Alternating Hemiplegia of ChildhoodPhase 2HEMIHEPNCT02408354
Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood
A Phase 2 study testing Triheptanoin in people with Alternating Hemiplegia of Childhood, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
Trial summary
The purpose of this project is to study the efficacy of triheptanoin oil in patients with Alternating Hemiplegia of Childhood (AHC) due to ATP1A3 gene mutation. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Alternating Hemiplegia of Childhood
- What is being tested?
- Triheptanoin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteFrance
- Study dates
- 25 Mar 2015to 5 Apr 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
The purpose of this project is to study the efficacy of triheptanoin oil in patients with Alternating Hemiplegia of Childhood (AHC) due to ATP1A3 gene mutation.
Read the detailed description
The clinical spectrum of Alternating Hemiplegia of Childhood (AHC) is wide and characterized by the association of permanent and paroxysmal (palsy, dystonia, ocular, epileptic, dysautonomic events) neurological events, with onset in childhood. Most of AHC patients carry mutations in the ATP1A3 gene. This gene encodes the Na+/K+ ATPase witch is a transmembrane ion pump generating chemical and electrical gradient of sodium and potassium across the plasma membrane. Those paroxystic events in AHC patients with mutations in the ATP1A3 gene could be associated with a glucidic/energetic metabolism or intracerebral excitability disorder.
Triheptanoin is a triglyceride, whose derivatives pass the blood - brain barrier and enhance the Krebs cycle functions. Triheptanoin could therefore allow energy supply to the brain, which is essential for the functioning of the Na+/ K+ ATPase that consumes a significant amount of energy in the brain.
The investigators goal is to do a pilot study to test the effectiveness on paroxystic manifestations and the safety of triheptanoin in a small group of patients with Alternating Hemiplegia of Childhood secondary to ATP1A3 mutations.
- Conditions
- Alternating Hemiplegia of Childhood
- Being tested
- Triheptanoin, Placebo
- Medical subject headings
- Alternating hemiplegia of childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TriheptanoinDrug
Also called UX007, glycerol triheptanoate
Triheptanoin is a triglyceride composed of three heptanoate (C7 fatty acid) esters. Triheptanoin is manufactured by chemical synthesis from glycerol and heptanoic acid. Triheptanoin is a liquid, intended for oral (PO) administration.
Participants will be given approximately 1g/kg of Triheptanoin divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose.
PlaceboDrug
Placebo is a oily liquid, intended for oral (PO) administration. Participants will be given approximately 1g/kg of Placebo divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose.
| Study group | Role | What they receive |
|---|---|---|
| Triheptanoin | Active comparator | Triheptanoin/ Placebo Randomized to receive active Triheptanoin first for 12 weeks. At cross-over, participants will receive placebo for 12 weeks.
Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between triheptanoine and placebo phases. Receives: Drug: Triheptanoin |
| Placebo | Placebo comparator | Placebo / Triheptanoin Randomized to receive active Placebo first for 12 weeks. At cross-over, participants will receive Triheptanoin for 12 weeks.
Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between placebo and triheptanoin phases. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of neurologic paroxystic events report in patient diary
visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
Measured: 7 months
Secondary outcomes (6)
Composite score allying the number of neurological paroxystic events, their duration and severity.
visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
Measured: 7 months
Clinical Global Impression Scales - Improvement
visit 2 at week 12, visit 4 at week 28
Measured: 7 months
The Short Form (36) Health Survey
visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
Measured: 7 months
Brain 31phosphorus magnetic resonance spectroscopy
Ratio of Inorganic Phosphate (Pi) over Phosphocreatine during visual stimulation visit 2 at week 12, visit 4 at week 28
Measured: 7 months
Clinical Safety as measured by questionnaire
visit 2 at week 12, visit 4 at week 28
Measured: 7 months
Biological Safety as measured by acylcarnitine profile, organic acid dosage
visit 2 at week 12, visit 4 at week 28
Measured: 7 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2015 (estimated)
- Study start25 Mar 2015
- Primary completion5 Apr 2017
- Study completion5 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alternating Hemiplegia of ChildhoodStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Emmanuel Flamand-Roze, MD, PhD, Principal investigator, INSERM UMRS 975, 47 bd de l'hôpital - 75651 Paris Cedex 13
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02408354
- Sponsor's protocol ID
- C14-53
- Acronym
- HEMIHEP
- Status last verified
- Feb 2025
- First submitted
- 24 Mar 2015
- Last updated
- 10 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
Publications
Hainque E, Caillet S, Leroy S, Flamand-Roze C, Adanyeguh I, Charbonnier-Beaupel F, Retail M, Le Toullec B, Atencio M, Rivaud-Pechoux S, Brochard V, Habarou F, Ottolenghi C, Cormier F, Meneret A, Ruiz M, Doulazmi M, Roubergue A, Corvol JC, Vidailhet M, Mochel F, Roze E. A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood. Orphanet J Rare Dis. 2017 Oct 2;12(1):160. doi: 10.1186/s13023-017-0713-2.
PubMed 28969699
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02408354
- Acronym / sponsor ID
- HEMIHEP / C14-53
- Conditions
- Alternating Hemiplegia of Childhood
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Study start
- 25 Mar 2015 (actual)
- Primary completion
- 5 Apr 2017 (actual)
- Study completion
- 5 Apr 2017 (actual)
- First posted
- 3 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this project is to study the efficacy of triheptanoin oil in patients with Alternating Hemiplegia of Childhood (AHC) due to ATP1A3 gene mutation.
Detailed description
The clinical spectrum of Alternating Hemiplegia of Childhood (AHC) is wide and characterized by the association of permanent and paroxysmal (palsy, dystonia, ocular, epileptic, dysautonomic events) neurological events, with onset in childhood. Most of AHC patients carry mutations in the ATP1A3 gene. This gene encodes the Na+/K+ ATPase witch is a transmembrane ion pump generating chemical and electrical gradient of sodium and potassium across the plasma membrane. Those paroxystic events in AHC patients with mutations in the ATP1A3 gene could be associated with a glucidic/energetic metabolism or intracerebral excitability disorder.
Triheptanoin is a triglyceride, whose derivatives pass the blood - brain barrier and enhance the Krebs cycle functions. Triheptanoin could therefore allow energy supply to the brain, which is essential for the functioning of the Na+/ K+ ATPase that consumes a significant amount of energy in the brain.
The investigators goal is to do a pilot study to test the effectiveness on paroxystic manifestations and the safety of triheptanoin in a small group of patients with Alternating Hemiplegia of Childhood secondary to ATP1A3 mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Triheptanoin | Active comparator | Drug: TriheptanoinTriheptanoin/ Placebo Randomized to receive active Triheptanoin first for 12 weeks. At cross-over, participants will receive placebo for 12 weeks. Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between triheptanoine and placebo phases. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo / Triheptanoin Randomized to receive active Placebo first for 12 weeks. At cross-over, participants will receive Triheptanoin for 12 weeks. Each drug will be dispensed successively. A one-month wash out period is planned for 4 weeks between placebo and triheptanoin phases. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TriheptanoinAlso: UX007, glycerol triheptanoateTriheptanoin is a triglyceride composed of three heptanoate (C7 fatty acid) esters. Triheptanoin is manufactured by chemical synthesis from glycerol and heptanoic acid. Triheptanoin is a liquid, intended for oral (PO) administration. Participants will be given approximately 1g/kg of Triheptanoin divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose. | Drug | Triheptanoin |
| PlaceboPlacebo is a oily liquid, intended for oral (PO) administration. Participants will be given approximately 1g/kg of Placebo divided at least in three doses (at 8 am, 12 noon, and 8 pm). A one-day titration period will be used, using 0.5 g/kg increments before arriving at the full dose. | Drug | Placebo |
Primary outcome
Number of neurologic paroxystic events report in patient diary
Time frame: 7 months
visit 1 at day 0, visit 2 at week 12, visit 3 at week 16, visit 4 at week 28
6 secondary outcomes
Composite score allying the number of neurological paroxystic events, their duration and severity.
Time frame: 7 months
Clinical Global Impression Scales - Improvement
Time frame: 7 months
The Short Form (36) Health Survey
Time frame: 7 months
Brain 31phosphorus magnetic resonance spectroscopy
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
- Results publication: PubMed 28969699Hainque E, Caillet S, Leroy S, Flamand-Roze C, Adanyeguh I, Charbonnier-Beaupel F, Retail M, Le Toullec B, Atencio M, Rivaud-Pechoux S, Brochard V, Habarou F, Ottolenghi C, Cormier F, Meneret A, Ruiz M, Doulazmi M, Roubergue A, Corvol JC, Vidailhet M, Mochel F, Roze E. A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood. Orphanet J Rare Dis. 2017 Oct 2;12(1):160. doi: 10.1186/s13023-017-0713-2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emmanuel Flamand-Roze, MD, PhD principal investigator
INSERM UMRS 975, 47 bd de l'hôpital - 75651 Paris Cedex 13
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Triheptanoin in people with Alternating Hemiplegia of Childhood, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of neurologic paroxystic events report in patient diary (time frame: 7 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02408354, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025.