CompletedPhase 2ACTRN12615000406505

A study to assess the long term retention on treatment and long-term safety and tolerability of Triheptanoin in male and female participants with drug-resistant epilepsy

An open-label extension study of oral Triheptanoin as an add-on treatment to adolescent and adult participants with medically refractory epilepsy


Sponsor

University of Queensland

Enrollment

12 participants

Start Date

Feb 17, 2015

Study Type

Interventional

Conditions

Summary

This is an open label study designed to assess the long term retention on treatment and long-term safety and tolerability of Triheptanoin in participants with drug-resistant epilepsy.


Eligibility

Sex: Both males and femalesMin Age: 16 Yearss

Inclusion Criteria4

  • TRIP-E study (ACTRN12612000226808) for cross referencing purposes
  • Participants randomised in the TRIP-E study and who finished the treatment period with appropriate compliance with diet and study procedures.
  • Ability to give informed consent (Participant, parent or person responsible)
  • Females of childbearing potential must have a negative serum betahCG at Visit 1 and either abstain or use acceptable contraception for the duration of the trial.

Exclusion Criteria5

  • Participants with a severe intellectual handicap
  • History of major psychiatric morbidity (such as psychiatric illness requiring hospitalisation or history of psychosis or major depression)
  • Participants with history of substance abuse, eating disorders or irritable bowel syndrome
  • Females who are pregnant or breast feeding
  • Participants with disorders affecting medium and short chain fatty acid oxidation.

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Interventions

Intervention: Triheptanoin (triglyceride of heptanoate), a stable, edible tasteless oil that has been used in patients with metabolic disorders. Dose:initial dose (e.g. 15ml oil/day)the dose is escal

Intervention: Triheptanoin (triglyceride of heptanoate), a stable, edible tasteless oil that has been used in patients with metabolic disorders. Dose:initial dose (e.g. 15ml oil/day)the dose is escalated every week. Treated with up to 35% of caloric input triheptanoin (max 100 ml oil/day). The oil will be added to a normal diet, titrated up over 3 weeks and full dose given for a period of 48 weeks. Route: Orally three times daily (with meals) for 48 weeks During the full time of the study seizures, adverse events, side effects, weight and quality of life will be recorded. To improve adherence the participants will have regular contact with a dietitian face to face and via phone. Participants/carers will fill in a food diary and treatment diary to monitor compliance and return the used and unused bottles of oil.


Locations(1)

Royal Melbourne Hospital - City campus - Parkville

VIC, Australia

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