Disease Area/ Indication

Name of PI

Name of Trial

phase I-III

Name of sponsor

Remarks

Mucopolysaccharidosis

CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren

II

PTC

Cancer - Solid Tumor

A phase II, multicenter study of EZH2 inhibitor in Adult subjects with INI1-negative tumors or relapsed/refractory synovial scarcoma

II

Epizyme

Opioid Dependence

An Open-Label Multicenter Study Assessing the Long-Term Safety of a Once-Weekly and Once-Monthly, Long-Acting Subcutaneous Injection Depot of Buprenorphine in Adult Outpatients with Opioid Use Disorder

II

Braeburn

Familial Amyloidotic Polyneuropahty

A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran in Transthyretin (TTR)-Mediated Polyneuropathy

III

Alnylam

Homozygous Familial Hypercholesterolemia

A Randomized, Double-Blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia

III

AstraZeneca

Urinary Tract Infection

A Multi-Center, Randomized, Double-Blind, Double-Dummy, Study to Evaluate the Efficacy, Safety, and Tolerability of Carbavance Compared to Piperacillin/Tazobactam in the Treatment of Complicated Urinary Tract Infections, Including Acute Pyelonephritis

III

Rempex

Heterozygous Familial Hypercholesterolemia

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients with Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol

III

Genzyme