DEXTRAN SULFATE LOW MOLECULAR WEIGHT for Amyotrophic Lateral Sclerosis

Inclusion criteria

• {"criterion_text":"- Age 18 to 80 years inclusive at the time of signing the informed consent.\n- Must be in a stable health condition as determined by medical evaluation including medical history, physical examination, laboratory tests, and cardiac monitoring at screening.\n- Male or female diagnosed with ALS according to the World Federation of Neurology revised Gold Coast criteria\n- Onset of ALS symptoms ≤ 24 months at screening visit.\n- Disease progression rate ∆FRS ≥ 0.4 at screening.\n- FVC ≥ 60 % (Forced Vital Capacity) of predicted valued for gender, height, and age at screening.\n- Must have started treatment of Riluzole (100 mg/day) at least 2 weeks prior to screening and willing to pause Riluzole 48 hours prior to the baseline visit (Day 1).\n- ALSFRS-R score of at least 28 points at screening."}

Exclusion criteria

• {"criterion_text":"- A participant with dementia, other neurodegenerative diseases (e.g. Parkinson disease, multiple sclerosis) or any significant uncontrolled neurological, psychiatric, neoplastic, systemic, or organic disease (e.g. significant renal, hepatic or pulmonary disorder with on-going treatment not attributed to ALS) that, in the opinion of the investigator or medical monitor, could interfere with the conduct of the trial or affect its results.\n- A participant who is pregnant or nursing.\n- A participant with a history (within 12 months before screening) of current alcohol, drug, or medication abuse, as assessed by the investigator. Alcohol abuse is defined as consuming more than 14 units per week\n- A participant with known allergy or intolerability to dextran sulfate, riluzole, and other ingredients of the IMPs.\n- Participants receiving active treatment with drugs warfarin and direct oral anticoagulants (DOACs) 14 days prior to screening visit.\n- A participant having clinically significant abnormal coagulation parameters: prothrombin complex-international normalized ratio (INR) > 1.5, fibrinogen <1.5 g/L, von Willebrand factor deficit and APTT > 41 seconds at screening"}

Primary endpoints

• {"endpoint_text":"- The change in Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) score from baseline at week 24","definition_or_measurement_approach":"Change from baseline in ALSFRS-R score measured at week 24."}

Secondary endpoints

• {"endpoint_text":"- Change in Serum Nfl from baseline to week 24\n- The change in the concentration of Extracellular domain of p75(P75ECD) in urine from baseline at week 24\n- The change in serum N-acetyl-aspartate (NAA) concentration from baseline at week 24\n- The change in Modified Norris Scale scores from baseline at week 24\n- The change in forced vital capacity (FVC) scores from baseline at week 24\n- Incidence and severity of adverse events (AEs) and serious AEs (SAEs)\n- Clinically meaningful changes in laboratory parameters, vital signs, and electrocardiogram (ECG) results\n- The change from baseline in Edinburgh Cognitive and Behavioral ALS screen (ECAS) scores at week 24\n- The change in mean scores as measured by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) and EuroQol-5-Dimension-5-Levels questionnaire (EQ-5D-5L) questionnaire from baseline at week 24\n- PK parameters of ILB® in approximately 10 participants\n- The change from baseline of serum Nfl, NAA and P75ECD in urine at week 48\n- The mean change from baseline in ALSFRS-R score, Modified Norris Scale scores, FVC scores, ECAS score, ALSAQ-40 and EQ-5D-5L at week 48\n- OS is assessed by time from baseline to death from any cause or tracheostomy, whichever comes first\n- Measure biomarker activity changes of e.g. Interleukin-6 (IL-6), HGF and glutamate from baseline to week 24 and to week 48\n- MRI changes from baseline at week 24 and week 48 in selected 50 participants with ALS\n- Healthcare utilization data, including hospital stays and emergency room (ER) visits, will be collected. HRQOL will be assessed with ALSAQ-40 and EQ-5D-5L. Economic analysis will assess changes in healthcare usage using both within-trial and model-based approaches, with an emphasis on long-term projections. Study-required procedures are excluded","definition_or_measurement_approach":"Endpoints mainly defined as change from baseline to specified timepoints (week 24, week 48). PK parameters measured for ~10 participants. Overall survival (OS) measured as time from baseline to death or tracheostomy. Biomarkers and MRI assessed as change from baseline at specified weeks. Safety endpoints assessed by incidence and severity of AEs/SAEs and by laboratory, vital signs and ECG changes."}

Norway

Latest Decision Or Authorization Date

03-02-2026

Number Of Sites

13

Number Of Participants

116

Primary sponsor

Full Name

Oslo University Hospital HF

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Norway