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Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs) (THRIVE)

This study is currently recruiting participants.
Verified November 2016 by Horizon Pharma Ireland, Ltd., Dublin Ireland
Sponsor:
ClinicalTrials.gov Identifier:
NCT01948427
First Posted: September 23, 2013
Last Update Posted: November 11, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland
September 16, 2013
September 23, 2013
November 11, 2016
September 2013
September 2026   (Final data collection date for primary outcome measure)
Assessment of UCD specific disease characteristics [ Time Frame: Patients will be followed for the duration of the registry. The expected duration is 10 years. ]
Assessment of: Blood chemistry, neurocognitive outcomes, growth and development and medications related to the management of UCD.
Assessment of UCD specific disease characteristics [ Time Frame: Patients will be followed for the duration of the registry. The expected duration is 10 years. ]

Assessment of:

Blood chemistry, neurocognitive outcomes, growth and development and medications related to the management of UCD

Complete list of historical versions of study NCT01948427 on ClinicalTrials.gov Archive Site
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Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years. As an observational study, enrolled patients will not be required to make any additional office visits or take any medicine outside of normal care.

UCDs disproportionately affect children and females: depending on the severity of the defect, a UCD can manifest shortly after birth or later in life. This study will track long-term outcomes in UCD patients and effects of ammonia-scavenging agents on neuropsychological functions of UCD patients.

This is a non-interventional, multi-center registry to be conducted in patients with UCDs. Investigators will prescribe treatments based on usual clinical practice, and there will be no restrictions on the use of commercially available medications. As an observational study, this study will not change the patient/ healthcare provider relationship, nor influence the healthcare provider's drug prescription or the therapeutic management of the patient.

Patients with UCDs will be recruited and invited to attend a Baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. Patients will be followed for up to 10 years, during which time they will be assessed by their healthcare provider. Patients and healthcare provider will be asked to report episodes of hyperammonemic crisis, available ammonia values, and other information.

Observational [Patient Registry]
Observational Model: Cohort
Time Perspective: Prospective
10 Years
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Non-Probability Sample
Patients with a confirmed or suspected diagnosis of UCD
Urea Cycle Disorder
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Recruiting
500
September 2026
September 2026   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Confirmed or suspected diagnosis of UCD
  • Signed informed consent/Health Insurance Portability and Accountability Act (HIPAA) Authorization and medical records release

Exclusion Criteria:

  • Any other reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact: United Biosource Corporation 855-UCD-2595 thriveregistry@unitedbiosource.com
United States
 
 
NCT01948427
HPN-100-014
No
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Horizon Pharma Ireland, Ltd., Dublin Ireland
Horizon Pharma Ireland, Ltd., Dublin Ireland
Not Provided
Not Provided
Horizon Pharma Ireland, Ltd., Dublin Ireland
November 2016