Actively Recruiting
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Led by RECORDATI GROUP · Updated on 2024-02-05
95
Participants Needed
32
Research Sites
549 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
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Trial Site Locations
Total: 32 locations
1
Hopital Des Enfants, CHU de Bordeaux-GH Pellegrin
Bordeaux, France, 33076
Actively Recruiting
2
Hopital de la Conception
Marseille, France, 13005
Actively Recruiting
3
Hôpital de la Timone
Marseille, France, 13005
Actively Recruiting
4
CHU de Nancy - Hopitaux de Brabois
Nancy, France, 54511
Actively Recruiting
5
Hôpital Necker Enfants Malades
Paris, France, 75015
Actively Recruiting
6
CHU de Rouen
Rouen, France, 76031
Actively Recruiting
7
Nouvel Hopital Civil
Strasbourg, France, 67091
Actively Recruiting
8
Hopital de Hautepierre
Strasbourg, France, 67098
Actively Recruiting
9
Klinikum Bremen Mitte, Prof. Hess Kinderklinik
Bremen, Germany, 28205
Actively Recruiting
10
Azienda Ospedaliero Universitaria Meyer
Florence, Italy, 50139
Actively Recruiting
11
San Paola Hospital, ASST Santi Paolo e Carlo
Milan, Italy, 20142
Actively Recruiting
12
Milan Policlinico University
Milan, Italy
Actively Recruiting
13
Azienda Ospedaliera San Gerardo Monza
Monza, Italy, 20900
Actively Recruiting
14
Azienda Ospedaliera Universitaria Federico II
Naples, Italy, 80131
Actively Recruiting
15
University Hospital of Padova
Padova, Italy
Actively Recruiting
16
Turin University Hospital
Turin, Italy
Actively Recruiting
17
Oslo University Hospital
Oslo, Norway, 0372
Actively Recruiting
18
AMM hospital val d'hebron
Barcelona, Spain
Actively Recruiting
19
Sant Joan de Deu
Barcelona, Spain
Actively Recruiting
20
Universitario de Cruces
Bilbao, Spain
Actively Recruiting
21
Hospital Infantil Universitario Nino Jesus
Madrid, Spain, 28009
Actively Recruiting
22
Hospital 12 de Octubre
Madrid, Spain, 28026
Actively Recruiting
23
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
24
Universitario Ramon y Cajal Madrid
Madrid, Spain
Actively Recruiting
25
Hospital Universitario Virgen de la Arrixaca
Murcia, Spain, 30120
Actively Recruiting
26
Hospital Universitario Son Espases
Palma de Mallorca, Spain, 07010
Actively Recruiting
27
Hospital Universitario Virgen del Rocio
Seville, Spain, 41013
Actively Recruiting
28
Universitario Miguel Servet
Zaragoza, Spain
Actively Recruiting
29
NÄL
Trollhättan, Sweden
Terminated
30
Guy's and St. Thomas' Hospital NHS Foundation Trust
London, United Kingdom, SE1 7EH
Actively Recruiting
31
Great Ormond Street Hospital
London, United Kingdom, WC1N3JH
Actively Recruiting
32
Sheffield Children's Hospital NHS Foundation Trust
Sheffield, United Kingdom, S10 2TH
Actively Recruiting
Research Team
V
Vincenzo Giordano, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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