NCT03505125

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
63

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Mar 2018

Geographic Reach
7 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 31, 2018

Completed
13 days until next milestone

First Submitted

Initial submission to the registry

April 13, 2018

Completed
10 days until next milestone

First Posted

Study publicly available on registry

April 23, 2018

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2019

Completed
Last Updated

January 14, 2020

Status Verified

January 1, 2020

Enrollment Period

1.3 years

First QC Date

April 13, 2018

Last Update Submit

January 13, 2020

Conditions

Phenylketonuria (PKU)

Outcome Measures

Primary Outcomes (1)

  • PKU Symptom Survey

    60 item survey asking patients, observers and clinical experts about the symptoms and behaviors associated with PKU and changes in Phe level.

    15 minutes

Study Arms (3)

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.

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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

You may qualify if:

  • Patients eligible to participate in this study must meet all of the following criteria:
  • ≥18 and ≤70 years of age at the time of consent;
  • Clinically-confirmed diagnosis of Phenylketonuria (PKU);
  • Blood Phe test completed within eight weeks prior to screening date or blood Phe test completed within eight weeks post screening date;
  • 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;
  • Willing and able to participate in a telephone interview lasting approximately 90 minutes;
  • Willing to be audio-recorded during the interview session;
  • 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.

You may not qualify if:

  • Patients meeting any of the following criteria will not be included in the study:
  • Currently pregnant;
  • Current or previous exposure to pegvaliase treatment or participation in a pegvaliase clinical trial;
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

University of Florida

Gainesville, Florida, 32610, United States

Location

Emory University Department of Human Genetics

Decatur, Georgia, 30033, United States

Location

Ann and Robert H Lurie Children's Hospital

Chicago, Illinois, 60614, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Adult Metabolic Diseases Clinic, Vancouver General Hospital

Vancouver, British Columbia, Canada

Location

Necker Children's Hospital

Paris, Cedex 15, 75743, France

Location

Bretonneau Hospital, Internal Medicine Department

Tours, Cedex 9, 37044, France

Location

University Klinik Jena, Klinik für Neuropädiatrie

Jena, Thuringia, Germany

Location

Rheumatology Unit, Clinic for Inborn Errors of Metabolism

Leipzig, Germany

Location

Hospital of Padova

Padua, 35128, Italy

Location

Umberto I Polyclinic of Rome

Roma, 00185, Italy

Location

Hacettepe University

Ankara, Turkey (Türkiye)

Location

Ege University School of Medicine

Izmir, 35040, Turkey (Türkiye)

Location

National Hospital for Neurology and Neurosurgery

London, England, WC1N 3BG, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Director, MD

    BioMarin Pharmaceutical

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 13, 2018

First Posted

April 23, 2018

Study Start

March 31, 2018

Primary Completion

July 31, 2019

Study Completion

July 31, 2019

Last Updated

January 14, 2020

Record last verified: 2020-01

Locations

FR(2)DE(2)TU(2)US(4)GB(1)IT(2)CA(1)