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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 February 2024 |
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Main ID: |
NCT04176523 |
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Date of registration:
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20/11/2019 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
PROTECT |
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Scientific title:
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Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach |
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Date of first enrolment:
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January 15, 2019 |
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Target sample size:
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95 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT04176523 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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France
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Germany
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Italy
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Norway
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Spain
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Sweden
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United Kingdom
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Contacts
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Name:
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Sufin Yap, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sheffield Children's NHS Trust |
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Name:
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Vincenzo Giordano, MD |
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Address:
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Telephone:
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33 1 79911282 |
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Email:
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giordano.v@recordati.com |
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Affiliation:
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Key inclusion & exclusion criteria
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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
Age minimum:
6 Months
Age maximum:
99 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Methylmalonic Acidemia
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Propionic Acidemia
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Intervention(s)
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Drug: Carglumic Acid
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Primary Outcome(s)
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Change in Number and Duration of Decompensation Events
[Time Frame: 54 months]
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Secondary Outcome(s)
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Caregiver Satisfaction Level
[Time Frame: 18 months]
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Caregiver Burden Exposure Frequency and Duration
[Time Frame: 18 months]
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Frequency and Duration of Healthcare Resource Utilization
[Time Frame: 18 months]
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Maintenance Dosage of NCG
[Time Frame: 54 months]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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