Health Trials

NAGS Deficiency

2

Review clinical trials related to NAGS Deficiency. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD

Phase 1/2 Study of KRRO-121 is a randomized, placebo-controlled study in Healthy Volunteers (Phase 1 SAD and MAD) and Study Participants with Urea Cycle Disorders (Phase 2). The primary objective of the study is to evaluate the safety of KRRO-121.

Participants needed: 30
Trial details
Phase: Phase 1, Phase 2Age: 18-65Biological sex: AllType: InterventionalSponsor: Korro Bio, Inc.Updated: Aug 20, 2026Locations: 1
Eligibility criteria

Adult male or female participants, 18 to 65 years of age [+3]

Any medical history that is clinically significant in the opinion of the Princip... [+4]

Read more
Status: Recruiting

Hepatic Histopathology in Urea Cycle Disorders

This is a multi-site, retrospective chart review as well as a prospective study to evaluate histopathologic findings in liver samples from individuals with any UCD diagnosis. This study will be conducted at all Urea Cycle Disorders Consortium (UCDC) sites: Baylor College of Medicine in Houston, TX and Children's National Medical Center in Washington D.C.

Participants needed: 70
Trial details
Biological sex: AllType: ObservationalSponsor: Baylor College of MedicineUpdated: Jul 20, 2025Locations: 2
Eligibility criteria

Diagnosis of primary urea cycle disorder based on clinical suspicion confirmed b... [+2]

Unavailability of histopathology report from the liver biopsy or explant, or una... [+2]

Read more