Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02408354
• Date of registration: 24/03/2015
• Prospective Registration: Yes
• Primary sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Public title: Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood HEMIHEP
• Scientific title: "Etude Pilote, Comparative, Monocentrique, randomisée, en Cross Over, en Double Aveugle, Contre Placebo, Testant l'efficacité de l'Huile triheptanoïne Dans Les Hémiplégies Alternantes de l'Enfant" HEMIHEP
• Date of first enrolment: April 15, 2015
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02408354
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Emmanuel Flamand-Roze, MD, PhD
• Affiliation: INSERM UMRS 975, 47 bd de l'hôpital - 75651 Paris Cedex 13

Key inclusion & exclusion criteria

Inclusion Criteria:

- AHC with mutation in ATP1A3 gene

- Age = 15 years and 3 months

- = 6 neurological paroxystic events during the last 3 months prior to the beginning of
the study

- No specific diet

- Covered by french social security

- Patients who freely agree to participate in this study and understand the nature,
risks and benefits of this study and give their written informed consent. (In addition
to the requirement for the consent of parents or the legal representative, adolescents
can provide additional informed consent to participate in clinical trials)

Exclusion Criteria:

- Age < 15 years and 3 months

- Evidence of psychiatric disorder

- Comorbid medical condition that would render them unsuitable for the study, e.g. HIV,
diabetes

- Pregnant or parturient or lactating women

- Absence of double effective contraception at the women old enough to procreate

- Unwillingness to be informed in case of abnormal MRI

- Absence of signed informed consent

- No covered by french social security

- Persons deprived of their liberty by judicial or administrative decision

- Person subject to an exclusion period for another research

- Subjects with exclusion criteria required by french law



Age minimum: 15 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Alternating Hemiplegia of Childhood

Intervention(s)

Drug: Placebo

Drug: Triheptanoin

Primary Outcome(s)

Number of neurologic paroxystic events report in patient diary [Time Frame: 7 months]

Secondary Outcome(s)

Clinical Global Impression Scales - Improvement [Time Frame: 7 months]

Composite score allying the number of neurological paroxystic events, their duration and severity. [Time Frame: 7 months]

Clinical Safety as measured by questionnaire [Time Frame: 7 months]

Biological Safety as measured by acylcarnitine profile, organic acid dosage [Time Frame: 7 months]

Brain 31phosphorus magnetic resonance spectroscopy [Time Frame: 7 months]

The Short Form (36) Health Survey [Time Frame: 7 months]

Secondary ID(s)

C14-53

Source(s) of Monetary Support

Please refer to primary and secondary sponsors