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

Status: Recruiting
Location: See all (32) locations...
Intervention Type: Drug
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 99
View:

• Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)

• Patient initiated treatment with carglumic acid for long-term management of MMA or PA

• Patient has been treated with carglumic acid for a minimum of 6 months

• Patient (or caregiver) is able to comply with all prospective study procedures

• Patient (or caregiver) is able to provide informed consent

Locations
Other Locations
France
Hopital Des Enfants, CHU de Bordeaux-GH Pellegrin
RECRUITING
Bordeaux
Hopital de la Conception
RECRUITING
Marseille
Hôpital de la Timone
RECRUITING
Marseille
CHU de Nancy - Hopitaux de Brabois
RECRUITING
Nancy
Hôpital Necker Enfants Malades
RECRUITING
Paris
CHU de Rouen
RECRUITING
Rouen
Hopital de Hautepierre
RECRUITING
Strasbourg
Nouvel Hopital Civil
RECRUITING
Strasbourg
Germany
Klinikum Bremen Mitte, Prof. Hess Kinderklinik
RECRUITING
Bremen
Italy
Azienda Ospedaliero Universitaria Meyer
RECRUITING
Florence
Milan Policlinico University
RECRUITING
Milan
San Paola Hospital, ASST Santi Paolo e Carlo
RECRUITING
Milan
Azienda Ospedaliera San Gerardo Monza
RECRUITING
Monza
Azienda Ospedaliera Universitaria Federico II
RECRUITING
Napoli
University Hospital of Padova
RECRUITING
Padua
Turin University Hospital
RECRUITING
Turin
Norway
Oslo University Hospital
RECRUITING
Oslo
Spain
AMM hospital val d'hebron
RECRUITING
Barcelona
Sant Joan de Deu
RECRUITING
Barcelona
Universitario de Cruces
RECRUITING
Bilbao
Hospital 12 de Octubre
RECRUITING
Madrid
Hospital Infantil Universitario Nino Jesus
RECRUITING
Madrid
Hospital Universitario La Paz
RECRUITING
Madrid
Universitario Ramon y Cajal Madrid
RECRUITING
Madrid
Hospital Universitario Virgen de la Arrixaca
RECRUITING
Murcia
Hospital Universitario Son Espases
RECRUITING
Palma De Mallorca
Hospital Universitario Virgen del Rocio
RECRUITING
Seville
Universitario Miguel Servet
RECRUITING
Zaragoza
Sweden
NÄL
TERMINATED
Trollhättan
United Kingdom
Great Ormond Street Hospital
RECRUITING
London
Guy's and St. Thomas' Hospital NHS Foundation Trust
RECRUITING
London
Sheffield Children's Hospital NHS Foundation Trust
RECRUITING
Sheffield
Contact Information
Primary
Vincenzo Giordano, MD
giordano.v@recordati.com
33 1 79911282
Time Frame
Start Date: 2019-01-15
Estimated Completion Date: 2029-07-30
Participants
Target number of participants: 95
Treatments
Methylmalonic_acidemia
Patients with confirmed diagnosis of methylmalonic acidemia, and treated with carglumic acid, at any dose form, any dosage,
Propionic_Acidemia
Patients with confirmed diagnosis of propionic acidemia, and treated with carglumic acid, at any dose
Sponsors
Leads: Recordati Rare Diseases

This content was sourced from clinicaltrials.gov