BIIB147 safety and how it behaves in adults with ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a first-in-human, phase 1/2 interventional study evaluating BIIB147 in adults with ALS. The trial includes a multiple-ascending-dose period to assess safety and tolerability, followed by an open-label extension to examine long-term safety and pharmacokinetics in serum and CSF. Secondary aims look at how BIIB147 affects neurodegeneration markers, disease progression, and survival, with comparisons between earlier and later initiation. Participants must meet genetic and functional criteria and may continue stable riluzole therapy. The study is conducted at two sites in the United States and is led by Biogen.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

MAD Period

* Participants must have a diagnosis of ALS consistent with the Gold Coast criteria.
* No pathogenic variants, likely pathogenic variants, or variants of uncertain significance in the superoxide dismutase 1 (SOD1) or fused in sarcoma (FUS) genes (confirmed by central genetic testing).
* Greater than or equal to (≥) 50% of predicted value for slow vital capacity (SVC) as adjusted for sex, age, and height (from the sitting position).
* If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit end of study (EOS)/early termination (ET), unless the Investigator determines that it should be discontinued for medical reasons, in which case it may not be restarted during the study.

OLE

* Participants must have completed Study 303AS101 MAD period; all pre-dose assessments for the end of MAD treatment period visit (Day 113 for Cohorts A, B, C, and Day 169 for Cohort D) must be completed prior to dosing in OLE.

Key Exclusion Criteria:

MAD Period

* History or positive test result at screening for human immunodeficiency virus (HIV). The requirement for testing at screening may be omitted if it is not permitted by local regulations.
* History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NfL, in the opinion of the Investigator.
* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
* Requires permanent ventilation (using \> 22 hours per day \[h/day\] invasive or non-invasive ventilation)
* History of a deep venous thrombosis or pulmonary embolism ≤2 years of screening, or since the date of ALS diagnosis, whichever is longer.
* Treatment with an approved ALS disease-modifying therapy other than riluzole or edaravone, or another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, before completion of Screening.
* Current enrollment or a plan to enroll in any interventional clinical study. 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.

OLE

* Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the ability of the participant to undergo study procedures.
* Clinically significant abnormalities (based on most recent assessments in MAD period) in hematology, blood chemistry parameters, or 12-lead electrocardiogram (ECG) prior to first OLE visit that would make the participant unsuitable for inclusion as assessed by the Investigator.

Note: Other protocol-defined Inclusion/Exclusion criteria do apply.

Where it's running

• Washington University School of Medicine_St. Louis
  St Louis, Missouri, United States Recruiting
• Austin Neuromuscular Center_Austin
  Austin, Texas, United States Recruiting

Study contacts

• Study coordinator: US Biogen Clinical Trial Center
• Email: clinicaltrials@biogen.com
• Phone: 866-633-4636

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.