A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PLL001 in ALS Patients

Sponsor
PLL TX AUSTRALIA PTY LTD
Study ID
NCT06513546
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • PLL001 or placebo daily subcutaneous injections — DRUG
    PLL001 consists of the combination of 4 drug substances (DS) each being a linear poly-lysine flexible backbone with an average length of 70 L-lysines linked with 10% of conjugated side chains all being small chain fatty acids (SCFAs) (acetate, butyrate, lactate and propionate). The remaining 90% of the lysine residues are present as bromide salt. PLL001 DP is presented as 20 mL vials containing 16 mL of a sterilised solution for SC injection with the following formula (5× concentration)

Study Details

FIH, Phase 1/2, 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.

Key Dates

Start date
Apr 8, 2026
Status verified
Mar 2026
Primary completion
Dec 15, 2026
Completion
Jun 15, 2027

Study Design

Enrollment
153 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: PLL001 dose 5x
    PLL001 lowest dose (Poly-l-Lysine conjugates with acetate, butirate, lactate, propionate) daily subcutaneous injections
  • Experimental: PLL001 dose 10x
    PLL001 highest dose (Poly-l-Lysine conjugates with acetate, butirate, lactate, propionate) daily subcutaneous injections
  • Placebo Comparator: placebo
    Saline daily subcutaneous injections

Primary Outcome Measure

Part 1: safety [ Time Frame: 7 days ]

Central Contacts

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