UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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 0 Trials 
19 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Sansone, Valeria
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Active, not recruiting
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
10/24
12/24
REACH, NCT05397470 / 2022-000389-16: Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Terminated
3
260
Europe, Canada, US
Losmapimod, Placebo oral tablet
Fulcrum Therapeutics
Facioscapulohumeral Muscular Dystrophy (FSHD)
11/24
11/24
TUDCA-ALS OLE, NCT05753852: Open Label Extension of TUDCA-ALS Study

Recruiting
3
184
Europe
Tauroursodeoxycholic Acid, TUDCA,Tudcabil, Taurolite
Humanitas Mirasole SpA, University of Ulm, University of Sheffield, University Hospital, Tours, KU Leuven, UMC Utrecht, University of Dublin, Trinity College, Bruschettini S.r.l., Istituto Superiore di Sanità, Motor Neurone Disease Association
Amyotrophic Lateral Sclerosis
03/25
03/25
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were 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
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
NCT06280209: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants with Duchenne Muscular Dystrophy

Recruiting
1/2
18
Europe, RoW
BMN 351
BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
12/25
12/25
ACHIEVE, NCT05481879 / 2022-000889-18: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Recruiting
1/2
104
Europe, RoW
DYNE-101, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Myotonic Dystrophy Type 1 (DM1)
07/26
07/26
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
NCT05575011 / 2022-000956-12: A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec

Recruiting
1
62
Europe, Canada, RoW
BIIB115, BIIB115-Matching Placebo
Biogen, Biogen Idec Research Limited
Healthy Volunteer, Muscular Atrophy, Spinal
09/27
09/27
CABLAMYD, NCT04712422: Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1

Completed
N/A
42
Europe
Healthy subjects, Pathologic group
Istituto Auxologico Italiano, Fondazione Serena Onlus - Centro Clinico NeMO Milano
Myotonic Dystrophy 1
10/21
12/22
NCT03458832: Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Recruiting
N/A
320
Europe, US
FSHD-specific functional rating scale, FSHD-COM, Electrical Impedance Myography, EIM
University of Kansas Medical Center, National Institute of Neurological Disorders and Stroke (NINDS), FSHD Society, Friends Research Institute, Inc., Muscular Dystrophy Association, AFM Telethon, University of Rochester, Leiden University Medical Center
Facioscapulohumeral Muscular Dystrophy
12/26
03/27
TREAT_CDM, NCT03059264: Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

Active, not recruiting
N/A
100
Europe, Canada, US
Natural history
Virginia Commonwealth University, Fondazione Serena Onlus - Centro Clinico NeMO Milano, University of Western Ontario, Canada, Children's Health Research Institute
Congenital Myotonic Dystrophy
03/25
03/25
NCT06579859: Development of a Registry to Assess Natural History in Duchenne Muscular Dystrophy

Not yet recruiting
N/A
200
Europe
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Duchenne Muscular Dystrophy
12/25
11/26
NCT03981575: Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Recruiting
N/A
700
Europe, Canada, US, RoW
Virginia Commonwealth University, University of Rochester, Stanford University, Ohio State University, University of Florida, University of Iowa, Ludwig-Maximilians - University of Munich, Fondazione Serena Onlus - Centro Clinico NeMO Milano, The Methodist Hospital Research Institute, Radboud University Medical Center, University College London Hospitals, University of California, Los Angeles
Myotonic Dystrophy 1, DM1
10/26
12/26
ASPIRE-DM1, NCT05224778: DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Recruiting
N/A
50
Europe, US
Virginia Commonwealth University
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, CDM, CHDM
10/26
12/26
INSIGHT FSHD2, NCT06079567: An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression

Recruiting
N/A
50
Europe
Validation of new COMs for FSHD2 patients
Centre Hospitalier Universitaire de Nice
Facioscapulohumeral Muscular Dystrophy Type 2
04/26
04/26
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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