Phase I trial of oral triheptanoin add-on treatment for children with medically refractory epilepsy

Phase 1

• Conditions: Medically refractory epilepsy; Neurological - Epilepsy

• Registration Number: ACTRN12614000187640
• Lead Sponsor: niversity of Queensland

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-02-20
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Stopped early
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. Male or female subjects (3-18 years old) with epilepsy who have experienced at least two motor seizures per fortnight over two months prior to enrolment despite treatment with at least one antiepileptic drug (AED) at clinically appropriate doses; (*Eligible seizure types are: complex partial seizures (focal dyscognitive seizures), secondary generalised seizures, simple partial seizures with motor features, primary generalised seizures, tonic seizures, atonic seizures);
2.There has been no change in anti-epileptic drugs over the four weeks prior to enrolment;
3.Females of childbearing potential must have a negative serum Beta subunit of human chorionic gonadotropin (Beta hCG) at Visit 1 and a negative urine pregnancy test prior to randomization at Visit 2. Female subjects of childbearing potential must also agree to be abstinent or to use at least 1 medically acceptable method of contraception. If the subject is on a hormonal type of contraceptive, they must agree to use an additional second approved method of contraception.

Exclusion Criteria

1. History of major psychiatric morbidity (such as psychiatric illness requiring hospitalisation or history of psychosis or major depression) in patients;
2.Patients and/or parents with history of substance abuse;
3.A history of having had psychogenic non-epileptic seizures;
4.Patients who have seizure clusters or other reasons that make counting of numbers of seizures inaccurate;
5.Females who are breast feeding;
6.Patients with propionic academia or methylmalonic acidemia or with disorders affecting medium and short chain fatty acid oxidation. This includes medium-chain acyl-CoA dehydrogenase deficiency - MCAD, short-chain acyl-CoA dehydrogenase deficiency - SCAD, short-chain-3 hydroxyacyl-CoA dehydrogenase deficiency –SCHAD and HMG CoA (3-hydroxy-3-methyl-glutaryl-CoA) synthase deficiency

Study & Design

• Study Type: Interventional
• Study Design: Not specified