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NCT03041363

Treatment Development of Triheptanoin (G1D)

Completed Phase 1 Last updated 22 December 2022
What this trial tests

Phase 1 trial testing Triheptanoin in Epilepsy in 12 participants. Completed in 22 December 2017.

Timeline
29 March 2017
Primary endpoint
22 December 2017
22 December 2017

Quick facts

Lead sponsorJuan Pascual
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment12
Start date29 March 2017
Primary completion22 December 2017
Estimated completion22 December 2017
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Juan Pascual

Who can join

Adults 2 to 35, any sex, with Epilepsy or GLUT1DS1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

To determine the maximum tolerated dose (MTD), as a percentage of calories consumed, of triheptanoin (C7 oil; C7) in a pediatric and adult patient population genetically diagnosed with glucose transporter type 1 deficiency disorder (G1D).

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Glut1 Deficiency Syndrome (Glut1DS): State of the art in 2020 and recommendations of the international Glut1DS study group.
    Klepper J, Akman C, Armeno M, Auvin S, et al · · 2020 · cited 186× · PMID 32913944 · DOI 10.1002/epi4.12414
  2. Development and validation of a LC-MS/MS method for quantitation of 3-hydroxypentanoic acid and 3-oxopentanoic acid in human plasma and its application to a clinical study of glucose transporter type I deficiency (G1D) syndrome.
    Kallem RR, Primeaux S, Avila A, Pascual JM, et al · · 2021 · cited 3× · PMID 34482182 · DOI 10.1016/j.jpba.2021.114335
  3. Maximum dose, safety, tolerability and ketonemia after triheptanoin in glucose transporter type 1 deficiency (G1D).
    Málaga I, Avila A, Primeaux S, Kallem RR, et al · · 2023 · cited 1× · PMID 36859467 · DOI 10.1038/s41598-023-30578-z

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Other trials of Triheptanoin

Trials testing the same drug.

Other recruiting trials for Epilepsy

Currently open trials in the same condition.

Other Juan Pascual trials

Trials by the same sponsor.

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