Phase 1/2Not yet recruiting
A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS) (HORIZON)
A Randomized, Double-Blind, Placebo-Controlled MAD Study With Open-Label Extension to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of BIIB147 Administered Intrathecally to Adult Participants With ALS
- Sponsor
- Biogen (Industry)
- Enrolment
- 69 planned
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- BIIB147; Placebo
- Ages
- adults, older adults
- Registry
- NCT07855211
From the registry
In this study, researchers will learn more about the effects of BIIB147, in participants with amyotrophic lateral sclerosis, also known as ALS. ALS is a rare disease that damages the motor neurons in the brain and spinal cord that help control the movement of muscles in the body. It is a progressive disease, which means that it gets worse with time. Over time, this causes increasing problems with speaking, swallowing, moving the arms and legs, and breathing. In the case of ALS, this eventually leads to death from patients not being able to breathe. This is a "first-in-human" study. This means that this study drug will be given to people for the very first time in this study. These studies are important because they help researchers learn about the safety of the study drug, how the body processes it, and what dose might be appropriate before testing it in larger groups. The main goal of the study is to learn about the safety of BIIB147 in adult participants with ALS. The main question researchers want to answer is: * How many participants have adverse events and serious adverse…
Primary outcomes
- MAD, OLE, Integrated MAD and OLE Periods: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)