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CompletedNCT03945279Updated Apr 18, 2023

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

A Phase 1 interventional study of BIIB100 and Placebo in Amyotrophic Lateral Sclerosis, sponsored by Biogen. Completed at 9 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-04-18.

Sponsored by Biogen · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Jun 2021, 5 years 3 months ago, and no results have been posted to the registry.
Phase
Phase 1
Study type
Interventional
Enrollment
49
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis
03

In context

Motor Neuron Disease

717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.

This study's enrollment of 49 is above the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.

Browse Motor Neuron Disease studies →

Lead sponsor

Biogen is the lead sponsor of 494 studies on the registry; 22 are open to participants now.

Of its 98 completed or terminated interventional studies of FDA-regulated products, 49 (50%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
49 participants (actual)

Study arms

  • Experimental
    Cohort 1: BIIB100 Dose 1

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Experimental
    Cohort 2: BIIB100 Dose 2

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Experimental
    Cohort 3: BIIB100 Dose 3

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Experimental
    Cohort 4: BIIB100 Dose 4

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Experimental
    Cohort 5: BIIB100 Dose 5

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Experimental
    Cohort 6: BIIB100 Dose 6

    Participants will receive single oral dose of BIIB100 on Day 1.

    Drug: BIIB100

  • Placebo comparator
    Cohort 1-6: Matching Placebo

    Participants will receive single oral dose of matching placebo on Day 1.

    Drug: Placebo

Interventions

  • DrugBIIB100

    Administered as specified in the treatment arm.

  • DrugPlacebo

    Administered as specified in the treatment arm.

06

What researchers measure

Primary outcomes

  1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.

    Time frame: Screening (Day -28 ) up to Day 15

Secondary outcomes

  1. Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  2. Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  3. Maximum Observed Concentration (Cmax) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  4. Time to Reach Cmax (Tmax) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  5. Terminal Elimination Half-life (t1/2) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  6. Apparent Clearance (CL/F) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

  7. Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100

    BIIB100 will be measured in the plasma.

    Time frame: Day 1 (pre-dose) up to Day 3

07

Study locations

9 sites
  • Barrow Neurological Institute
    Phoenix, Arizona 85013, United States
  • University of California San Diego Medical Center
    San Diego, California 92121, United States
  • Mayo Clinic Hospital
    Jacksonville, Florida 32224, United States
  • University of South Florida
    Tampa, Florida 33612, United States
  • Johns Hopkins University, Dept of Neurology
    Baltimore, Maryland 21205, United States
  • Research Site
    Boston, Massachusetts 21219, United States
  • Research Site
    Saint Louis, Missouri 63110, United States
  • Research Site
    Lincoln, Nebraska 68506, United States
  • Alliance for Multispecialty Research NOCCR/VRG
    Knoxville, Tennessee 37920, United States
08

References and documents

Individual participant data

Plan to share: Yes — In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 18, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03945279
Lead sponsor
Biogen
Responsible party
Sponsor
First posted
May 10, 2019
Start date
May 30, 2019
Primary completion
Jun 21, 2021
Completion
Jun 21, 2021
Last update
Apr 18, 2023

Study contacts

Medical Director
study director · Biogen

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.

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