diseases, myasthenic syndromes, Polyneuropathies, Myopathies
- Medical subject headings
- Muscular Atrophy, Spinal, Muscular Dystrophies, Lambert-Eaton Myasthenic Syndrome, Polyneuropathies, Muscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Neuromuscular diseases | Prospective cohort of children with inherited or acquired neuromuscular diseases. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional motor ability
Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital.
Functional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.
Measured: 1 year
Secondary outcomes (1)
Cardio-pulmonary function
Pulmonary and cardiac function impairment.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2014 (estimated)
- Study startApr 2014
- Primary completionApr 2020 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by Ain Shams 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 only
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sahar M.A. Hassanein, MD, Ain Shams University
- Investigators
- Sahar MA Hassanein, MD, PhD, Study chair, Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02124616
- Sponsor's protocol ID
- EGYPT PED-NMD 2014
- Status last verified
- Apr 2014
- First submitted
- 25 Apr 2014
- Last updated
- 28 Apr 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- National Registry for Egyptian Pediatric Neuromuscular Diseases
- ClinicalTrials.gov ID
- NCT02124616
- Acronym / sponsor ID
- EGYPT PED-NMD 2014
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes, Polyneuropathies
- Keywords
- spinal muscular atrophy, Muscular dystrophy, Muscle diseases, myasthenic syndromes, Polyneuropathies, Myopathies
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- Apr 2014
- Primary completion
- Apr 2020 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 28 Apr 2014 (estimated)
- 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.
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
Detailed description
Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis).
Participants: Eligible infants and children with inherited and acquired neuromuscular diseases.
DESIGN: This study is a prospective cohort study.
Outcome measures: Motor development assessment, respiratory and cardiac examination.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neuromuscular diseases | – | –Prospective cohort of children with inherited or acquired neuromuscular diseases. |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.
Key inclusion criteria (2 listed)
- Weakness, hypotonia.
- Nerve conduction study and electromyographic confirmation of lower motor neuron affection.
Key exclusion criteria (2 listed)
- Chromosomal diseases.
- Malformations and deformations.
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
Functional motor ability
Time frame: 1 year
Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital. Functional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.
1 secondary outcome
Cardio-pulmonary function
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2014.
- Faculty of Medicine, Ain Shams University websiteLink provided by the sponsor.
- Ain Shams University web siteLink provided by the sponsor.
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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.
Sahar MA Hassanein, MD, PhD study chair
Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
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 Spinal Muscular Atrophy and Muscular Dystrophy, run by Ain Shams University at 1 site in Egypt.
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 2 inclusion and 2 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 28 Apr 2014. 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 Egypt. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sahar MA Hassanein, MD, PhD (201223183943, saharhassanein@med.asu.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional motor ability (time frame: 1 year). 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 28 Apr 2014.