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Last Updated: 01/06/2023

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A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine β-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177

Enrollment Status: Recruiting
Publish Date: December 15, 2022
Intervention Type: Drug
Study Phase: Phase 1/Phase 2

Summary: The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in up to 25 subjects with homocystinuria due to CBS deficiency.

A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058), Administered Subcutaneously in Patients With Cystathionine Beta-Synthase Deficient Homocystinuria (COMPOSE)

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...

A Phase 1, Dose-escalation, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, and Pharmacodynamics of SYNB1353 in Healthy Volunteers

Enrollment Status: Recruiting
Publish Date: November 03, 2022
Intervention Type: Drug
Study Phase: Phase 1

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.

A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients

Enrollment Status: Recruiting
Publish Date: February 04, 2022

Summary: The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 5 to 65 years under current clinical management practices

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Last Updated: 01/06/2023