Clinical Trial Site
Philadelphia, Pennsylvania 19144
8 studies enrolling now · 29 studies all time
What they study most
ALA Dehydratase Deficient Porphyria (ADP), ANCA-Associated Vasculitis, ATTR Amyloidosis, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Acid Lipase Deficiency, Acute Hepatic Porphyria, Acute Intermittent Porphyria, Acute Porphyria, Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Amyloidosis, Hereditary, Transthyretin-Related
Studies at this site
- TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy — Recruiting now
- A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy — Recruiting now
- Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease — Recruiting now
- A Phase 2/3 Study of Brepocitinib in Adults With Lichen Planopilaris — Recruiting now
- ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) — Recruiting now
- ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) — Recruiting now
- Lysosomal Acid Lipase (LAL) Deficiency Registry — Recruiting now
- Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs) — Recruiting now
- A Phase 2 Study of the Safety and Efficacy of Brepocitinib in Adults With Cutaneous Sarcoidosis (BEACON) — Recruiting now
- Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution — Running, not enrolling
- A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis — Running, not enrolling
- A Study of ARV-766 Given by Mouth in Men With Metastatic Prostate Cancer — Running, not enrolling
- APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) — Completed
- Zilebesiran as Add-on Therapy in Patients With High Cardiovascular Risk and Hypertension Not Adequately Controlled by Standard of Care Antihypertensive Medications (KARDIA-3) — Completed
- A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy — Enrolling by invitation
- A Study of Brepocitinib in Adults With Active, Non-Infectious, Non-Anterior Uveitis — Running, not enrolling
- HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy — Running, not enrolling
- HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) — Completed
- Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia — Completed
- A Phase 3 Clinical Trial of CCX168 (Avacopan) in Patients With ANCA-Associated Vasculitis — Completed
- A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder — Stopped early
- ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) — Completed
- Safety, Tolerability, and Antiviral Activity of ACH-0141625 or Placebo in Combination With Peginterferon and Ribavirin in Hepatitis C Virus (HCV) Positive Participants — Completed
- Trial of Andexanet in Patients Receiving an Oral FXa Inhibitor Who Require Urgent Surgery — Stopped early
- 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
- EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP) — Completed
- ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) — Completed
- DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis — Completed