A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS)

A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS) - Phoenix

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON54389

Enrollment

Registered

2021-07-06

Start date

2021-12-03

Completion date

Unknown

Last updated

2024-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Disease of nerve cells that control muscles Neurodegenerative syndrome

Interventions

Participants will be randomly assigned in a3:2 ratio to oral (or feeding tube) AMX0035 treatment (a fixed-dose combination of sodium phenylbutyrate [PB] and taurursodiol) or matching placebo. For th

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. Male or female, at least 18 years of age; 2. Diagnosis of ALS (clinically definite or clinically probable), made by a physician who is experienced with management of ALS, as defined by the World Federation of Neurology revised El Escorial criteria; 3. Time since onset of first symptom of ALS should be

Exclusion criteria

Exclusion criteria: 1. Presence of tracheostomy or PAV; defined as >22 hours daily of mechanical ventilation for more than 1 week (7 days); 2. Slow Vital Capacity (SVC) less than 55%; 3. History of known allergy to phenylbutyrate or bile salts; 4. Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 5 times the upper limit of the normal (obtained within 12 weeks from first dose); 5.5. Renal insufficiency as defined by estimated glomerular filtration rate (eGFR)

Design outcomes

Primary

Measure	Time frame
1. To assess the impact of AMX0035 treatment compared to placebo on disease progression over 48 weeks based on change form baseline of ALSFRS-R and survival.	—

Secondary

Measure	Time frame
1. To assess the impact of AMX0035 compared to placebo on Slow Vital Capacity (SVC) in adult patients with ALS over 48 weeks; 2. To assess patient quality of life (QOL; using the 40-item ALS assessment questionnaire [ALSAQ-40] patient-reported outcome [PRO]) during treatment with AMX0035 compared to placebo in adult patients with ALS over 48 weeks; 3. To assess AMX0035 compared to placebo on the time to any decline in King's and MiToS stages as derived from ALSFRS-R data over 48 weeks; 4. To assess the impact of AMX0035 compared to placebo on ventilation free survival (defined as death, tracheostomy for respiratory distress or permanent non-invasive ventilation [>22 hours per day for 7 consecutive days]) in adult patients with ALS over 48 weeks; 5. To assess AMX0035 compared to placebo on patient health status (using the EQ-5D descriptive system and the EQ visual analogue scale [EQ VAS] PRO during treatment with AMX0035 over 48 weeks. Finally, a secondary objective of the trial is to assess the long-term overall survival of all-cause mortality beyond the 48 week Treatment Period of adult patients with ALS treated with AMX0035 or placebo until time of death or End of Study.	—

Measure

Time frame

1. To assess the impact of AMX0035 compared to placebo on Slow Vital Capacity (SVC) in adult patients with ALS over 48 weeks; 2. To assess patient quality of life (QOL; using the 40-item ALS assessment questionnaire [ALSAQ-40] patient-reported outcome [PRO]) during treatment with AMX0035 compared to placebo in adult patients with ALS over 48 weeks; 3. To assess AMX0035 compared to placebo on the time to any decline in King's and MiToS stages as derived from ALSFRS-R data over 48 weeks; 4. To assess the impact of AMX0035 compared to placebo on ventilation free survival (defined as death, tracheostomy for respiratory distress or permanent non-invasive ventilation [>22 hours per day for 7 consecutive days]) in adult patients with ALS over 48 weeks; 5. To assess AMX0035 compared to placebo on patient health status (using the EQ-5D descriptive system and the EQ visual analogue scale [EQ VAS] PRO during treatment with AMX0035 over 48 weeks. Finally, a secondary objective of the trial is to assess the long-term overall survival of all-cause mortality beyond the 48 week Treatment Period of adult patients with ALS treated with AMX0035 or placebo until time of death or End of Study.

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)