ACETYLCYSTEINE for Amyotrophic Lateral Sclerosis (ALS)

Inclusion criteria

• {"criterion_text":"- Patients diagnosed with ALS according to Gold Coast criteria.\n- Duration of disease ≤ 18 months\n- Males and females aged 18 to 75 years\n- ALSFRS-R total score ≥ 30 for all 12 categories\n- Forced vital capacity (FVC) ≥70%\n- Participant must be treated with Riluzole (50 mg twice daily) for ≥30 days prior to Day 1 (Visit 2) and is expected to remain on that dose until the final study visit.\n- Willingness and ability of the patient to comply with protocol requirements during the study\n- Sign written informed consent prior to any study-related procedures\n- Acceptance by the women of the use of contraceptive measures 30 days prior to taking the nutraceutical and investigational drug and throughout the study. Acceptable contraceptive method means: non-hormonal intrauterine device (IUD), barrier method (condom), bilateral tubal occlusion, vasectomized partner (provided the partner has been provided with a medical evaluation of the success of the surgery), or abstinence from sexual intercourse, in women of childbearing age (e.g., women who are not postmenopausal or have not been surgically sterilized)."}

Exclusion criteria

• {"criterion_text":"- Presence of other neurodegenerative diseases.\n- Genetically confirmed mitochondrial disease\n- Tracheostomized and/or gastrostomized patients\n- Participation in any clinical trial with investigational product in the within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing\n- Any clinically significant laboratory abnormalities that may directly affect compliance or safety\n- Allergy to NAC or any excipient of either the investigational drug or the nutraceutical EH301\n- Patients with short life expectancy as judged by the investigator\n- [Females only] Pregnancy or lactation for women of childbearing age (i.e., <2 years postmenopausal or not surgically sterile)\n- Patient is unwilling to use highly effective contraceptive methods during.\n- Significant cognitive impairment and/or dementia\n- Any psychiatric illness that could interfere with the study\n- Use of dietary supplements with high doses of vitamin B3 in the 30 days prior to inclusion in the study\n- Severe cardiac disease\n- Moderate to severe pulmonary disease, such as emphysema, stage III-IV COPD\n- Chronic uncontrolled asthma\n- Active cancer\n- Any metabolic, neoplastic, physically or mentally debilitating disease that could put the subject at risk or may interfere with the results of the study"}

Primary endpoints

• {"endpoint_text":"- Changes in the ALSFRS-R scale.","definition_or_measurement_approach":"Change measured using the ALS Functional Rating Scale - Revised (ALSFRS-R) score."}
• {"endpoint_text":"- Time to progression of patients suffering a 20% reduction in the ALSFRS-R scale 3.","definition_or_measurement_approach":"Time-to-event: time until a patient experiences a 20% reduction in the ALSFRS-R score."}

Secondary endpoints

• {"endpoint_text":"- Muscle strength measured by manual dynamometry.","definition_or_measurement_approach":"Muscle strength assessed using manual dynamometry."}
• {"endpoint_text":"- Survival.","definition_or_measurement_approach":"Overall survival (time to death)."}
• {"endpoint_text":"- Quality of life measured by the ALSAQ-40 scale.","definition_or_measurement_approach":"Health-related quality of life assessed using the ALSAQ-40 questionnaire."}
• {"endpoint_text":"- Respiratory function through the percentage of forced capacity (%FVC).","definition_or_measurement_approach":"Respiratory function measured as percent predicted forced vital capacity (%FVC)."}
• {"endpoint_text":"- Time elapsed until gastrostomy.","definition_or_measurement_approach":"Time-to-event: time from baseline to placement of gastrostomy."}
• {"endpoint_text":"- Combined assessment of function and survival (CAFS).","definition_or_measurement_approach":"Combined rank-based assessment incorporating function and survival (CAFS)."}
• {"endpoint_text":"- Nfl rate in CSF and changes in serum Nfl rate.","definition_or_measurement_approach":"Neurofilament light (NfL) measured in cerebrospinal fluid and serum; analysis of change/rate."}

Spain

Earliest CTIS Part Ii Submission Date

04-03-2025

Latest Decision Or Authorization Date

13-03-2026

Processing Time Days

374

Number Of Sites

1

Number Of Participants

90

Primary sponsor

Full Name

Asociacion Instituto Biogipuzkoa

Organisation Type

Patient organisation/association

Country Of Registered Address

Spain