Clinical trials: Homocystinuria
Homocystinuria
Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers
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.
- Trial name
- HCU (NCT05462132)
- Testing
- SYNB1353
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Synlogic
Phenylketonurias, PKU
Evaluation of the Express Plus Range
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease
- Trial name
- express plus (NCT05051657)
- Testing
- PKU express plus
- Where
- 6 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd
Homocystinuria
Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition").
- Trial name
- NCT03406611
- Testing
- Pegtibatinase
- Where
- 12 sites in United States, France and 1 more
- Volunteers
- 39 people
- Sponsor
- Travere Therapeutics, Inc.
Classical Homocystinuria
The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria.
Classical homocystinuria (HCU) is an autosomal recessive disorder caused by the deficiency of an enzyme cystathionine β-synthase (CβS) that affects the catabolic pathway of the amino acid methionine (Met) which leads to an…
- Trial name
- NCT06622577
- Testing
- Cysteine
- Where
- Qatar
- Volunteers
- 50 people
- Sponsor
- Hamad Medical Corporation
Homocystinuria
Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment
Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition.
- Trial name
- HARMONY (NCT06247085)
- Testing
- Pegtibatinase
- Where
- 52 sites in United States, Australia and 12 more
- Volunteers
- 70 people
- Sponsor
- Travere Therapeutics, Inc.
Homocystinuria
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU).
- Trial name
- ENSEMBLE (NCT06431893)
- Testing
- Pegtibatinase
- Where
- 52 sites in United States, Australia and 12 more
- Volunteers
- 100 people
- Sponsor
- Travere Therapeutics, Inc.
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency
The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in approximately 36 subjects with homocystinuria due to CBS deficiency.
- Trial name
- NCT05154890
- Testing
- Pegtarviliase IV, Pegtarviliase SC
- Where
- 9 sites in United States, Australia and 1 more
- Volunteers
- 13 people
- Sponsor
- Aeglea Biotherapeutics
Homocystinuria Due to CBS Deficiency
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
- Trial name
- NCT02998710
- Where
- 11 sites in United States, Ireland and 2 more
- Volunteers
- 110 people
- Sponsor
- Travere Therapeutics, Inc.