Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise for SMA patients in major trial

NCT ID NCT05337553

Disease control Sponsor: Biohaven Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to see if the drug helps with motor skills like moving arms and legs.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

269 people

The number who actually took part.

Started

Jul 2022

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number * Ambulant or Non-Ambulant * Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen and/or risdiplam and/or a history of onasemnogene abeparvovec Key Exclusion Criteria: * Cannot have previously taken anti-myostatin therapies * Must weigh at least 15kg * Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable) * History of Spinal Fusion within 6 months of Screening. MAGEC rod nonsurgical adjustments are allowed during the study * Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Neuromuscular diseases are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

neuromuscular disease proximal spinal muscular atrophy spinal muscular atrophy

As listed by the trial registrant

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

Neuromuscular diseases SMA Spinal muscular atrophy

Contacts and locations

Locations

  • BSHS Office of Research

    Grand Rapids, Michigan, 49503, United States

  • Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience

    Roma, 00165, Italy

  • Boston Children's Hospital - Harvard

    Boston, Massachusetts, 02115, United States

  • CHOP Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23507, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Cook Children's Hospital

    Fort Worth, Texas, 76104, United States

  • Donostia University Hospital

    Donostia / San Sebastian, 20014, Spain

  • Dr. Von Haunersches Children'S Hospital - Lmu Munich

    Munich, 80337, Germany

  • Duke University Medicine

    Durham, North Carolina, 27705, United States

  • Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic

    Poznan, 60-355, Poland

  • Hospital Sant Joan de Déu

    Esplugues de Llobregat, 08950, Spain

  • IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT

    Pavia, 27100, Italy

  • Indiana University -Riley Research

    Indianapolis, Indiana, 46202, United States

  • Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital

    Bologna, 40139, Italy

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • La Fe University and Polytechnic Hospital

    Valencia, 46026, Spain

  • Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit

    Málaga, 29011, Spain

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Motol University Hospital

    Prague, 150 06, Czechia

  • MultiCare Institute of Research and Innovation

    Tacoma, Washington, 98405, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nemo-Brescia Clinical Center For Neuromuscular Diseases

    Gussago, 25064, Italy

  • Neurology Rare Disease Center

    Denton, Texas, 75208, United States

  • Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Penn State College of Medicine

    Hershey, Pennsylvania, 17033, United States

  • Phoenix Children's

    Phoenix, Arizona, 85016, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Royal Hospital For Children

    Glasgow, Scotland, G51 4TF, United Kingdom

  • Stony Brook University Hospital

    Stony Brook, New York, 11794, United States

  • T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept.

    Wroclaw, 54-049, Poland

  • The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology

    Warsaw, 04-730, Poland

  • UCSD & Rady Children's

    La Jolla, California, 92037, United States

  • UCSF Benioff Children's Hospital, Medical Center

    San Francisco, California, 94158, United States

  • UF Health, Shands Hospital

    Gainesville, Florida, 32610, United States

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • UT Pediatric Neurosciences/Dell Children's Medical Center

    Austin, Texas, 78723, United States

  • University Clinical Centre in Gdansk - Dept. of Developmental Neurology

    Gdansk, 80-925, Poland

  • University Hospital Antwerp

    Edegem, 02650, Belgium

  • University Hospital Brno - Dept. of Pediatric Neurology

    Brno, 625 00, Czechia

  • University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I

    Essen, 45147, Germany

  • University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders

    Freiburg im Breisgau, 79106, Germany

  • University Hospital Ghent

    Ghent, 09000, Belgium

  • University Hospital Leuven

    Leuven, 03000, Belgium

  • University Medical Center Utrecht

    Utrecht, 3584 CX, Netherlands

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Fairway, Kansas, 66205, United States

  • University of Virginia Children's Hospital

    Charlottesville, Virginia, 22903, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.