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

NCT04176523 · tracked via the Priya Life Science UK tracker

Sponsor

RECORDATI GROUP

Phase

Observational

Started

2019-01-15

Last updated

2024-02-05

Condition(s) studied

Methylmalonic AcidemiaPropionic Acidemia

Investigational drug(s) / intervention(s)

Carglumic Acid

Carglumic Acid: Maintenance therapy with carglumic acid

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.

Eligibility

Sex

ALL

Min age

6 Months

Max age

99 Years

Healthy volunteers

Inclusion Criteria: 1. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA) 2. Patient initiated treatment with carglumic acid for long-term management of MMA or PA 3. Patient has been treated with carglumic acid for a minimum of 6 months 4. Patient (or caregiver) is able to comply with all prospective study procedures 5. Patient (or caregiver) is able to provide informed consent Exclusion Criteria: * None

Primary outcome measure(s)

Change in Number and Duration of Decompensation Events — 54 months
Change in the number of decompensation events before and after treatment with carglumic acid

Trial sites (32)

Facility	City	Region	Status
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
Azienda Ospedaliera San Gerardo Monza	Monza	Italy	Recruiting
Azienda Ospedaliera Universitaria Federico II	Naples	Italy	Recruiting
University Hospital of Padova	Padova	Italy	Recruiting
Turin University Hospital	Turin	Italy	Recruiting
Oslo University Hospital	Oslo	Norway	Recruiting
AMM hospital val d'hebron	Barcelona	Spain	Recruiting
Sant Joan de Deu	Barcelona	Spain	Recruiting
Universitario de Cruces	Bilbao	Spain	Recruiting
Hospital Infantil Universitario Nino Jesus	Madrid	Spain	Recruiting
Hospital 12 de Octubre	Madrid	Spain	Recruiting
Hospital Universitario La Paz	Madrid	Spain	Recruiting
Universitario Ramon y Cajal Madrid	Madrid	Spain	Recruiting
Hospital Universitario Virgen de la Arrixaca	Murcia	Spain	Recruiting
Hospital Universitario Son Espases	Palma de Mallorca	Spain	Recruiting
Hospital Universitario Virgen del Rocio	Seville	Spain	Recruiting
Universitario Miguel Servet	Zaragoza	Spain	Recruiting
NÄL	Trollhättan	Sweden	Terminated
Guy's and St. Thomas' Hospital NHS Foundation Trust	London	United Kingdom	Recruiting
Great Ormond Street Hospital	London	United Kingdom	Recruiting
Sheffield Children's Hospital NHS Foundation Trust	Sheffield	United Kingdom	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04176523 on ClinicalTrials.gov ↗ ← All trials in the UK