OXYTAHANE - Oxygen therapy: Acute Treatment for paroxysmal dystonic or plegic attacks in alternating hemiplegia of childhood

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

7 Aug 2024 → 3 Mar 2026

Decision date (initial)

2024-03-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

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

Secondary objectives 1

1. 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 possible other modalities of the usual management of crises over 5 weeks 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 crises that begin less than 2 hours after the end of the previous crisis. 9. the duration of the first crisis in each of the 5-week periods 10. the perception of patients and caregivers at the end of the 5 weeks of treatment, assessed with recorded interviews

Conditions and MedDRA coding

Alternating Hemiplegia of Childhood

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 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

1. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary outcome will be the proportion of motor attacks stopped 30 minutes after the beginning of motor symptoms over 5 weeks.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Flamand-Roze

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Flamand-Roze

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications