Sachi Bioworks Inc.
This is a first-in-human study of NI112, an investigational medicine given as an injection under the skin, in adults with amyotrophic lateral sclerosis (ALS). The purpose of the study is to find out how safe NI112 is, how well it is tolerated, and how the body handles it after a single dose. Up to 40 adults with ALS will take part at up to three study centers in the United States. Participants will be assigned by chance to receive either a single dose of NI112 or a single dose of placebo (sterile salt water). The study tests up to seven dose levels, one after another, beginning with the lowest dose. A Safety Review Board reviews the safety information from each group of participants before the next, higher dose is given to a new group. Each participant stays in the hospital for 24 hours after the injection for safety monitoring and blood sampling, and returns for follow-up visits on Day 8 and Day 28. The study also measures the amount of NI112 in blood and in spinal fluid.
This is a Phase 1, first-in-human, multi-center, randomized, double-blind, placebo-controlled, sequential single ascending dose-escalation study evaluating the safety, tolerability and pharmacokinetics of NI112 administered by subcutaneous injection to adults with amyotrophic lateral sclerosis. Up to seven sequential dose cohorts are planned at 2.5, 5, 10, 20, 40, 80 and 120 mg. Cohorts at 20 mg and below enroll 4 subjects randomized 3 active to 1 placebo; cohorts above 20 mg enroll 8 subjects randomized 6 active to 2 placebo. The 5 mg cohort may be omitted only under pre-specified pharmacokin…
Inclusion Criteria: * • Able to understand the purpose and risks of the study and to provide signed, dated informed consent and authorization for use of protected health information. A legally authorized representative may consent if the subject is legally unable to do so. * Aged ≥ 18 years at the time of informed consent. No upper age limit. * Weakness attributable to ALS and a diagnosis of possible, laboratory-supported probable, probable or definite ALS per the revised El Escorial criteria (Airlie House, 1998). * Forced vital capacity ≥ 50% of predicted, adjusted for sex, age, heigh…
Antisense Nanoligomer administered at 40 mg/mL, as a single subcutaneous injection on Day 1. Doses above 40 mg are divided across two injection sites with no more than 1.5 mL per site.
Sterile normal saline (0.9% sodium chloride), administered as a single subcutaneous injection on Day 1.