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History of Changes for Study: NCT03505125
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (165-901)
Latest version (submitted January 13, 2020) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 April 13, 2018 None (earliest Version on record)
2 July 9, 2018 Study Status and Contacts/Locations
3 July 20, 2018 Contacts/Locations and Study Status
4 August 29, 2018 Study Status and Contacts/Locations
5 September 25, 2018 Study Status and Contacts/Locations
6 November 13, 2018 Study Status and Contacts/Locations
7 January 13, 2020 Recruitment Status, Study Status, Contacts/Locations and Study Design
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Study NCT03505125
Submitted Date:  April 13, 2018 (v1)

Open or close this module Study Identification
Unique Protocol ID: 165-901
Brief Title: A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (165-901)
Official Title: A Non-Interventional Observational Study of Pegvaliase-Naïve Adults With Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews
Secondary IDs:
Open or close this module Study Status
Record Verification: April 2018
Overall Status: Enrolling by invitation
Study Start: March 31, 2018
Primary Completion: September 30, 2018 [Anticipated]
Study Completion: December 31, 2018 [Anticipated]
First Submitted: April 13, 2018
First Submitted that
Met QC Criteria:
April 13, 2018
First Posted: April 23, 2018 [Actual]
Last Update Submitted that
Met QC Criteria:
April 13, 2018
Last Update Posted: April 23, 2018 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: BioMarin Pharmaceutical
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: No
U.S. FDA-regulated Device: No
Data Monitoring: No
Open or close this module Study Description
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.
Open or close this module Conditions
Conditions: Phenylketonuria (PKU)
Keywords:
Open or close this module Study Design
Study Type: Observational
Observational Study Model: Other
Time Perspective: Retrospective
Biospecimen Retention:
Biospecimen Description:
Enrollment: 88 [Anticipated]
Number of Groups/Cohorts 3
Open or close this module Groups and Interventions
Groups/Cohorts Interventions
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.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 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.
Open or close this module Eligibility
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
Sampling Method: Non-Probability Sample
Minimum Age: 18 Years
Maximum Age: 70 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
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.
Open or close this module Contacts/Locations
Study Officials: Jan Iles, MD
Study Director
BioMarin Pharmaceutical
Locations: United States, Illinois
Ann and Robert H Lurie Children's Hospital
Chicago, Illinois, United States, 60614
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:

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