Alternating Hemiplegia of ChildhoodPhase 2OXYTAHANENCT06248645
Oxygen as an Acute Treatment in Alternating Hemiplegia of Childhood
A Phase 2 study testing Oxygen in people with Alternating Hemiplegia of Childhood, run by Assistance Publique - Hôpitaux de Paris at 2 sites in France.
Trial summary
Alternating hemiplegia of childhood (AHC) is a rare early-onset neurodevelopmental encephalopathy frequently caused by mutations in the ATP1A3 gene. It is typically characterized by a variable degree of intellectual disability, motor dysfunction and various paroxysmal events (dystonic and plegic attacks). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Alternating Hemiplegia of Childhood
- What is being tested?
- Oxygen
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 2 sitesFrance
- Study dates
- 16 Sep 2024to 31 Mar 2026
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age > 1 year old
- Patients able to administrate high-flow oxygen through a non rebreathing facial mask, either alone or with assistance
- Patients with a diagnosis of AHC caused by ATP1A3 mutations presenting at least one dystonic or plegic attack per week lasting more than 30 minutes
- Absence of long-term treatment or long-term treatment stable over the last month and during the duration of the trial
- Patients able to give written informed consent
- Affiliation to social insurance
Who cannot join
- Inability for the patients and/or caregivers to follow the protocol (inability to read or understand the instructions, no access to smartphone or computer to fill the informatics patient's form)
- Patients with severe or uncontrolled obstructive or restrictive chronic respiratory disease (asthma, COPD, obesity, neuromuscular disease), acute systemic aggression (acute coronary syndrome, stroke, sepsis, head trauma) or congenital cardiopathy with a risk of hypercapnia following oxygen administration
- Women that are pregnant (a negative pregnancy test is required for inclusion) or breath feeding
- Patients involved in an other clinical trial
Trial locations and contacts
2 sites in 1 country.
About this study
Official title: Oxygen Therapy as an Acute Treatment for Dystonic and/or Plegic Attacks in Alternating Hemiplegia of Childhood
Alternating hemiplegia of childhood (AHC) is a rare early-onset neurodevelopmental encephalopathy frequently caused by mutations in the ATP1A3 gene. It is typically characterized by a variable degree of intellectual disability, motor dysfunction and various paroxysmal events (dystonic and plegic attacks). Dystonic and plegic attacks are very disabling and current treatments are disappointing with limited efficacy and poor tolerability. The investigators recently reported the efficacy of high-flow oxygen administration (100% O2 at a flow rate of 12 L/min) as an acute treatment for the dystonic attacks in a 25-year-old patient suffering from AHC. The aim of the study is to assess the effect of high-flow oxygen administration (against placebo) as an acute treatment of dystonic and plegic attacks. The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
Read the detailed description
This is a multicenter, randomized, placebo-controlled, double-blind crossover study, with two successive periods of 5 weeks.
Participants will be randomized in a 1:1 ratio to receive one of the two treatment sequences: oxygen followed by placebo or placebo followed by oxygen. The two treatment periods will be separated by a wash-out period of 10 days +/- 4 days.
The placebo will consist in the administration of medical air. The same procedure will be used for both treatments (oxygen and medical air): administration with a flow rate of 12 L/min through a non-rebreathing facial mask using indiscernible bottles.
The treatment will be administered as soon as possible after the beginning of the attack and for 15 minutes. If the attack hasn't stopped 30 minutes after the beginning of motor symptoms, the patients will be allowed to receive their usual acute pharmacological treatment if they (or their caregiver) judge it necessary.
The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks. The secondary outcomes will be:
- the median duration of dystonic and plegic attacks over 5 weeks
- the evaluation of the quality of life for patients (PELHS-QOL-2) and caregivers (adapted from the PELHS-QOL-2) at the end of the 5 weeks of treatment
- the frequency of motor attacks over 5 weeks
- the consumption of sedative treatments over 5 weeks (number of doses used)
- treatment tolerance, in particular mouth and nasal dryness, cutaneous irritation, cough, nasal congestion, nausea, other unexpected side effects
- the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
- the number and proportion of treated attacks over 5 weeks
- the number and proportion of attacks starting less than 2 hours after the end of the previous attack
- the duration of the first attack for each of the two periods of treatment
- the perception of patients and caregivers at the end of the 5 weeks of treatment, assessed with recorded interviews
- Conditions
- Alternating Hemiplegia of Childhood
- Being tested
- Oxygen, 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.
OxygenDrug
High-flow oxygen (12 L/min) administered with a non-rebreathing facial mask, started as soon as possible after the beginning of the attack and for 15 minutes.
PlaceboDrug
Also called médical Air
High-flow medical air (12 L/min) administered with a non-rebreathing facial mask, started as soon as possible after the beginning of the attack and for 15 minutes.
| Study group | Role | What they receive |
|---|---|---|
| Oxygen followed by placebo | Other | Treatment of each motor attacks by oxygen (gaz) during 15 minutes in the first 5 week-period and placebo (medical air) during 15 minutes in the second 5 week-period. Receives: Drug: Oxygen; Drug: Placebo |
| Placebo followed by oxygen | Other | Treatment of each motor attacks by placebo (medical air) during 15 minutes, in the first 5 week-period and oxygen (gaz) during 15 minutes in the second 5 week-period. Receives: Drug: Oxygen; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
Measured: 5 weeks
Secondary outcomes (9)
the median duration of dystonic and plegic attacks over 5 weeks
Measured: 5 weeks
the evaluation of the quality of life for patients (PELHS-QOL-2 Pediatric Epilepsy Learning Healthcare System Quality of Liferated, from 2 to 8) and caregivers (adapted from the PELHS-QOL-2) at the end of the 5 weeks of treatment
Measured: 5 weeks
the frequency of motor attacks over 5 weeks
Measured: 5 weeks
the consumption of sedative treatments over 5 weeks (number of doses used)
Measured: 5 weeks
treatment tolerance, in particular mouth and nasal dryness, cutaneous irritation, cough, nasal congestion, nausea, other unexpected side effects
Measured: 5 weeks
the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
Measured: 5 weeks
the number and proportion of attacks starting less than 2 hours after the end of the previous attack
Measured: 5 weeks
the duration of the first attack for each of the two periods of treatment
Measured: 5 weeks
the perception of patients and caregivers at the end of the 5 weeks of treatment, assessed with recorded interviews
Measured: 5 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2024
- Study start16 Sep 2024
- Primary completion3 Mar 2026
- Study completion31 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alternating Hemiplegia of ChildhoodStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06248645
- Sponsor's protocol ID
- APHP230673
- Acronym
- OXYTAHANE
- Status last verified
- Apr 2026
- First submitted
- 31 Jan 2024
- Last updated
- 13 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oxygen Therapy as an Acute Treatment for Dystonic and/or Plegic Attacks in Alternating Hemiplegia of Childhood
- ClinicalTrials.gov ID
- NCT06248645
- Acronym / sponsor ID
- OXYTAHANE / APHP230673
- Conditions
- Alternating Hemiplegia of Childhood
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 16 Sep 2024 (actual)
- Primary completion
- 3 Mar 2026 (actual)
- Study completion
- 31 Mar 2026 (actual)
- First posted
- 8 Feb 2024 (actual)
- 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.
Alternating hemiplegia of childhood (AHC) is a rare early-onset neurodevelopmental encephalopathy frequently caused by mutations in the ATP1A3 gene. It is typically characterized by a variable degree of intellectual disability, motor dysfunction and various paroxysmal events (dystonic and plegic attacks). Dystonic and plegic attacks are very disabling and current treatments are disappointing with limited efficacy and poor tolerability. The investigators recently reported the efficacy of high-flow oxygen administration (100% O2 at a flow rate of 12 L/min) as an acute treatment for the dystonic attacks in a 25-year-old patient suffering from AHC. The aim of the study is to assess the effect of high-flow oxygen administration (against placebo) as an acute treatment of dystonic and plegic attacks. The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
Detailed description
This is a multicenter, randomized, placebo-controlled, double-blind crossover study, with two successive periods of 5 weeks.
Participants will be randomized in a 1:1 ratio to receive one of the two treatment sequences: oxygen followed by placebo or placebo followed by oxygen. The two treatment periods will be separated by a wash-out period of 10 days +/- 4 days.
The placebo will consist in the administration of medical air. The same procedure will be used for both treatments (oxygen and medical air): administration with a flow rate of 12 L/min through a non-rebreathing facial mask using indiscernible bottles.
The treatment will be administered as soon as possible after the beginning of the attack and for 15 minutes. If the attack hasn't stopped 30 minutes after the beginning of motor symptoms, the patients will be allowed to receive their usual acute pharmacological treatment if they (or their caregiver) judge it necessary.
The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks. The secondary outcomes will be:
- the median duration of dystonic and plegic attacks over 5 weeks
- the evaluation of the quality of life for patients (PELHS-QOL-2) and caregivers (adapted from the PELHS-QOL-2) at the end of the 5 weeks of treatment
- the frequency of motor attacks over 5 weeks
- the consumption of sedative treatments over 5 weeks (number of doses used)
- treatment tolerance, in particular mouth and nasal dryness, cutaneous irritation, cough, nasal congestion, nausea, other unexpected side effects
- the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
- the number and proportion of treated attacks over 5 weeks
- the number and proportion of attacks starting less than 2 hours after the end of the previous attack
- the duration of the first attack for each of the two periods of treatment
- the perception of patients and caregivers at the end of the 5 weeks of treatment, assessed with recorded interviews
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 |
|---|---|---|
| Oxygen followed by placebo | Other | Drug: Oxygen, Drug: PlaceboTreatment of each motor attacks by oxygen (gaz) during 15 minutes in the first 5 week-period and placebo (medical air) during 15 minutes in the second 5 week-period. |
| Placebo followed by oxygen | Other | Drug: Oxygen, Drug: PlaceboTreatment of each motor attacks by placebo (medical air) during 15 minutes, in the first 5 week-period and oxygen (gaz) during 15 minutes in the second 5 week-period. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Age > 1 year old
- Patients able to administrate high-flow oxygen through a non rebreathing facial mask, either alone or with assistance
- Patients with a diagnosis of AHC caused by ATP1A3 mutations presenting at least one dystonic or plegic attack per week lasting more than 30 minutes
- Absence of long-term treatment or long-term treatment stable over the last month and during the duration of the trial
- Patients able to give written informed consent
Key exclusion criteria (4 listed)
- Inability for the patients and/or caregivers to follow the protocol (inability to read or understand the instructions, no access to smartphone or computer to fill the informatics patient's form)
- Patients with severe or uncontrolled obstructive or restrictive chronic respiratory disease (asthma, COPD, obesity, neuromuscular disease), acute systemic aggression (acute coronary syndrome, stroke, sepsis, head trauma) or congenital cardiopathy with a risk of hypercapnia following oxygen administration
- Women that are pregnant (a negative pregnancy test is required for inclusion) or breath feeding
- Patients involved in an other clinical trial
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 |
|---|---|---|
| OxygenHigh-flow oxygen (12 L/min) administered with a non-rebreathing facial mask, started as soon as possible after the beginning of the attack and for 15 minutes. | Drug | Oxygen followed by placebo, Placebo followed by oxygen |
| PlaceboAlso: médical AirHigh-flow medical air (12 L/min) administered with a non-rebreathing facial mask, started as soon as possible after the beginning of the attack and for 15 minutes. | Drug | Oxygen followed by placebo, Placebo followed by oxygen |
Primary outcome
Proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
Time frame: 5 weeks
The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.
9 secondary outcomes
the median duration of dystonic and plegic attacks over 5 weeks
Time frame: 5 weeks
the evaluation of the quality of life for patients (PELHS-QOL-2 Pediatric Epilepsy Learning Healthcare System Quality of Liferated, from 2 to 8) and caregivers (adapted from the PELHS-QOL-2) at the end of the 5 weeks of treatment
Time frame: 5 weeks
the frequency of motor attacks over 5 weeks
Time frame: 5 weeks
the consumption of sedative treatments over 5 weeks (number of doses used)
Time frame: 5 weeks
treatment tolerance, in particular mouth and nasal dryness, cutaneous irritation, cough, nasal congestion, nausea, other unexpected side effects
Time frame: 5 weeks
the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
Time frame: 5 weeks
the number and proportion of attacks starting less than 2 hours after the end of the previous attack
Time frame: 5 weeks
the duration of the first attack for each of the two periods of treatment
Time frame: 5 weeks
the perception of patients and caregivers at the end of the 5 weeks of treatment, assessed with recorded interviews
Time frame: 5 weeks
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 13 Apr 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Oxygen in people with Alternating Hemiplegia of Childhood, run by Assistance Publique - Hôpitaux de Paris at 2 sites 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 4 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 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. 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?
Proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks. (time frame: 5 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.