Trial from ANZCTR


Trial ID ACTRN12612000226808
Trial Status: Registered
Date Submitted: 15/02/2012
Date Registered: 22/02/2012
Prospectively registered

Page 1

Public title A safety and feasibility study of oral Triheptanoin as an add on treatment for patients (12 years or older) with medical refractory epilepsy.
Update:
 
Reason:
 
Study title in 'Participant- Intervention- Comparator- Outcome (PICO)' format A Phase IIa randomized double-blind placebo controlled study to evaluate the safety and feasibility of oral triheptanoin as an add-on treatment to adolescent and adult patients with medically refractory epilepsy
Update:
 
Reason:
 
Secondary ID [1] 279945 0
Nil
Update:
 
Reason:
 
UTN Nil
Update:
 
Reason:
 
Trial acronym TRIP - E
Update:
 

Page 2

Health condition(s) or problem(s) studied:
Medical refractory epilepsy 285862 0
 Update:
 Reason:
Condition category: Condition code:
Neurological Epilepsy
 Update:
 Update:
  Reason:
286045 286045 0 0

Page 3

Descriptions of intervention(s) / exposure The patient will be up titrated to a maximum tolerated dose of Triheptanoin during a three week titration period. (Start dose 15 ml) The total daily dose will be between 15 and 100ml daily depending on the patients tolerability (Up to 35% of caloric input per day). Patients will be take the maximum tolerated dose over a period of 16 weeks and assessed during this time. Patients will take Triheptanoin orally three to four times per day with meals. They will be asked to complete a food and seizure diary
Update:
 
Reason:
 
Intervention Code:
Treatment: drugs 284275 0
Update:
 
Reason:
 
Comparator / control treatment The patient will be up titrated to a maximum tolerated dose of C8/C10 (Comparator) during a three week titration period. (Start dose 15 ml) The total daily dose will be between 15 and 100ml daily depending on the patients tolerability (Up to 35% of caloric input per day). Patients will be take the maximum tolerated dose over a period of 16 weeks and assessed during this time. Patients will take C8/C10 (Comparator)orally three to four times per day with meals. They will be asked to complete a food and seizure diary
Update:
 
Reason:
 
Control group Placebo
Update:
 
Reason:
 

Page 4

Primary Outcome: Safety. Possible adverse events include gastrointestinal upset and diarrhoea. 286528 0
Update:
 
Reason:
 
Timepoint: Monitored weekly during 3 week titration period and then four weekly over the 16 week treatment period. Safety will be assessed based on reported adverse events. 286528 0
Update:
 
Reason:
 
Secondary Outcome: Complience with treatment 296088 0
Update:
 
Reason:
 
Timepoint: Will be monitored weekly during the titration period and then 4 weekly during the 16 week treatment period. Complience will be assessed by amount of oil returned at each study visit. 296088 0
Update:
 
Reason:
 
Secondary Outcome: Tolerability of treatment 296151 0
Update:
 
Reason:
 
Timepoint: Will be monitored weekly during the titration period and then 4 weekly during the 16 wekk treatment period. Tolerability will be assessed based on discussions with the patient, assessment of the food diary and assessment of adverse events. 296151 0
Update:
 
Reason:
 

Page 5

Key inclusion criteria Male or female patients (12 years or older) with epilepsy who have experienced at least 4 seizures of an eligible type per month over two months prior to enrolment despite treatment with at least one anti-epileptic drug 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) Patients anti-epileptic drugs over the four weeks prior to enrolment must remain stable with no change.
Patients must be able to provide informed consent.
Update:
 
Reason:
 
Minimum age 12 Years
Update:
 
Reason:
 
Update:
 
Reason:
 
Maximum age No limit
Update:
 
Reason:
 
Update:
 
Reason:
 
Gender Both males and females
Update:
 
Reason:
 
Healthy volunteers? No
Update:
 
Reason:
 
Key exclusion criteria Patients with a severe intellectual handicap

Patients with a history of major psychiatric morbidity (such as psychiatric illness requiring hospitalisation or history of psychosis or major depression)

Patients with history of substance abuse

Patients with a history of having had psychogenic non-epileptic seizures

Patients who have seizure clusters or other reasons that make counting of numbers of seizures inaccurate

Females who are pregnant or breast feeding

Patients 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.
Update:
 
Reason:
 

Page 6

Study type Interventional
Update:
 
Reason:
 
Purpose of the study Treatment
Update:
 
Allocation to intervention Randomised controlled trial
Update:
 
Reason:
 
Describe the procedure for enrolling a subject and allocating the treatment (allocation concealment procedures) Subjects will be randomised to recieve either Triheptanoin or Placebo at Week 8 (Visit 2). Patients will be assigned a unique Identification Number, which will be allocated in ascending order of random numbers based on the predetermined randomisation schedule, and according to their chronological order of inclusion in the study. Subjects will then be allocated corresponding treatment, labelled with the same number. Confirmation of the treatment number allocated will be documented in the drug accountability records and recorded in the CRF. Triheptanoin oil and placebo oil will be identical in appearance. The nature of treatment each subject will receive will not be disclosed to the investigator, study site personnel or subjects.
Update:
 
Reason:
 
Describe the methods used to generate the sequence in which subjects will be randomised (sequence generation) A computer generated randomisation schedule (1:1 ratio triheptanoin to placebo oil treatments) will be prepared by a statistician prior to the start of the study and stratified by each study centre. This schedule will be administered by the CRO, NTA.
Update:
 
Reason:
 
Masking / blinding Blinded (masking used)
Update:
 
Reason:
 
Who is / are masked / blinded (choose all that apply)


Update:
       
Reason:
 
Assignment Parallel
Update:
 
Reason:
 
Other design features Nil
Update:
 
Reason:
 
Type of endpoint(s) Safety
Update:
 
Reason:
 
Statistical Methods/Analysis
Update:
 
Reason:
 

Page 7

Phase Phase 2
Update:
 
Reason:
 
Anticipated date of first participant enrolment 1/04/2012
Update:
 
Reason:
 
Date of first participant enrolment
Update:
 
Reason:
 
Anticipated date last participant recruited/enrolled
Update:
 
Reason:
 
Actual date last participant recruited/enrolled
Update:
 
Reason:
 
Target sample size 60
Update:
 
Reason:
 
Recruitment status Recruiting
Update:
 
Reason:
 

Recruitment in Australia

Recruitment state(s)
Update:
 
Reason:
 

Recruitment outside Australia

Page 8

Funding Source: University 284714 0
Update:
 
Reason:
 
Name: University of Queensland 284714 0
Update:
 
Reason:
 
Address: St Lucia, Brisbane QLD 4072 284714 0
Update:
 
Reason:
 
Country: Australia 284714 0
Update:
 
Reason:
 
Funding Source: Charities/Societies/Foundations 284715 0
Update:
 
Reason:
 
Name: Epilepsy Therapy Project 284715 0
Update:
 
Reason:
 
Address: PO Box 742
10N Pendleton St
Middleburg VA 20118
284715 0
Update:
 
Reason:
 
Country: United States of America 284715 0
Update:
 
Reason:
 
Primary Sponsor University
Update:
 
Reason:
 
Name: University of Queensland
Update:
 
Reason:
 
Address: St Lucia, Brisbane, QLD 4072
Update:
 
Reason:
 
Country: Australia
Update:
 
Reason:
 
Secondary Sponsor: None 283615 0
Update:
 
Reason:
 
Name: 283615 0
Update:
 
Reason:
 
Address: 283615 0
Update:
 
Reason:
 
Country: 283615 0
Update:
 
Reason:
 

Page 9

Has the study received approval from at least one Ethics Committee? Yes
Update:
 
Reason:
 
Ethics Committee name: Royal Melbourne Hospital 286720 0
Update:
 
Reason:
 
Address: Grattan Street Parkville 3050 286720 0
Update:
 
Reason:
 
Country: Australia 286720 0
Update:
 
Reason:
 
Approval Date: 30/03/2012 286720 0
Update:
 
Reason:
 
Submitted Date: 22/02/2012 286720 0
Update:
 
Reason:
 
HREC: HREC 2012.059 286720 0
Update:
 
Reason:
 
Brief summary A study to determine the safety, tolerability and complience tripheptanoin oil, taken as an oral supplement by patients 12 years and older, who have medical refractory epilepsy.
Update:
 
Reason:
 
Trial website nil
Update:
 
Trial related presentations / publications nil
Update:
 
Public Notes
Update:
 

Page 10

Principal Investigator
Title:
Update:
 
Reason:
 
33779 0
Name:
Update:
 
Reason:
 
33779 0
Address:
Update:
 
Reason:
 
33779 0
Country:
Update:
 
33779 0
Reason:
 
Tel:
Update:
 
Reason:
 
33779 0
Fax:
Update:
 
Reason:
 
33779 0
Email:
Update:
 
Reason:
 
33779 0
Contact person for public queries
Title:
Update:
 
Reason:
 
17026 0
Name: Dr Karin Borges
Update:
 
Reason:
 
17026 0
Address: Department of Pharmacology School of Biomedical Sciences University of Queensland Brisbane, QLD 4072 Australia
Update:
 
Reason:
 
17026 0
Country: Australia
Update:
 
17026 0
Reason:
 
Tel: +61 7 3365 3113
Update:
 
Reason:
 
17026 0
Fax:
Update:
 
Reason:
 
17026 0
Email: k.borges@uq.edu.au
Update:
 
Reason:
 
17026 0

Contact person for scientific queries
Title:
Update:
 
Reason:
 
7954 0
Name: Dr Karin Borges
Update:
 
Reason:
 
7954 0
Address: Department of Pharmacology School of Biomedical Sciences University of Queensland Brisbane, QLD 4072 Australia
Update:
 
Reason:
 
7954 0
Country: Australia
Update:
 
7954 0
Reason:
 
Tel: +61 7 3365 3113
Update:
 
Reason:
 
7954 0
Fax:
Update:
 
Reason:
 
7954 0
Email: k.borges@uq.edu.au
Update:
 
Reason:
 
7954 0

Contact person responsible for updating information
Title:
Update:
 
Reason:
 
26098 0
Name: Fiona Ellery
Update:
 
Reason:
 
26098 0
Address: Neuroscience Trials Australia 245 Burgundy Street Heidelberg VICTORIA 3084
Update:
 
Reason:
 
26098 0
Country: Australia
Update:
 
26098 0
Reason:
 
Tel: +61 3 9035 7042
Update:
 
Reason:
 
26098 0
Fax: +61 3 9496 2251
Update:
 
Reason:
 
26098 0
Email: fellery@nsri.org.au
Update:
 
Reason:
 
26098 0
   

Addition Cancer fields
Update:
 
Reason:
 
Update:
 
Reason:
 
Update:
 
Reason:
 
Update:
 
Reason:
 
Update:
 
Reason:
 
Update:
 
Reason: