RecruitingPhase 1 / Phase 2interventionalRules rewrite

Phase 1 / Phase 2: testing BIIB147 for Amyotrophic Lateral Sclerosis

In this study, researchers will learn more about the effects of BIIB147, in participants with amyotrophic lateral sclerosis, also known as ALS. ALS is a rare disease that damages the motor neurons in the brain and spinal cord that help control the movement of muscles in the body.

Official registry: https://clinicaltrials.gov/study/NCT07855211 · Biogen · enrollment listed as 69

What this phase means

Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are BIIB147, or Placebo. The registry lists allocation as randomized. In this study, researchers will learn more about the effects of BIIB147, in participants with amyotrophic lateral sclerosis, also known as ALS.

They plan to enroll about 69 people at 2 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

A Randomized, Double-Blind, Placebo-Controlled MAD Study With Open-Label Extension to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of BIIB147 Administered Intrathecally to Adult Participants With ALS

Interventions: BIIB147, Placebo

Primary outcome (registry wording): MAD, OLE, Integrated MAD and OLE Periods: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Who it is for (from inclusion criteria)

Ages 18 Years to any
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)
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

Who the registry excludes

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
Current enrollment or a plan to enroll in any treatment 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
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

Public study contact (from the registry)

US Biogen Clinical Trial Center

866-633-4636

clinicaltrials@biogen.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

2 listed

Washington University School of Medicine_St. Louis — St Louis, Missouri (recruiting)
Austin Neuromuscular Center_Austin — Austin, Texas (recruiting)

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FAQ about this NCT record

Is NCT07855211 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT07855211?

Phase 1 / Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT07855211?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary