Ignite Creation Date:
2024-05-06 @ 8:04 PM
Last Modification Date:
2024-10-26 @ 3:20 PM
Study NCT ID:
NCT06248645
Status:
RECRUITING
Last Update Posted:
2024-04-12
First Post:
2024-01-31
Brief Title:
Oxygen As an Acute Treatment in Alternating Hemiplegia of Childhood
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
Official Title:
Oxygen Therapy As an Acute Treatment for Dystonic Andor Plegic Attacks in Alternating Hemiplegia of Childhood
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
OXYTAHANE
Brief 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 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 Lmin 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 11 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 Lmin 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 hasnt 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
1 the median duration of dystonic and plegic attacks over 5 weeks
2 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
3 the frequency of motor attacks over 5 weeks
4 the consumption of sedative treatments over 5 weeks number of doses used
5 treatment tolerance in particular mouth and nasal dryness cutaneous irritation cough nasal congestion nausea other unexpected side effects
6 the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
7 the number and proportion of treated attacks over 5 weeks
8 the number and proportion of attacks starting less than 2 hours after the end of the previous attack
9 the duration of the first attack for each of the two periods of treatment
10 the perception of patients and caregivers at the end of the 5 weeks of treatment assessed with recorded interviews
Participants will be randomized in a 11 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 Lmin 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 hasnt 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
1 the median duration of dystonic and plegic attacks over 5 weeks
2 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
3 the frequency of motor attacks over 5 weeks
4 the consumption of sedative treatments over 5 weeks number of doses used
5 treatment tolerance in particular mouth and nasal dryness cutaneous irritation cough nasal congestion nausea other unexpected side effects
6 the proportion of dystonic and plegic attacks respectively stopped 30 minutes after the beginning of motor symptoms over 5 weeks
7 the number and proportion of treated attacks over 5 weeks
8 the number and proportion of attacks starting less than 2 hours after the end of the previous attack
9 the duration of the first attack for each of the two periods of treatment
10 the perception of patients and caregivers at the end of the 5 weeks of treatment assessed with recorded interviews
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None