Early Prediction and Prevention of Cardiac Insult in Pediatric Patients with Lysosomal Storage Diseases.

Early Prediction and Prevention of Asymptomatic Cardiac Insult in Pediatric Patients with Lysosomal Storage Diseases: (Evaluation of CoQ10 as a Cardioprotective Drug)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

PACTR

Registry ID

PACTR202107466690046

Enrollment

Registered

2021-07-13

Start date

2019-08-01

Completion date

Unknown

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paediatrics

Interventions

coenzyme q10

placebo

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Pediatric patients with Lysosomal storage disease aged from 1 to 18 years diagnosed by enzyme assay.

Exclusion criteria

Exclusion criteria: 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.

Design outcomes

Primary

Measure	Time frame
The primary outcome is to perform comprehensive evaluation of cardiac function in patients with a lysosomal storage disease.	—

Secondary

Measure	Time frame
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. ;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.	—

Measure

Time frame

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. ;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.

—

Countries

Egypt

Contacts

Public ContactOsama Toulba

Professor of pediatrics

usama.toulba@med.tanta.edu.eg0201026572236

Outcome results

None listed

Source: PACTR (via WHO ICTRP) · Data processed: Aug 9, 2026