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New drug BIIB115 aims to build on gene therapy for spinal muscular atrophy

NCT ID NCT05575011

Ongoing Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

First seen Jul 09, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This early-stage trial tests a new drug called BIIB115 for spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The study first gives a single dose to healthy adult volunteers to check safety, then moves to children with SMA who have already received the gene therapy Zolgensma. Researchers will monitor for side effects and measure how the drug moves through the body. The goal is to see if BIIB115 is safe and how it behaves, paving the way for larger studies.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
BIIB115
What this could lead to
If successful, BIIB115 could offer a new treatment option for children with spinal muscular atrophy, potentially improving muscle function and slowing disease progression.
What could go wrong
This is an early Phase 1 trial, so safety and dosing are still being established. The drug may cause side effects or may not provide meaningful benefit. Results in healthy adults may not predict outcomes in children with SMA.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEALTHY VOLUNTEER MUSCULAR ATROPHY, SPINAL

Contacts and locations

Locations

  • Centre For Human Drug Research

    Leiden, 2333, Netherlands

  • Children's Hospital of Eastern Ontario

    Ontario, K1H 8L1, Canada

  • Fondazione Serena Onlus - Centro Clinico Nemo

    Milan, 20162, Italy

  • Great Ormond Street Hospital for Children

    Bloomsbury, WC1N 3JH, United Kingdom

  • Hôpital Armand Trousseau

    Paris, 75012, France

  • Instytut "Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • Instytut Centrum Zdrowia Matki Polki Dept of Neurology

    Lodz, 93-338, Poland

  • Kyungpook National University Hospital

    Daegu, 700-721, South Korea

  • PRATIA S.A. MTZ Clinical Research Powered by Pratia

    Warsaw, 02-172, Poland

  • Pediatric Neurology Unit, Catholic University

    Rome, 00168, Italy

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Sheffield Childrens Hospital

    Sheffield, S10 2TH, United Kingdom

  • UMC Utrecht

    Utrecht, 3584 CX, Netherlands

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • Universitaetsklinikum Heidelberg

    Heidelberg, 69120, Germany

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • Universitatsklinikum Essen

    Essen, 45147, Germany

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