Condition: Methylmalonic Acidemia · Propionic Acidemia · Sponsor: RECORDATI GROUP
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.
This description comes directly from the study's public registry record.
Vincenzo Giordano, MD · 33 1 79911282 · giordano.v@recordati.com
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| Hopital Des Enfants, CHU de Bordeaux-GH Pellegrin | Bordeaux, France | Recruiting |
| Hopital de la Conception | Marseille, France | Recruiting |
| Hôpital de la Timone | Marseille, France | Recruiting |
| CHU de Nancy - Hopitaux de Brabois | Nancy, France | Recruiting |
| Hôpital Necker Enfants Malades | Paris, France | Recruiting |
| CHU de Rouen | Rouen, France | Recruiting |
| Nouvel Hopital Civil | Strasbourg, France | Recruiting |
| Hopital de Hautepierre | Strasbourg, France | Recruiting |
| Klinikum Bremen Mitte, Prof. Hess Kinderklinik | Bremen, Germany | Recruiting |
| Azienda Ospedaliero Universitaria Meyer | Florence, Italy | Recruiting |
| San Paola Hospital, ASST Santi Paolo e Carlo | Milan, Italy | Recruiting |
| Milan Policlinico University | Milan, Italy | Recruiting |
+ 20 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT04176523