U.O. Complessa di Neuropsichiatria Infantile
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 5 Trials 
12 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Mercuri, Eugenio
RESPOND, NCT04488133 / 2020-003492-18: A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

Active, not recruiting
4
46
Europe, US, RoW
Nusinersen, ISIS 396443, BIIB058, Spinraza
Biogen
Muscular Atrophy, Spinal
10/25
10/25
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
ASCEND, NCT05067790 / 2021-001294-23: A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
ULYSSES, NCT05933057: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

Recruiting
3
138
Europe, Canada
Givinostat, ITF2357, Placebo
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
02/28
02/28
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
SYNAPSE-SMA, NCT05794139 / 2022-002301-24: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Recruiting
2
54
Europe, Canada, US
NMD670, Placebo
NMD Pharma A/S, NMD Pharma A/S
Spinal Muscular Atrophy
07/25
07/25
NCT06769633: Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

Not yet recruiting
2
18
Europe
Givinostat Hydrochloride, Cohort 1, Givinostat Hydrochloride - Cohort 2, Cohort 2
Italfarmaco, Fortrea
Duchenne Muscular Dystrophy
12/29
12/29
CircSMA, NCT05760209: SMN Circular RNAs as Potential New Targets and Biomarkers for SMA

Recruiting
N/A
30
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
07/24
07/24
NCT05779956: Personalized Medicine for SMA: a Translational Project

Recruiting
N/A
90
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Ospedale Pediatrico Bambino Gesu
Spinal Muscular Atrophy
08/24
08/24
NCT05772416: Neonatal Neurological Examination to Detect Infants at Risk

Recruiting
N/A
100
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Neonatal Encephalopathy
08/25
08/25
NCT05768048: Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

Recruiting
N/A
500
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Bambino Gesù Hospital and Research Institute, University of Messina, Gaslini Children's Hospital
Spinal Muscular Atrophy
11/27
11/27
iSMAR, NCT05755451: Natural History of SMA

Recruiting
N/A
1200
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS, University of Milan, Istituto Giannina Gaslini, Ospedale Pediatrico Bambin Gesù, University of Messina
Muscular Atrophy, Spinal
12/33
12/33

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