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

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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
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

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