NCT04066244

Study of Safety and Proof of the Mechanism of BLZ945 in ALS Patients

An Open-label, Adaptive Design Study in Patients With Amyotrophic Lateral Sclerosis (ALS) to Characterize Safety, Tolerability and Brain Microglia Response, as Measured by TSPO Binding, Following Multiple Doses of BLZ945 Using Positron Emission Tomography (PET) With the Radioligand [11C]-PBR28

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

It was an open label study to evaluate safety, tolerability and brain microglia response in participants with Amyotrophic Lateral Sclerosis (ALS) following multiple doses of BLZ945.

Detailed description

The purpose of the study was to identify a dose (or doses) of BLZ945, that measurably decrease(s) 18 KDa Translocator Protein (TSPO) binding in the brain of participants with ALS. The study also aimed to evaluate the safety and tolerability of BLZ945 in participants with ALS at different doses and dosing regimens, and safety related effects on extracellular matrix (ECM) accumulation. This was an exploratory, adaptive , open label study of approximately 16 participants in cohorts of 4 participants per cohort at increasing doses of BLZ945 with the last dose determined after the completion of cohorts 1 to 3. Each cohort received treatment for 4 days and continued with a 32 day follow up period with an end of study visit at day 36. After completion of the 4 initial cohorts a fifth cohort was initiated with two parallel arms receiving BLZ945 for up to 12 weeks at two different treatment regimens, either once weekly or 4 days of treatment followed by 10 days off drug.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	BLZ945	Investigational drug capsules taken orally or via enteral infusion

Timeline

• Start date: 2019-12-30
• Primary completion: 2024-02-01
• Completion: 2024-02-01
• First posted: 2019-08-26
• Last updated: 2025-10-16
• Results posted: 2025-03-19

Locations

4 sites across 3 countries: United States, Finland, Sweden

Regulatory

• FDA-regulated drug study