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Amyotrophic Lateral SclerosisPhase 1NCT03945279

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

A Phase 1 study testing BIIB100 in people with Amyotrophic Lateral Sclerosis, run by Biogen at 9 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
BIIB100
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
49 participants
Where
9 sitesUnited States
Study dates
30 May 2019to 21 Jun 2021
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).

Who cannot join

  • 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.

Trial locations and contacts

9 sites in 1 country.

9 sites in 1 country

United States9 sites

  • Johns Hopkins University, Dept of Neurology

    Baltimore, Maryland, 21205

  • Research Site

    Boston, Massachusetts, 21219

  • Mayo Clinic Hospital

    Jacksonville, Florida, 32224

  • Alliance for Multispecialty Research NOCCR/VRG

    Knoxville, Tennessee, 37920

  • Research Site

    Lincoln, Nebraska, 68506

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013

  • University of California San Diego Medical Center

    San Diego, California, 92121

  • Research Site

    St Louis, Missouri, 63110

  • University of South Florida

    Tampa, Florida, 33612

Other trials in United States

About this study

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

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.

Being tested
BIIB100, Placebo
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BIIB100Drug

    Administered as specified in the treatment arm.

  • PlaceboDrug

    Administered as specified in the treatment arm.

Study groups in NCT03945279
Study groupRoleWhat they receive
Cohort 1: BIIB100 Dose 1ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 2: BIIB100 Dose 2ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 3: BIIB100 Dose 3ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 4: BIIB100 Dose 4ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 5: BIIB100 Dose 5ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 6: BIIB100 Dose 6ExperimentalParticipants will receive single oral dose of BIIB100 on Day 1.
Receives: Drug: BIIB100
Cohort 1-6: Matching PlaceboPlacebo comparatorParticipants will receive single oral dose of matching placebo on Day 1.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Screening (Day -28 ) up to Day 15

Secondary outcomes (7)

  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.

    Measured: 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.

    Measured: Day 1 (pre-dose) up to Day 3

  3. Maximum Observed Concentration (Cmax) of BIIB100

    BIIB100 will be measured in the plasma.

    Measured: Day 1 (pre-dose) up to Day 3

  4. Time to Reach Cmax (Tmax) of BIIB100

    BIIB100 will be measured in the plasma.

    Measured: Day 1 (pre-dose) up to Day 3

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

    BIIB100 will be measured in the plasma.

    Measured: Day 1 (pre-dose) up to Day 3

  6. Apparent Clearance (CL/F) of BIIB100

    BIIB100 will be measured in the plasma.

    Measured: 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.

    Measured: Day 1 (pre-dose) up to Day 3

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 May 2019
  2. Study start30 May 2019
  3. Primary completion21 Jun 2021
  4. Study completion21 Jun 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
49 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03945279
Sponsor's protocol ID
261AS101
Status last verified
Apr 2023
First submitted
8 May 2019
Last updated
18 Apr 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BIIB100 in people with Amyotrophic Lateral Sclerosis, run by Biogen at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Screening (Day -28 ) up to Day 15). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03945279.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2023.

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