Study to Evaluate DNL747 in Subjects With Amyotrophic Lateral Sclerosis

Phase 1

Terminated

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: DNL747; Drug: Placebo

• Registration Number: NCT03757351
• Lead Sponsor: Sanofi

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-11-27
• Study First Submitted QC: 2018-11-27
• Study First Posted: 2018-11-28
• Study Start: 2018-12-14
• Primary Completion: 2020-06-18
• Study Completion: 2020-06-18
• Status Verified: 2021-03
• Last Update Submitted: 2022-05-11
• Last Update Posted: 2022-05-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Open-Label Extension	DNL747	Conducted in the Netherlands only.
DNL747 First, Placebo Second	Placebo	-
Placebo First, DNL747 Second	DNL747	-
DNL747 First, Placebo Second	DNL747	-
Placebo First, DNL747 Second	Placebo	-

Primary Outcome Measures

Name	Time	Method
Number of Subjects with Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	Randomization - Day 86
Number of Subjects with clinically significant neurological examination abnormalities	Randomization - Day 86
Number of Subjects with laboratory test abnormalities	Randomization - Day 86

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic measure of maximum observed plasma concentration (Cmax) of DNL747	Randomization - Day 86
Pharmacokinetic measure of time to reach maximum observed plasma concentration (Tmax) of DNL747	Randomization - Day 86
Pharmacokinetic measure of area under the plasma drug concentration-time curve (AUC) of DNL747	Randomization - Day 86
Pharmacokinetic terminal disposition rate constant (λz) with the respective t1/2 of DNL747	Randomization - Day 86
Pharmacokinetic measure of CSF concentrations of DNL747	Randomization - Day 86
Pharmacodynamic measure of pS166 in PBMCs	Randomization - Day 86

Trial Locations

Locations (3)

CHDR; 🇳🇱 Leiden, South Holland, Netherlands

Bioclinica; 🇺🇸 Orlando, Florida, United States

PRA Health Sciences; 🇺🇸 Salt Lake City, Utah, United States