A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants with Amyotrophic Lateral Sclerosis

Condition studied

Amyotrophic Lateral Sclerosis

Investigational medicinal product(s)

Eligibility

Primary endpoint

Incidence and severity of AEs, TEAEs and SAEs, Clinical laboratory tests (serum chemistry and hematology; urinalysis), ECGs, Vital signs (blood pressure, heart rate, respiratory rate, body temperature), Physical and neurological exams.

Endpoint detail

CSF levels of LTX-002, Single-dose plasma PK parameters for the first and last of the three doses, including Cmax, Tmax, AUC0-t, AUC0-∞, Tlag if applicable, Vd/F, λz, T1/2, and CL/F, Where data are available, AUC0-∞ AUC0 t, and Cmax will be tested for dose proportionality using a power model approach.