Clinical trial NCT03945279: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
Official title: A Phase 1, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2019-05-10
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.


Eligibility criteria: Key Inclusion Criteria:

* Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
* Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (\>=) 30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.
* Participants taking concomitant edaravone at study entry must be on a stable dose for \>= 60 days prior to the first dose of study treatment (Day 1).
* Adequate respiratory function as indicated by slow vital capacity (SVC) \>= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).

Key Exclusion Criteria:

* Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.
* Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (\<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.
* Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.
* Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Screening (Day -28 ) up to Day 15
Secondary outcomes:
measure: Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Maximum Observed Concentration (Cmax) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Time to Reach Cmax (Tmax) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Terminal Elimination Half-life (t1/2) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Apparent Clearance (CL/F) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
measure: Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100
timeFrame: Day 1 (pre-dose) up to Day 3
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: San Diego
country: United States
facility: University of California San Diego Medical Center
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Hospital
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins University, Dept of Neurology
state: Maryland
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Lincoln
country: United States
facility: Research Site
state: Nebraska
city: Knoxville
country: United States
facility: Alliance for Multispecialty Research NOCCR/VRG
state: Tennessee
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: