| CLINICAL TRIAL TITLE | CODE | INVESTIGATOR | PHASE | DISEASE | STATUS | ENROLLED PATIENTS |
|---|---|---|---|---|---|---|
Phase 2b/3 trial on the efficacy and safety of AMX0035 in Progressive Supranuclear Palsy.
| Giovanni Fabbrini | Phase 3 | Progressive Supranuclear Palsy | 3 | ||
| Study R3918-MG-2018: Efficacy and safety of combination therapy with pozelimab and cemdisiran, and cemdisiran monotherapy in patients with symptomatic generalized myasthenia gravis (NIMBLE). Objective: Drug efficacy in treating the disease | R3918-MG-2018 | Maurizio Inghilleri | Myasthenia gravis | 2 | ||
| Study: Role of cardio-specific miRNA as a screening test for asymptomatic carriers of transthyretin-mediated hereditary amyloidosis. Objective: Search for early disease screening test | Maurizio Inghilleri | TTR Amyloidosis | 6 | |||
| IMVT-1402-240: Phase 2b, multicenter, randomized, double-blind, placebo-controlled study of IMVT-1402 in adults with CIDP Objective: Drug efficacy in treating the disease | IMVT-1402-240 | Maurizio Inghilleri | Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | |||
| study ALCALS: 2023-509853-29-00 A randomized, phase II/III trial on the biological and clinical effects of acetyl-L-carnitine in ALS | ALCALS: 2023-509853-29-00 | Maurizio Inghilleri | Amyotrophic Lateral Sclerosis (ALS) | |||
Study: ARGX-117-2402 A Phase 3, Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Empasiprubart in Adults With Chronic Inflammatory Demyelinating Polyneuropathy Obiettivo: Efficacia farmaco nel trattare la patologia | ARGX-117-2402 | Maurizio Inghilleri | CIDP | |||
| A multicentric prospective study to investigate the effect of cladribrine tablets on clinical outcomes in aged population of patients with relapsing remitting multiple sclerosis (RRMS) -CLAUSE-study | Antonella Conte | Relapsing-Remitting Multiple Sclerosis (RRMS) | ||||
| Sanofi Study EFC17504 – FREVIVA: Phase 3, randomized, double-blind trial of frexalimab (SAR441344) vs placebo in adults with non-relapsing secondary progressive MS | EFC17504 | Antonella Conte | Phase 3 | Multiple Sclerosis | 3 | |
| Sanofi Study EFC17919B – FREXALT: Phase 3, randomized, double-blind trial of frexalimab (SAR441344) vs teriflunomide in adults with relapsing MS | EFC17919B | Antonella Conte | Phase 3 | Multiple Sclerosis | ||
| Librexia Trial: Secondary prevention in patients with non-cardioembolic stroke; randomized to single/dual antiplatelet vs same + milvexian (anti-XIa drug) Safety endpoint: mortality; hemorrhagic complications | Danilo Toni | Stroke Recurrence | 33 | |||
| Multicenter, randomized, double-blind, placebo-controlled trial to assess the safety, tolerability, and efficacy of XEN1101 as adjunctive therapy in focal-onset epilepsy, with an open-label extension. | XPF-008-201 | Carlo Di Bonaventura | II | Drug-resistant focal-onset epilepsy | Enrollment completed, follow-up ongoing | 5 (out of 9 in double-blind phase; 5 transitioned to open-label and are still under follow-up) |
| Cenobamate in Adults with Focal-Onset Seizures: a Real-World Multicenter Observational Italian Study – BLESS Study | BLESS | Carlo Di Bonaventura | IV (post-marketing observational) | Drug-resistant focal-onset epilepsy | Enrollment completed, follow-up ongoing | 38 |
| Double-blind, randomized, placebo-controlled, multicenter, outpatient, parallel-group study to evaluate the efficacy and safety of Staccato Alprazolam in participants aged ≥12 years with prolonged epileptic seizures. | EP00162 | Carlo Di Bonaventura | III (interventional) | Cluster seizures in patients with focal or generalized epilepsy | Enrollment ongoing | 1 screening-failure |
| Open-label extension, multicenter, outpatient study to evaluate the safety and tolerability of Staccato Alprazolam in participants aged ≥12 years with stereotyped prolonged seizures. | EP0165 | Carlo Di Bonaventura | III (interventional – open-label extension of EP00162) | Cluster seizures in patients with focal or generalized epilepsy | Enrollment ongoing | 0 |
| Phase 3 multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and efficacy of XEN1101 as adjunctive therapy in focal-onset seizures. | XPF-010-301 (X-TOLE2) | Carlo Di Bonaventura | III (interventional) | Drug-resistant focal-onset epilepsy | Enrollment ongoing | 2 enrolled (+1 screening failure) |
| Phase 3 randomized, double-blind, placebo-controlled multicenter study to evaluate the safety, tolerability, and efficacy of XEN1101 as adjunctive therapy in primary generalized tonic-clonic seizures. | XPF-010-303 (X-ACKT) | Carlo Di Bonaventura | III (interventional) | Drug-resistant primary (or likely primary) generalized tonic-clonic seizures | Enrollment ongoing | 0 |
| Multicenter, open-label, long-term study evaluating the safety, tolerability, and efficacy of XEN1101 in adults with epilepsy. | XPF-010-304 (X-TOLE4) | Carlo Di Bonaventura | III (interventional) | Open-label extension of X-TOLE2 and X-ACKT | Planned roll-over | First patient roll-over from X-TOLE2 expected on 23/06/2025 |
| Prospective observational cohort study evaluating the long-term safety of Epidyolex®, with a focus on drug-induced liver injury (DILI). | GWEP21042 | Carlo Di Bonaventura | IV (post-marketing observational) | Epileptic encephalopathies (Dravet syndrome, Lennox-Gastaut syndrome) or Tuberous Sclerosis Complex | Enrollment ongoing | Coordinating center in Italy Patients: 18 |
| A Double-Blind, Randomized, Multicenter, Trial Evaluating the Efficacy and Safety of PRAX-628 in Adults with Focal Seizures | PRAX-628-321 | Carlo Di Bonaventura | II-III (interventional) | Drug-resistant focal-onset epilepsy | Planned start date: 11/06/2025 | |
A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC) | TrustTSC | Carlo Di Bonaventura | III (interventional) | Tuberous Sclerosis Complex | Completed (October 2024) | 1 pz arruolato |
| A Phase 3, Open-Label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy (TrustTSC OLE). | TrustTSC OLE | Carlo Di Bonaventura | III (interventional – open-label extension of TrustTSC) | Tuberous Sclerosis Complex | Closed by sponsor in April 2025 | 1 pz arruolato |