Who is this study for: Patients with homocystinuria caused by cystathionine beta-synthase deficiency
Enrollment Status: Recruiting
Publish Date: November 21, 2022
Intervention Type: Drug
Study Drug: OT-58
Study Phase: Phase 1/Phase 2
Summary: Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocysteine (Hcy) in the plasma, tissues and urine. It is due to reduced or absent activity of the CBS enzyme, and is also known as classical homocystinuria. The symptoms associated with homocystinuria are variable in severity and time of onset acro...
Summary: This is a Phase 1, double-blind (Sponsor-open), placebo-controlled, randomized, dose-escalation, inpatient study using a multiple-ascending dose (MAD) design to assess the safety, tolerability, and PD of SYNB1353 in HVs.