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

ConditionsAmyotrophic Lateral SclerosisALS
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorPLL TX AUSTRALIA PTY LTD

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Males and females ≥18 years of age at the time of informed consent.

Diagnosed within the previous 1 year with laboratory-supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria.

Must have familial or sporadic ALS.

First ALS symptoms occurred no more than two (2) years prior to screening visit ALS disease duration from diagnosis no longer than 12 months at the Screening visit.

Disqualifiers

Has dementia or significant neurological, psychiatric, systemic, or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results.

Pregnant or nursing females.

History of drug/chemical/substance/alcohol abuse within the past 2 years prior to Screening, including cannabinoid therapies.

Significant symptomatic, viral, bacterial (including upper respiratory infection), or fungal (non-cutaneous) infection within the past 2 weeks prior to study medication administration (at the discretion of the Investigator).

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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)

Treatment groups

153 Participants
are divided into 3 treatment groups
Group A: PLL001 dose 5xExperimental treatment 1 intervention
Group B: PLL001 dose 10xExperimental treatment 1 intervention
Group C: placeboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Part 1: safety

AEs, change from Baseline in other safety parameters (metric values) Haemotology: Haemoglobin g/L; Haematocrit L/L; Red Blood Cells X10\^12/L; White Blood Cells X10\^9/L; Platelets X10\^9/L; Neutrophils X10\^9/L; Eosinophils X10\^9/L; Lymphocytes X10\^9/L; Monocytes X10\^9/L; Basophils X10\^9/L; Mean corpuscular volume fL Biochemistry: Sodium mmol/L; Potassium mmol/L; Phosphate mmol/L; Calcium mmol/L; Urea mmol/L; Uric Acid mmol/L; Creatinine umol/L; Glucose mmol/L; Protein g/L; Albumin g/L; Bilirubin umol/L; ALP U/L; AST U/L; ALT U/L; GGT U/L; LDH U/L Coagulation: Prothrombin time sec; APTT sec; International Normalised Ratio; Fibrinogen g/L Urinalysis: RBC x10\^6/L; WBC x10\^6/L

Time frame
7 days
2

Part 2: efficacy of PLL001 compared to placebo after 8, 16, and 24 weeks of once daily (QD) treatment

Change from Baseline in ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) scores. Minimum score of 0 and a maximum score of 48. Higher scores indicate better functional status and retained abilities. Specifically, a score of 48 represents the best functional status, while a score of 0 indicates the worst functional status

Time frame
24 weeks
3

Part 2: survival after 8, 16, and 24 weeks of QD treatment for PLL001 compared to placebo

Survival

Time frame
24 weeks

Secondary outcomes

1

Part 2: safety

AEs, change from Baseline in other safety parameters (metric values) Haemotology: Haemoglobin g/L; Haematocrit L/L; Red Blood Cells X10\^12/L; White Blood Cells X10\^9/L; Platelets X10\^9/L; Neutrophils X10\^9/L; Eosinophils X10\^9/L; Lymphocytes X10\^9/L; Monocytes X10\^9/L; Basophils X10\^9/L; Mean corpuscular volume fL Biochemistry: Sodium mmol/L; Potassium mmol/L; Phosphate mmol/L; Calcium mmol/L; Urea mmol/L; Uric Acid mmol/L; Creatinine umol/L; Glucose mmol/L; Protein g/L; Albumin g/L; Bilirubin umol/L; ALP U/L; AST U/L; ALT U/L; GGT U/L; LDH U/L Coagulation: Prothrombin time sec; APTT sec; International Normalised Ratio; Fibrinogen g/L Urinalysis: RBC x10\^6/L; WBC x10\^6/L

Time frame
24 weeks
2

Part 2: efficacy over 24 weeks

Progression-free survival (PFS) * Quality of life based on ALSAQ-40 score (Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 questions) * Respiratory function regression on Slow Vital Capacity score (SVC)

Time frame
24 weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

PLL TX AUSTRALIA PTY LTD

Lead sponsor

Source ClinicalTrials.gov

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