None listed
Conditions
Brief summary
Ambroxol is a simple cough medicine that is predicted to slow ALS disease progression. This study is the open label extension (OLE) of the parent study, which aims to investigate if ambroxol in high doses is effective in treating ALS. This OLE study will be carried out across 5 research sites in Australia (2 NSW, 1 VIC, 1 SA and 1 TAS), where ALS patients whole have successfully completed the randomised phase of the parent study, will be asked to participate in the OLE phase. Participation will be over a 52-week period, where they will come in for a an OLE baseline, followed by 48-week treatment, and 4-week end of study safety follow-up period. All participants will active drug that they will take three times a day, Throughout the study their disease progression will be assessed using tests, questionnaires, and blood biomarkers.
Interventions
Participants who have successfully completed the randomised phase of the parent AMBALS study, will be invited to participate in the Open Label Extension (OLE). The OLE will involve participants taking 420mg capsules of ambroxol, orally, three times a day (total dose 1260mg per day).
Participants will complete an OLE baseline visit, for consenting and OLE eligibility assessments. They will then start their OLE dosing. This will then be followed 48 weeks of follow-up (4x in clinic follow-up visits
Participants who have successfully completed the randomised phase of the parent AMBALS study, will be invited to participate in the Open Label Extension (OLE). The OLE will involve participants taking 420mg capsules of ambroxol, orally, three times a day (total dose 1260mg per day). Participants will complete an OLE baseline visit, for consenting and OLE eligibility assessments. They will then start their OLE dosing. This will then be followed 48 weeks of follow-up (4x in clinic follow-up visits - one every 12 weeks). These 48 weeks will be the drug administration period, meaning that the total duration of drug administration is 48 weeks. Following this drug administration and follow-up period, there will be an End of Study safety-follow up visit that will occur 4 weeks after the final follow-up visit (52 weeks from OLE baseline). Participants will return IP bottles at each of the 4 in-clinic follow-up visits, to monitor IP compliance.
Sponsors
The Florey Institute of Neuroscience and Mental Health (part of the University of Melbourne)
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
Refer to ACTRN12622001380785 for the inclusion criteria of the randomized placebo-controlled Treatment Period of the AMBALS trial. 1) Participants must have completed the randomized placebo-controlled Treatment Period of the AMBALS trial without compliance issues 2) Able to understand and give written informed consent to participant in the open-label extension.
Exclusion criteria
Refer to ACTRN12622001380785 for the exclusion criteria of the randomized placebo-controlled Treatment Period of the AMBALS trial. 1) Lack of treatment compliance during the randomized placebo-controlled Treatment Period. 2) Positive pregnancy test at the Week 24 visit of the AMBALS study randomised phase, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension. 3) Based on the investigator’s judgment, participants who may have difficulty complying with the protocol and/or any study procedures. 4)Participants with any clinically significant medical conditions based on the Investigator’s judgment, which may interfere with continued participation.
Outcome results
None listed