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Results for “Homocystinuria Due to Cystathionine Beta-Synthase Deficiency”

4 trials

Showing 4 of 4 results

Large-scale testing (Phase 3)Enrolling By InvitationNCT06431893
What this trial is testing

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)

Who this might be right for
Homocystinuria
Travere Therapeutics, Inc. 100
Not applicableStudy completedNCT02998710
What this trial is testing

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

Who this might be right for
Homocystinuria Due to CBS Deficiency
Travere Therapeutics, Inc. 110
Large-scale testing (Phase 3)Looking for participantsNCT06247085
What this trial is testing

Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Who this might be right for
Homocystinuria
Travere Therapeutics, Inc. 70
Testing effectiveness (Phase 2)Ended earlyNCT05154890
What this trial is testing

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Who this might be right for
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Aeglea Biotherapeutics 13

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