Clinical trials

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Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.

Participants needed: 67
Trial details
Phase: Phase 2Age: 1-17Biological sex: AllType: InterventionalSponsor: Travere Therapeutics, Inc.Updated: May 12, 2026Locations: 47
Eligibility criteria

The subject or parent/legal guardian (as appropriate) is willing and able to pro... [+13]

The subject weighs <7.3 kg at screening. [+21]

Status: Recruiting

A Study to 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

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care. Study details include: * Total Study duration: up to 38 weeks * Screening: * Initial Screening duration: up to 4 weeks * Pre-treatment Diet Standardization Period duration: up to 6 weeks * Blinded Treatment Duration: 24 weeks * 2-week blinded dose titration period * 22-week blinded assessment period * Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)

Participants needed: 70
Trial details
Phase: Phase 3Age: 12-65Biological sex: AllType: InterventionalSponsor: Travere Therapeutics, Inc.Updated: May 8, 2026Locations: 52
Eligibility criteria

Must be ≥12 to ≤65 years of age, at the time of signing the informed consent [+5]

Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) defici... [+6]

Status: Recruiting

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

Participants needed: 150
Trial details
Age: 1-65Biological sex: AllType: ObservationalSponsor: Travere Therapeutics, Inc.Updated: Nov 22, 2024Locations: 11
Eligibility criteria

Patients who are clinically diagnosed with homocystinuria [+3]

Medically significant postnatal complications or congenital anomalies that are n... [+1]