Fact CheckerSign in

NCT05337553Active, not recruitingPhase 3

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension

Source: ClinicalTrials.gov · Last updated 1 Jun 2026

Brief summary (registry, in English)

This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.

Status (registry)
Active, not recruiting
Phase (registry)
Phase 3
Study type
Interventional
Enrollment
269 (actual)
First submitted
14 Apr 2022
Start date
6 Jul 2022
Primary completion date
25 Sept 2024
Lead sponsor
Biohaven Pharmaceuticals, Inc.
NCT number
NCT05337553
EU CT number
2024-511852-42-00
EudraCT number
2022-000193-25

What you commit to

  • Study type: Interventional · Phase 3
  • Participants enrolled: 269
  • From 6 Jul 2022 to the primary completion date, 25 Sept 2024
  • 53 sites in 9 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Cannot have previously taken anti-myostatin therapies

Other illness

  • 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

Other

  • Must weigh at least 15kg
  • Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter

Who the study is looking for

  • 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

Age in the registry: 4 years to 21 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (53)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Antwerp University Hospital
Coordinator of this site? Confirm its status
EdegemBelgiumActive, not recruiting
Universiteit Gent
Coordinator of this site? Confirm its status
GentBelgiumActive, not recruiting
UZ Leuven
Coordinator of this site? Confirm its status
LeuvenBelgiumActive, not recruiting
Fakultni nemocnice Brno
Coordinator of this site? Confirm its status
BrnoCzechiaActive, not recruiting
Fakultni nemocnice v Motole
Coordinator of this site? Confirm its status
PragueCzechiaActive, not recruiting
Universitaetsklinikum Essen (AoR)
Coordinator of this site? Confirm its status
EssenGermanyActive, not recruiting
Medical Center- University of Freiburg
Coordinator of this site? Confirm its status
Freiburg im BreisgauGermanyActive, not recruiting
Klinikum der Universitaet Muenchen AöR
Coordinator of this site? Confirm its status
MunichGermanyActive, not recruiting
Hospital Universitario Donostia
Coordinator of this site? Confirm its status
DonostiaSpainActive, not recruiting
Hospital Sant Joan De Deu Barcelona
Coordinator of this site? Confirm its status
Esplugues de LlobregatSpainActive, not recruiting
Hospital Universitario Regional de Málaga
Coordinator of this site? Confirm its status
MálagaSpainActive, not recruiting
Hospital Universitario Y Politecnico La Fe
Coordinator of this site? Confirm its status
ValenciaSpainActive, not recruiting
Royal Hospital for Children
Coordinator of this site? Confirm its status
GlasgowUnited KingdomUnknown status
John Radcliffe Hospital
Coordinator of this site? Confirm its status
OxfordUnited KingdomUnknown status
Azienda Unita Sanitaria Locale Di Bologna
Coordinator of this site? Confirm its status
BolognaItalyActive, not recruiting
Centro Clinico Nemo
Coordinator of this site? Confirm its status
GussagoItalyActive, not recruiting
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Coordinator of this site? Confirm its status
PaviaItalyActive, not recruiting
Ospedale Pediatrico Bambino Gesù
Coordinator of this site? Confirm its status
RomeItalyActive, not recruiting
Universitair Medisch Centrum Utrecht
Coordinator of this site? Confirm its status
UtrechtNetherlandsActive, not recruiting
Uniwersyteckie Centrum Kliniczne
Coordinator of this site? Confirm its status
GdanskPolandActive, not recruiting
Uniwersytecki Szpital Kliniczny w Poznaniu
Coordinator of this site? Confirm its status
PoznanPolandActive, not recruiting
Instytut Pomnik Centrum Zdrowia Dziecka
Coordinator of this site? Confirm its status
WarsawPolandActive, not recruiting
Dolnośląski Szpital Specjalistyczny im. T. Marciniaka - Centrum Medycyny Ratunkowej
Coordinator of this site? Confirm its status
WroclawPolandActive, not recruiting
Rare Disease Research
Coordinator of this site? Confirm its status
AtlantaUnited StatesUnknown status
Children's Hospital Colorado
Coordinator of this site? Confirm its status
AuroraUnited StatesUnknown status
UT Pediatric Neurosciences/Dell Children's Medical Center
Coordinator of this site? Confirm its status
AustinUnited StatesUnknown status
Boston Children's Hospital - Harvard
Coordinator of this site? Confirm its status
BostonUnited StatesUnknown status
University of Virginia Children's Hospital
Coordinator of this site? Confirm its status
CharlottesvilleUnited StatesUnknown status
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Coordinator of this site? Confirm its status
ChicagoUnited StatesUnknown status
Cincinnati Children's Hospital Medical Center
Coordinator of this site? Confirm its status
CincinnatiUnited StatesUnknown status
Nationwide Children's Hospital
Coordinator of this site? Confirm its status
ColumbusUnited StatesUnknown status
Neurology Rare Disease Center
Coordinator of this site? Confirm its status
DentonUnited StatesUnknown status
Duke University Medicine
Coordinator of this site? Confirm its status
DurhamUnited StatesUnknown status
University of Kansas Medical Center
Coordinator of this site? Confirm its status
FairwayUnited StatesUnknown status
Cook Children's Hospital
Coordinator of this site? Confirm its status
Fort WorthUnited StatesUnknown status
UF Health Shands Hospital
Coordinator of this site? Confirm its status
GainesvilleUnited StatesUnknown status
BSHS Office of Research
Coordinator of this site? Confirm its status
Grand RapidsUnited StatesUnknown status
Penn State College of Medicine
Coordinator of this site? Confirm its status
HersheyUnited StatesUnknown status
Indiana University -Riley Research
Coordinator of this site? Confirm its status
IndianapolisUnited StatesUnknown status
University of Iowa
Coordinator of this site? Confirm its status
Iowa CityUnited StatesUnknown status
UCSD & Rady Children's
Coordinator of this site? Confirm its status
La JollaUnited StatesUnknown status
Children's Hospital of Los Angeles
Coordinator of this site? Confirm its status
Los AngelesUnited StatesUnknown status
Medical College of Wisconsin
Coordinator of this site? Confirm its status
MilwaukeeUnited StatesUnknown status
Vanderbilt University Medical Center
Coordinator of this site? Confirm its status
NashvilleUnited StatesUnknown status
Columbia University Medical Center
Coordinator of this site? Confirm its status
New YorkUnited StatesUnknown status
Children's Hospital of The King's Daughters
Coordinator of this site? Confirm its status
NorfolkUnited StatesUnknown status
CHOP Children's Hospital of Philadelphia
Coordinator of this site? Confirm its status
PhiladelphiaUnited StatesUnknown status
Phoenix Children's
Coordinator of this site? Confirm its status
PhoenixUnited StatesUnknown status
UPMC Children's Hospital of Pittsburgh
Coordinator of this site? Confirm its status
PittsburghUnited StatesUnknown status
UCSF Benioff Children's Hospital, Medical Center
Coordinator of this site? Confirm its status
San FranciscoUnited StatesUnknown status
Washington University in St. Louis
Coordinator of this site? Confirm its status
St LouisUnited StatesUnknown status
Stony Brook University Hospital
Coordinator of this site? Confirm its status
Stony BrookUnited StatesUnknown status
MultiCare Institute of Research and Innovation
Coordinator of this site? Confirm its status
TacomaUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.