Clinical trials: Homocystinuria Due to CBS Deficiency
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.
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