Clinical Trial Site

Rome 00168

2 studies enrolling now · 11 studies all time

What they study most

Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Amyloidosis, Hereditary, Amyloidosis, Hereditary, Transthyretin-Related, Angelman Syndrome, Elevated Urinary Oxalate Levels, Familial Amyloid Polyneuropathies, Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Primary Hyperoxaluria, Primary Hyperoxaluria Type 1

Studies at this site

• A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) — Recruiting now
• TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy — Recruiting now
• HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) — Completed
• Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures — Stopped early
• A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 — Completed
• A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder — Stopped early
• A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels — Stopped early
• APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis — Completed
• The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) — Completed
• Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome — Stopped early
• A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome — Stopped early