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An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

NCT05040178 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 20

Last updated 2025-04-15

No results posted yet for this study

Summary

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Conditions

Interventions

DRUG

Carglumic Acid

Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Sponsors & Collaborators

  • Target PharmaSolutions, Inc.

    collaborator INDUSTRY

  • Recordati Rare Diseases

    lead INDUSTRY

Principal Investigators

  • William Ludlum, MD · Recordati Rare Diseases Inc.

  • Nicholas Ah Mew, MD · Children's National Research Institute

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-30
Primary Completion
2032-06-30
Completion
2032-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05040178 on ClinicalTrials.gov