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

Denali Therapeutics

Status and phase

Completed

Phase 1

Conditions

Amyotrophic Lateral Sclerosis

Treatments

Drug: Placebo

Drug: DNL343

Study type

Interventional

Funder types

Industry

Identifiers

NCT05006352

DNLI-F-0003

2021-001766-37 (EudraCT Number)

Details and patient eligibility

About

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)

Full description

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Enrollment

29 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

Diagnosis of sporadic or familial ALS
≤ 4 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, be postmenopausal, or for participants of childbearing potential, must not be pregnant, and both the participant and the male partner must use highly effective contraception
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