A Phase 2, Randomized, Double-Blind, Double-Dummy Study Evaluating the Efficacy, Safety and Biomarkers Effect of ILB® versus Riluzole in participants with Amyotrophic Lateral Sclerosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Oslo University Hospital HF

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

16 Feb 2026 → ongoing

Decision date (initial)

2025-04-25

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the efficacy of ILB® compared to Riluzole in reducing disease progression in participants with amyotrophic lateral sclerosis (ALS)

Secondary objectives 15

1. To evaluate the efficacy of ILB® compared to Riluzole on reducing levels of axonal damage biomarkers, measured by Serum Nfl
2. To evaluate the efficacy of ILB® compared to Riluzole on other axonal damage biomarkers in participants with ALS
3. To evaluate the efficacy of ILB® compared to Riluzole on motor limb and bulbar function
4. To evaluate the efficacy of ILB® compared to Riluzole on lung function
5. To evaluate the safety and tolerability of ILB® compared to Riluzole in participants with ALS
6. To evaluate the efficacy of ILB® compared to Riluzole on cognitive function in participants with ALS
7. To evaluate the efficacy of ILB® compared to Riluzole on health-related quality of life patient reported outcome measures
8. To evaluate pharmacokinetics (PK) of ILB® compared to Riluzole in participants with ALS
9. To evaluate the long-term efficacy of ILB® on axonal damage biomarkers
10. To evaluate the long-term efficacy of ILB® on disease progression, motoric limb and bulbar function, FVC, cognition and Health-Related Quality of Life (HRQOL)
11. To evaluate overall survival (OS) of ILB® compared to Riluzole
12. To evaluate long-term safety and tolerability of ILB®
13. To study the ILB® effects on exploratory biomarker activity in participants with ALS
14. To study the effects of ILB® using advanced Magnetic Resonance Imaging (MRI) modalities in participants with ALS
15. To study the ILB® effects on long-term health- related quality of life and health-economic aspects

Conditions and MedDRA coding

Amyotrophic Lateral Sclerosis

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Age 18 to 80 years inclusive at the time of signing the informed consent.
2. Must be in a stable health condition as determined by medical evaluation including medical history, physical examination, laboratory tests, and cardiac monitoring at screening.
3. Male or female diagnosed with ALS according to the World Federation of Neurology revised Gold Coast criteria
4. Onset of ALS symptoms ≤ 24 months at screening visit.
5. Disease progression rate ∆FRS ≥ 0.4 at screening.
6. FVC ≥ 60 % (Forced Vital Capacity) of predicted valued for gender, height, and age at screening.
7. 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).
8. ALSFRS-R score of at least 28 points at screening.

Exclusion criteria 6

1. 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.
2. A participant who is pregnant or nursing.
3. 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
4. A participant with known allergy or intolerability to dextran sulfate, riluzole, and other ingredients of the IMPs.
5. Participants receiving active treatment with drugs warfarin and direct oral anticoagulants (DOACs) 14 days prior to screening visit.
6. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The change in Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) score from baseline at week 24

Secondary endpoints 16

1. Change in Serum Nfl from baseline to week 24
2. The change in the concentration of Extracellular domain of p75(P75ECD) in urine from baseline at week 24
3. The change in serum N-acetyl-aspartate (NAA) concentration from baseline at week 24
4. The change in Modified Norris Scale scores from baseline at week 24
5. The change in forced vital capacity (FVC) scores from baseline at week 24
6. Incidence and severity of adverse events (AEs) and serious AEs (SAEs)
7. Clinically meaningful changes in laboratory parameters, vital signs, and electrocardiogram (ECG) results
8. The change from baseline in Edinburgh Cognitive and Behavioral ALS screen (ECAS) scores at week 24
9. 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
10. PK parameters of ILB® in approximately 10 participants
11. The change from baseline of serum Nfl, NAA and P75ECD in urine at week 48
12. 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
13. OS is assessed by time from baseline to death from any cause or tracheostomy, whichever comes first
14. Measure biomarker activity changes of e.g. Interleukin-6 (IL-6), HGF and glutamate from baseline to week 24 and to week 48
15. MRI changes from baseline at week 24 and week 48 in selected 50 participants with ALS
16. 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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 1

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oslo University Hospital HF

Sponsor organisation

Oslo University Hospital HF

Address

Taarnbygget, Kirkeveien 166 Kirkeveien 166

City

Oslo

Postcode

0450

Country

Norway

Scientific contact point

Organisation

Oslo University Hospital HF

Contact name

Angelina Maniaol

Public contact point

Organisation

Oslo University Hospital HF

Contact name

Angelina Maniaol

Locations

1 EU/EEA country · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 16 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications