Pan African Clinical Trials Registry
South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0506 / +27 21 938 0834 Fax: +27 21 938 0836
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR202107466690046 Date of Approval: 13/07/2021
Trial Status: Retrospective registration - This trial was registered after enrolment of the first participant
TRIAL DESCRIPTION
Public title Early Prediction and Prevention of Cardiac Insult in Pediatric Patients with Lysosomal Storage Diseases.
Official scientific title Early Prediction and Prevention of Asymptomatic Cardiac Insult in Pediatric Patients with Lysosomal Storage Diseases: (Evaluation of CoQ10 as a Cardioprotective Drug)
Brief summary describing the background and objectives of the trial Cardiac disease is particularly important in lysosomal Glycogen storage diseases (Pompe and Danon disease), mucopolysaccharidoses and in sphingolipidoses. Cardiac involvement may be seen in the form of cardiac hypertrophy, coronary artery disease, valvular disease, rhythm disorders or other vascular involvements. The aim of this study will be to perform comprehensive evaluation of cardiac function in patients with a lysosomal storage disease. Our secondary aim will be to measure a biomarker which could be associated with impaired cardiac function and correlate this biomarker with echo findings. The third aim will be to study the possible cardioprotective effect of Co enzyme Q10 as cardioprotective drug in those patients.
Type of trial RCT
Acronym (If the trial has an acronym then please provide)
Disease(s) or condition(s) being studied Paediatrics
Sub-Disease(s) or condition(s) being studied
Purpose of the trial Treatment: Drugs
Anticipated trial start date 01/08/2019
Actual trial start date 01/08/2019
Anticipated date of last follow up 01/08/2021
Actual Last follow-up date 01/08/2021
Anticipated target sample size (number of participants) 20
Actual target sample size (number of participants) 25
Recruitment status Completed
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Parallel: different groups receive different interventions at same time during study Non-randomised Sealed opaque envelopes Masking/blinding used Care giver/Provider,Outcome Assessors,Participants
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group coenzyme q10 2mg per kilogram. 24 weeks patients will receive oral coenzyme q10 ,2mg/kg, once daily for 24 weeks.Echocardiography will be done for all participants before and after treatment and blood samples also will be taken before and after treatment to measure biomarkers like N-terminal prpBNP and serum malondialdehyde. 12
Control Group placebo once daily 24 weeks ptients will receive placebo once daily for 24 weeks.Echocardiography will be done for all participants before and after treatment and blood samples also will be taken before and after treatment to measure biomarkers like N-terminal prpBNP and serum malondialdehyde. 12 Placebo
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Pediatric patients with Lysosomal storage disease aged from 1 to 18 years diagnosed by enzyme assay. Lysosomal storage disease patients with diabetes mellitus, cancer and bronchial asthma as these diseases may cause cardiac diseases and may alter patient's response to the used drug and may affect the level of the studied biomarkers. Adolescent: 13 Year-18 Year,Child: 6 Year-12 Year,Preschool Child: 2 Year-5 Year 1 Year(s) 18 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes 24/07/2019 Tanta university ethics committee.
Ethics Committee Address
Street address City Postal code Country
Tanta, El geish street, faculty of medecine. Tanta 31511 Egypt
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome The primary outcome is to perform comprehensive evaluation of cardiac function in patients with a lysosomal storage disease. at the begeining of the study.
Secondary Outcome the secondary outcom will be to measure biomarkers which could be associated with impaired cardiac function and correlate this biomarker with echo findings before and after treatment with coenzyme q10. after end of treatment which is 24 weeks.
Secondary Outcome The secondary aim will be to study the possible cardioprotective effect of Co enzyme Q10 as cardioprotective antioxidant drug in those patients by evaluating serum malondialdehyde before and after treatment with coenzyme q10. after 24 weeks of using coenzymeq10.
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Tanta university hospitals El geish street. Tanta 31511 Egypt
FUNDING SOURCES
Name of source Street address City Postal code Country
self fund 10 El mawardy street from el Helw street Tanta Egypt
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Faculty of medecine of Tanta university el geish street. Tanta 31511 Egypt University
COLLABORATORS
Name Street address City Postal code Country
Yasmine buzaid el mawardy street from el helw street. Tanta Egypt
Heba Elsayed Dawoud Elgeish Tanta Egypt
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Yasmine Abuzaid yasmine.abuzaid@med.tanta.edu.eg 0201000421060 10,Elmawardy street from el Helw street
City Postal code Country Position/Affiliation
Tanta Egypt assistant lecturer
Role Name Email Phone Street address
Scientific Enquiries Heba Dawoud hebadawoud173@yahoo.com 0201094623100 pediatric department,tanta university ,Elgeish street.
City Postal code Country Position/Affiliation
Tanta Egypt lecturer of pediatrics
Role Name Email Phone Street address
Public Enquiries Osama Toulba usama.toulba@med.tanta.edu.eg 0201026572236 Pediatric department,Tanta university ,ElGeish street.
City Postal code Country Position/Affiliation
Tanta Egypt Professor of pediatrics
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes All of the individual participant data collected during the trial after deidentification will be available for researchers who provide a sound proposal. Informed Consent Form,Statistical Analysis Plan,Study Protocol Beginning 3 months and ending 5 years following article publication Proposal should be directed to 'yasmine.abuzaid@med.tanta.edu.eg'. To gain access, data requestors will need to sign a data access agreement
URL Results Available Results Summary Result Posting Date First Journal Publication Date
No
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information