What is PL001?
PLL001 is an investigational medicine being studies as a potential treatment for ALS/ In this clinical trial, PLL001 is given as an injection and compared with a placebo to see whether it is safe, well tolerated, and may help with ALS. Some participants may also have the option to continue receiving PLL001 for a longer period so researchers can better understand its effect over time.
The PL001 Trial:
The trial will be carried out in stages. The first stage will have a small number of participants receive a single injection of either PLL001 or a placebo, with participants staying in hospital over night for researchers to closely monitor safety. In the second stage, a larger group of participants receive daily injections of either PLL001 (at one of two dose levels) or placebo for several months, mostly at home, with regular clinic visits and weekly check-ins to monitor safety, treatment use, and any changes in symptoms; participants may continue taking Riluzole if they are already prescribed it. In the final optional stage, participants who wish to continue can receive PLL001 for a longer period, allowing researchers to collect information on longer-term safety and effects.
Eligibility Criteria:
- Male or female, aged 18 years or older.
- Diagnosed with ALS (laboratory-supported probable, clinically probable, or definite) within the past 12 months.
- Familial or sporadic ALS.
- First ALS symptoms occurred no more than 2 years before screening.
- If on riluzole, edaravone, or other approved ALS treatments, dose must be stable for at least 4 weeks before Day 1.
- Documented ALSFRS-R score progression of >0.3 points/month from disease onset, confirmed by ≥1 point progression in the 12 weeks before randomisation.
- ALSFRS-R score of at least 26 at screening, with a minimum score of 3 on item #3 and at least 2 on all 12 individual components.
- Seated slow vital capacity (SVC) ≥50% of predicted value for age, gender, and height.
- Able and willing to comply with study requirements and complete the study.
- Provides written informed consent.
| NSW | Liverpool Hospital | Cecilia Cappelen-Smith, MD 61 2 8738 3646 [email protected] |
| NSW | Neuroscience Research Australia | Matthew Kiernan [email protected] |
| QLD | Sunshine Coast University Hospital | Antony Winkel, MD [email protected] |
| QLD | Wesley Research Institute | Robert Henderson, MD [email protected] |
| QLD | Gold Coast University Hospital | Arman Sabet, Professor, MD [email protected] |
| SA | Flinders Medical Centre | David Schulz, MD 61 8 8204 4187 [email protected] |
| VIC | Alfred Health | Edrich Rodrigues, MD [email protected] |
| VIC | Calvary Health Care, Bethlehem | Susan Mathers, MD, Professor 61 (0) 3 9596 2853 [email protected] |
| VIC | Monash Medical Centre, Neuroscience Research | Susan Mathers, MD, professor 61 (0) 3 9596 2853 [email protected] |
| WA | The Perron Institute for Neurological and Translational Science | Dev Nathani, MD [email protected] |
Want to know more?
For more information Visit Study Details | NCT06513546 | A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PLL001 in ALS Patients | ClinicalTrials.gov