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Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Sponsor: RECORDATI GROUP
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.
Key Details
Gender
All
Age Range
6 Months - 99 Years
Study Type
OBSERVATIONAL
Enrollment
95
Start Date
2019-01-15
Completion Date
2029-07-30
Last Updated
2024-02-05
Healthy Volunteers
Not specified
Conditions
Interventions
Carglumic Acid
Maintenance therapy with carglumic acid
Locations (32)
Hopital Des Enfants, CHU de Bordeaux-GH Pellegrin
Bordeaux, France
Hopital de la Conception
Marseille, France
Hôpital de la Timone
Marseille, France
CHU de Nancy - Hopitaux de Brabois
Nancy, France
Hôpital Necker Enfants Malades
Paris, France
CHU de Rouen
Rouen, France
Nouvel Hopital Civil
Strasbourg, France
Hopital de Hautepierre
Strasbourg, France
Klinikum Bremen Mitte, Prof. Hess Kinderklinik
Bremen, Germany
Azienda Ospedaliero Universitaria Meyer
Florence, Italy
San Paola Hospital, ASST Santi Paolo e Carlo
Milan, Italy
Milan Policlinico University
Milan, Italy
Azienda Ospedaliera San Gerardo Monza
Monza, Italy
Azienda Ospedaliera Universitaria Federico II
Naples, Italy
University Hospital of Padova
Padova, Italy
Turin University Hospital
Turin, Italy
Oslo University Hospital
Oslo, Norway
AMM hospital val d'hebron
Barcelona, Spain
Sant Joan de Deu
Barcelona, Spain
Universitario de Cruces
Bilbao, Spain
Hospital Infantil Universitario Nino Jesus
Madrid, Spain
Hospital 12 de Octubre
Madrid, Spain
Hospital Universitario La Paz
Madrid, Spain
Universitario Ramon y Cajal Madrid
Madrid, Spain
Hospital Universitario Virgen de la Arrixaca
Murcia, Spain
Hospital Universitario Son Espases
Palma de Mallorca, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Universitario Miguel Servet
Zaragoza, Spain
NÄL
Trollhättan, Sweden
Guy's and St. Thomas' Hospital NHS Foundation Trust
London, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sheffield Children's Hospital NHS Foundation Trust
Sheffield, United Kingdom