WALKALS : A pilot phase II study to evaluate the effect of salbutamol on walking capacity in ambulatory ALS patients.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2023-05-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To test the efficacy of salbutamol on walking capacity in ALS patients.

Secondary objectives 1

1. The secondary objective of this study is to measure the target engagement of salbutamol on the neuromuscular junction (NMJ) at EMG (decrement of repetitive nerve stimulation in three nerves/muscle couples), as well as safety and tolerability.

Conditions and MedDRA coding

Amyotrophic Lateral Sclerosis (ALS)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. - Subjects who meet the revised El Escorial criteria for probable or definite sporadic ALS - Adult patients between 18 and 75 years of age - Patients who are ambulatory and able to perform the 6MWT and quantitative muscle testing at screening (ALSFRS-R-walking = 3) - Patients able and willing to travel to the site, and, in the investigator’s opinion, who are likely to attend visits for at least 6 months - Patients who signed written informed consent - Stable dose of riluzole for a minimum of 14 day4 weeks prior to baseline or has not taken it for 14 days4 weeks prior to baseline - Affiliation to French social security system excluding AME - For child-bearing aged women, efficient contraception - Forced vital capacity (fVC) in a sitting position > 70 %

Exclusion criteria 1

1. - Patients with significant spasticity of the lower limbs interfering with walking capacity (Ashworth scale score  2) - Patients with fronto-temporal dementia associated with ALS - Patients presenting respiratory insufficiency causing dyspnea during walking - Patients taking drugs that could interfere with NMJ function (anticholinesterase …) or muscle function (steroids, statins…)- Patients taking any forbidden drugs (see list in annex) - Hypersensitivity to salbutamol or to excipients of the drug and placebo - Known contraindication for the studied drug such as ischemic cardiomyopathy or risk of ischemic cardiomyopathy - Pregnancy or women seeking a pregnancy during the study or breastfeeding - Patients under curatorship or guardianship - Participation in another interventional trial up to 3 months before inclusion - Patients having any relevant concomitant disease considered at risk of interfering with study procedures in the opinion of the investigator - Patients not affiliated to the French social security system

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Two primary endpoints will be used: change from baseline of the 6 minutes walking test distance (6MWT) at M3 (2mg TID) and at M6 (4mg TID) with type I error controlled through Bonferroni correction.

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

Dr Giorgia QUERIN

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Giorgia QUERIN

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 2 files

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

Application history

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