A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 24 April 2023
• Main ID: NCT03945279
• Date of registration: 08/05/2019
• Prospective Registration: Yes
• Primary sponsor: Biogen
• Public title: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
• Scientific 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
• Date of first enrolment: May 30, 2019
• Target sample size: 49
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03945279
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Medical Director
• Affiliation: Biogen

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: BIIB100

Drug: Placebo

Primary Outcome(s)

Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Screening (Day -28 ) up to Day 15]

Secondary Outcome(s)

Apparent Clearance (CL/F) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Maximum Observed Concentration (Cmax) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Time to Reach Cmax (Tmax) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Terminal Elimination Half-life (t1/2) of BIIB100 [Time Frame: Day 1 (pre-dose) up to Day 3]

Secondary ID(s)

261AS101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors