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

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01948427
Recruitment Status : Active, not recruiting
First Posted : September 23, 2013
Last Update Posted : May 15, 2019
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Tracking Information
First Submitted Date September 16, 2013
First Posted Date September 23, 2013
Last Update Posted Date May 15, 2019
Actual Study Start Date September 2013
Estimated Primary Completion Date September 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 16, 2016)
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.
Original Primary Outcome Measures
 (submitted: September 18, 2013)
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
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Official Title Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
Brief Summary 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.
Detailed Description

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.

Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients with a confirmed or suspected diagnosis of UCD
Condition Urea Cycle Disorder
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: May 14, 2019)
209
Original Estimated Enrollment
 (submitted: September 18, 2013)
500
Estimated Study Completion Date September 2026
Estimated Primary Completion Date September 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01948427
Other Study ID Numbers HPN-100-014
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Horizon Pharma Ireland, Ltd., Dublin Ireland
Study Sponsor Horizon Pharma Ireland, Ltd., Dublin Ireland
Collaborators Not Provided
Investigators Not Provided
PRS Account Horizon Pharma Ireland, Ltd., Dublin Ireland
Verification Date May 2019