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A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (165-901)

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: NCT03505125
Recruitment Status : Completed
First Posted : April 23, 2018
Last Update Posted : January 14, 2020
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date April 13, 2018
First Posted Date April 23, 2018
Last Update Posted Date January 14, 2020
Actual Study Start Date March 31, 2018
Actual Primary Completion Date July 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 13, 2018)
PKU Symptom Survey [ Time Frame: 15 minutes ]
60 item survey asking patients, observers and clinical experts about the symptoms and behaviors associated with PKU and changes in Phe level.
Original Primary Outcome Measures Same as current
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 A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
Official Title A Non-Interventional Observational Study of Pegvaliase-Naïve Adults With Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews
Brief Summary The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.
Detailed Description Clinical studies have suggested that treatment with pegvaliase results in lowering of blood Phenylalanine levels in adults with Phenylketonuria (PKU). Because these individuals often exhibit inattention and mood difficulties, and blood Phe concentration affects cognition and mood, life-long blood Phe control with pegvaliase may improve cognitive abilities and emotional and mental health in patients with PKU. In order to adequately quantify potential pegvaliase treatment impact on cognition and mood, it is necessary to employ clinical outcome assessments (COAs) that are fit for purpose and that comprehensively address concepts important, relevant, and meaningful to PKU patients. This is an observational study to identify symptom, function, and health-related quality of life (HRQoL) concepts of interest (COIs) to patients with PKU and to seek additional patient, observer, and expert input on the appropriateness of concepts of BioMarin's proposed COAs for use in future efficacy trials in PKU.
Study Type Observational
Study Design Observational Model: Other
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population
  1. Individuals with PKU ≥18 years to ≤ 70 years of age who are naïve to pegvaliase
  2. Observers ≥18 years of an individual with PKU ≥18 years to ≤ 70 years of age who are naïve to pegvaliase
  3. Clinical experts currently treating PKU adults
Condition Phenylketonuria (PKU)
Intervention Not Provided
Study Groups/Cohorts
  • PKU Patients
    Adults with PKU will be interviewed about the symptoms and impacts of PKU.
  • Observers
    Close friends and family members of adults with PKU will be interviewed about the behaviors they have observed in adults with PKU
  • Clinical Experts
    Experienced, practicing clinicians currently treating adults with PKU will be interviewed about the symptoms and impacts of PKU on their patients.
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 Completed
Actual Enrollment
 (submitted: January 13, 2020)
63
Original Estimated Enrollment
 (submitted: April 13, 2018)
88
Actual Study Completion Date July 31, 2019
Actual Primary Completion Date July 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

Patients eligible to participate in this study must meet all of the following criteria:

  1. ≥18 and ≤70 years of age at the time of consent;
  2. Clinically-confirmed diagnosis of Phenylketonuria (PKU);
  3. Blood Phe test completed within eight weeks prior to screening date or blood Phe test completed within eight weeks post screening date;
  4. Able to read, understand, and speak fluently the language(s) of their designated country sufficiently to participate in an interview and/or complete all assessments;
  5. Willing and able to participate in a telephone interview lasting approximately 90 minutes;
  6. Willing to be audio-recorded during the interview session;
  7. Willing and able to provide written informed consent indicating that they understand the purpose and procedures required for the study and are willing to participate.

Exclusion Criteria:

Patients meeting any of the following criteria will not be included in the study:

  1. Currently pregnant;
  2. Current or previous exposure to pegvaliase treatment or participation in a pegvaliase clinical trial;
  3. Has a significant cognitive impairment, hearing difficulty, visual impairment, acute psychopathology, medical condition, or insufficient knowledge of the interview language that, in the opinion of the investigator or interviewer, would interfere with his or her ability to provide written consent and participate/complete an interview.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   France,   Germany,   Italy,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03505125
Other Study ID Numbers 165-901
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Medical Director, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date January 2020