A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis

• Status: Recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL343 in participants with amyotrophic lateral sclerosis (ALS)

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Denali Therapeutics Inc.

Criteria

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