A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study, Followed by an Open-Label Extension, to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis

Minimum Age:

18 Years

Maximum Age:

80 Years

Can participants use Riluzole?

Yes

Key Inclusion Criteria:

- Diagnosis of sporadic or familial ALS

- Less than 3 years since ALS symptom onset

- Stable doses of approved ALS treatments (riluzole and/or edaravone) for at least 2 months prior to screening

- Participants must be able to swallow the study intervention

- Vital capacity >50% predicted at screening

- Women must have been surgically sterilized or be postmenopausal

- Men, and sex partner if a woman of childbearing potential, must use highly effective contraception

Key Exclusion Criteria:

- Any history of unstable or poorly controlled psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders

- Positive serum pregnancy test or currently lactating or breastfeeding

- History of malignancy within 5 years

- History of clinically significant neurologic disorders other than ALS