Using oxygen to treat acute attacks in children with alternating hemiplegia
Oxygen Therapy As an Acute Treatment for Dystonic And/or Plegic Attacks in Alternating Hemiplegia of Childhood
This study is testing whether high-flow oxygen can help stop sudden attacks in children with Alternating Hemiplegia of Childhood.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 1 Year and up |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 2 sites (Lyon and 1 other locations) |
| Trial ID | NCT06248645 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of high-flow oxygen as an acute treatment for dystonic and plegic attacks in children with Alternating Hemiplegia of Childhood (AHC), a rare neurodevelopmental disorder. The study is designed as a multicenter, randomized, placebo-controlled, double-blind crossover trial, where participants will receive either oxygen or a placebo (medical air) in two successive treatment periods. The primary outcome is the proportion of motor attacks that stop within 30 minutes of symptom onset over a 5-week period. Secondary outcomes include the duration of attacks, quality of life assessments, frequency of attacks, and treatment tolerance.
Who should consider this trial
Good fit: Ideal candidates are children over 1 year old diagnosed with AHC due to ATP1A3 mutations who experience at least one dystonic or plegic attack per week.
Not a fit: Patients with severe respiratory diseases or those unable to follow the study protocol may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of acute attacks in children with AHC, enhancing their quality of life.
How similar studies have performed: While this approach is novel for AHC, previous studies have shown promising results with oxygen therapy in other conditions, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Age \> 1 year old 2. Patients able to administrate high-flow oxygen through a non rebreathing facial mask, either alone or with assistance 3. 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 4. Absence of long-term treatment or long-term treatment stable over the last month and during the duration of the trial 5. Patients able to give written informed consent 6. Affiliation to social insurance Exclusion Criteria: 1. 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) 2. 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 3. Women that are pregnant (a negative pregnancy test is required for inclusion) or breath feeding 4. Patients involved in an other clinical trial
Where this trial is running
Lyon and 1 other locations
- Hcl Lyon — Lyon, France (Not_yet_recruiting)
- Hôpital Pitié Salpêtrière — Paris, France (Recruiting)
Study contacts
- Study coordinator: Emmanuel Flamand-Roze, MD, PhD
- Email: flamand.roze.75012@gmail.com
- Phone: 0142162748
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.