National Registry for Egyptian Pediatric Neuromuscular Di... | NCT02124616

Studies
National Registry for Egyptian Pediatric Neuromuscular Diseases

NCT02124616

Sponsor

Ain Shams University

Status

Unknown status

Last Update Posted

Apr 28, 2014Estimated

Enrollment

200Estimated

Phase

Not provided

Conditions

Spinal Muscular Atrophy
Muscular Dystrophy
Muscle Diseases
Myasthenic Syndromes
Polyneuropathies

Interventions

Not provided

Countries

Egypt

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2014

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2014

Primary Completion Date

Apr 2020Estimated

Completion Date

Dec 2020Estimated

First Submitted Date

Apr 25, 2014

First Submission Date that Met QC Criteria

Apr 25, 2014

First Posted Date

Apr 28, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 25, 2014

Last Update Posted Date

Apr 28, 2014Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Sahar M.A. Hassanein, MD, Professor of Pediatrics, Ain Shams UniversityPrincipal Investigator

Lead Sponsor

Ain Shams UniversityOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

5 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

200Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Neuromuscular diseases		Prospective cohort of children with inherited or acquired neuromuscular diseases.

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	1 year

Secondary Outcomes

Measure	Description	Time Frame
Cardio-pulmonary function	Pulmonary and cardiac function impairment.	12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Weakness, hypotonia.
Nerve conduction study and electromyographic confirmation of lower motor neuron affection.

Exclusion Criteria:

Chromosomal diseases.
Malformations and deformations.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Month

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Sahar MA Hassanein, MD, PhD	Contact	201223183943		saharhassanein@med.asu.edu.eg

Overall Officials

Name	Affiliation	Role
Sahar MA Hassanein, MD, PhD	Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University	Study Chair

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University	Recruiting	Cairo	Abassia	11381	Egypt

References Module

Citations

Not provided

Label	URL
Faculty of Medicine, Ain Shams University website	View source
Ain Shams University web site	View source

ID	Term
D009134	Muscular Atrophy, Spinal
D009136	Muscular Dystrophies
D015624	Lambert-Eaton Myasthenic Syndrome
D011115	Polyneuropathies
D009135	Muscular Diseases

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease