A Multicenter, Randomized, Placebo-Controlled, Double-Blind, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL747 in Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL747 in subjects with Amyotrophic Lateral Sclerosis in a cross-over design

Study Status:

Not recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

Drug

Intervention Name:

DNL747, Placebo

Placebo:

Yes

Phase:

Phase 1

Study Chair(s)/Principal Investigator(s):

Clinical Sciences & Operations, Sanofi

Clinicaltrials.gov ID:

NCT03757351

Neals Affiliated?

No

Coordinating Center Contact Information

Full Study Summary:

This is a Phase 1b randomized, placebo-controlled, double-blind, crossover study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL747 in subjects with Amyotrophic Lateral Sclerosis (ALS)

Study Sponsor:

Sanofi

Estimated Enrollment:

15

Estimated Study Start Date:

12 / 14 / 2018

Estimated Study Completion Date:

06 / 18 / 2020

Posting Last Modified Date:

05 / 12 / 2022

Date Study Added to neals.org:

11 / 28 / 2018

Minimum Age:

21 Years

Maximum Age:

80 Years

Can participants use Riluzole?

Yes

Key Inclusion Criteria (Double-Blind Part):

- Women of non-childbearing potential and men, aged 21-80 years

- Willingness and ability to complete all aspects of the study; participant should be capable of completing assessments either alone or with help of a caregiver

- Diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria

- Less than 3 years since symptom onset

- Forced vital capacity (FVC) >50% predicted measured within 30 days of screening

- If subject is taking approved ALS treatments (riluzole and/or edaravone), doses must be stable for ≥2 months prior to screening and subject is expected to stay on a stable regimen throughout the study

Key Exclusion Criteria (Double-Blind Part):

- History of a clinically significant non-ALS neurologic disorder (other than frontal temporal lobe dementia), including, but not limited to, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies, AD, Parkinson's disease, Lewy body dementia, vascular dementia, Huntington's disease, epilepsy, stroke, multiple sclerosis, brain tumor, or brain infection or abscess

- Unstable or poorly controlled comorbid disease process of any organ system currently requiring active treatment or likely to require treatment adjustment during the study

Key Inclusion Criteria (Open-Label Extension):

- Successful completion of both periods of the the double-blind, crossover part of the study

- Continued diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria

Key Exclusion Criteria (Open-Label Extension):

- Presence of laboratory abnormalities, physical examination findings, or AEs determined to be clinically significant by the investigator from the double-blind part of the study that have not resolved by the final follow-up visit as part of the double-blind study period

- New diagnosis of clinically significant neurological disorder (other than frontal temporal lobe dementia)

Bioclinica

Orlando, Florida 32806
United States

PRA Health Sciences

Salt Lake City, Utah 84124
United States

CHDR

Leiden, South Holland 2333
Netherlands