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Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

NCT04176523 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 95

Last updated 2024-02-05

No results posted yet for this study

Summary

This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.

Conditions

Interventions

DRUG

Carglumic Acid

Maintenance therapy with carglumic acid

Sponsors & Collaborators

  • Recordati Rare Diseases

    lead INDUSTRY

Principal Investigators

  • Sufin Yap, MD · Sheffield Children's NHS Trust

Eligibility

Min Age
6 Months
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-15
Primary Completion
2024-12-30
Completion
2029-07-30
FDA Drug
Yes

Countries

  • France
  • Germany
  • Italy
  • Norway
  • Spain
  • Sweden
  • United Kingdom

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 NCT04176523 on ClinicalTrials.gov