Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06513546
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-17
Brief Title:
A Study to Evaluate the Safety Efficacy and Pharmacodynamics of PLL001 in ALS Patients
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Multi-centre Phase 12 Randomised Double-blind Placebo Controlled Study With an Optional Open-label Extension to Evaluate the Safety Tolerability Efficacy and Pharmacodynamics of PLL001 for the Treatment of ALS
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
FIH Phase 12 multi-centre randomised double-blind placebo controlled study with an optional open-label dosing extension to assess the safety tolerability efficacy and Pharmacodynamics PD of single or multiple up to 48 weeks QD subcutaneous SC doses of PLL001 compared to placebo in subjects diagnosed with ALS
Detailed Description:
Part 1 Single Ascending Dose A total of 12 subjects will be randomised to 1 of 3 dose level cohorts 4 subjects per cohort In each cohort subjects will be randomised at a ratio of 31 to receive a single SC dose of PLL001 or placebo in a double-blind manner The safety data of placebo subjects will be pooled
Subjects will be admitted to the unit on the day prior to dosing Day -1 and will be administered in a double-blind manner PLL001 or placebo via SC injection in the morning on Day 1 Subjects will remain hospitalised for a minimum of 24 hours for safety evaluation and will be discharged in the morning on Day 2 Safety data will be collected daily up to Day 7 End of Study EOS
Subjects that have completed Part 1 of the study will be eligible for screening in Part 2
Part 2 Multiple Dose Expansion
Up to 141 subjects will be randomised to 1 of 3 treatments groups 2 dose level groups of PLL001 and 1 group of placebo at a ratio of 111 40 subjects per treatment group plus 21 patients for drop-out replacements
The first 21 subjects ie 7 subjects per treatment group will be dosed initially and will be randomised to 1 of the 3 treatment groups at a ratio of 111 These first 21 subjects will be monitored for the first 14 days of QD dosing Following review of the safety data by the SRC and in the absence of any clinically significant safety signals the remaining subjects will be enrolled
Patients under riluzole will be allowed to continue their riluzole treatment and will be stratified at randomisation in Part 2 to ensure comparable numbers between each treatment group Patients will also be stratified at randomisation in Part 2 based on TRICALS risk profile calculator score httpstricalsshinyappsiorisk-profile to ensure comparable patients ALS profiles between each treatment group
Subjects will self-administer or have carers administer PLL001 or placebo daily QD via 2 SC injections of 1 mL in a double-blind manner
Addmionsitrationbs will be on-site on the morning of Day 1 and will self-administer or have carers administer on all other dosing days stopping the day before the visit on Day 169 5 days Subjects will be discharged after a 4-hour observation period post-administration on Day 1
Efficacy safety and compliance data will be collected during site visits which will occur every 8 weeks on Day 57 113 and 169 5 days plus on Day 15 for all treated subjects
Safety and compliance data will be collected via tele contact weekly 3 days on the weeks subjects do not attend a site visit
Part 3 Open-label Extension
Subjects that consent to with the optional open-label treatment will commence open-label treatment at the visit on Day 169 5 days and continue daily self dosing or carer-administered dosing of PLL001 10 via 2 SC injections of 1 mL for an additional 24 weeks for a total of 48 weeks of treatment stopping the day before the visit on Day 337 5 days
Subjects will be admitted to the unit on the day prior to dosing Day -1 and will be administered in a double-blind manner PLL001 or placebo via SC injection in the morning on Day 1 Subjects will remain hospitalised for a minimum of 24 hours for safety evaluation and will be discharged in the morning on Day 2 Safety data will be collected daily up to Day 7 End of Study EOS
Subjects that have completed Part 1 of the study will be eligible for screening in Part 2
Part 2 Multiple Dose Expansion
Up to 141 subjects will be randomised to 1 of 3 treatments groups 2 dose level groups of PLL001 and 1 group of placebo at a ratio of 111 40 subjects per treatment group plus 21 patients for drop-out replacements
The first 21 subjects ie 7 subjects per treatment group will be dosed initially and will be randomised to 1 of the 3 treatment groups at a ratio of 111 These first 21 subjects will be monitored for the first 14 days of QD dosing Following review of the safety data by the SRC and in the absence of any clinically significant safety signals the remaining subjects will be enrolled
Patients under riluzole will be allowed to continue their riluzole treatment and will be stratified at randomisation in Part 2 to ensure comparable numbers between each treatment group Patients will also be stratified at randomisation in Part 2 based on TRICALS risk profile calculator score httpstricalsshinyappsiorisk-profile to ensure comparable patients ALS profiles between each treatment group
Subjects will self-administer or have carers administer PLL001 or placebo daily QD via 2 SC injections of 1 mL in a double-blind manner
Addmionsitrationbs will be on-site on the morning of Day 1 and will self-administer or have carers administer on all other dosing days stopping the day before the visit on Day 169 5 days Subjects will be discharged after a 4-hour observation period post-administration on Day 1
Efficacy safety and compliance data will be collected during site visits which will occur every 8 weeks on Day 57 113 and 169 5 days plus on Day 15 for all treated subjects
Safety and compliance data will be collected via tele contact weekly 3 days on the weeks subjects do not attend a site visit
Part 3 Open-label Extension
Subjects that consent to with the optional open-label treatment will commence open-label treatment at the visit on Day 169 5 days and continue daily self dosing or carer-administered dosing of PLL001 10 via 2 SC injections of 1 mL for an additional 24 weeks for a total of 48 weeks of treatment stopping the day before the visit on Day 337 5 days
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None