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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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28 February 2022 |
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Main ID: |
NCT03694353 |
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Date of registration:
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04/09/2018 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
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Scientific title:
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An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria |
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Date of first enrolment:
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September 13, 2018 |
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Target sample size:
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37 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03694353 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Director, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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BioMarin Pharmaceutical |
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Key inclusion & exclusion criteria
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Inclusion criteria:
- Must be enrolled in PAL-003 or 165-302 Part 4 at the time of screening for 165-304 and
most recently receiving pegvaliase at a dose > 40 mg/day.
- Has identified a competent person or persons = 18 years of age who can observe the
subject during study drug administration and for a minimum of 1 hour following
administration in situations required per protocol.
- For females with childbearing potential, must have negative pregnancy test at
screening and be willing to have additional pregnancy tests during the study.
- If sexually active and not planning to become pregnant (self or partner), must be
willing to use 2 acceptable methods of contraception while participating in the study
and for 4 weeks after the study.
- Is willing and able to provide written, signed informed consent after the nature of
the study has been explained and prior to any research-related procedures; a legally
authorized representative may provide written consent and assent may be requested.
- Is willing and able to comply with all study procedures.
- Is in generally good health, as evidenced by physical examination and/or clinical
laboratory evaluations (hematology, chemistry, and urinalysis).
Exclusion criteria:
- Use of any investigational product (except pegvaliase) or investigational medical
device within 30 days prior to screening or requirement for any investigational agent
prior to completion of all scheduled study assessments.
- Use of any medication (except pegvaliase) intended to treat PKU, including the use of
large neutral amino acids, within 2 days prior to the administration of pegvaliase
(Day 1).
- Use or planned use of any injectable drugs containing PEG (other than pegvaliase),
including medroxyprogesterone injection, within 3 months prior to screening and during
study participation.
- A history of organ transplantation or on chronic immunosuppressive therapy
- A history of substance abuse (as defined by the American Psychiatric Association:
Diagnostic and Statistical Manual of Mental Disorders [DSM]) in the past 12 months or
current alcohol or drug abuse
- Current participation in the Kuvan® registry study (PKU Demographics, Outcomes and
Safety [PKUDOS])
- Concurrent disease or condition that would interfere with study participation or
safety (eg, history or presence of clinically significant cardiovascular, pulmonary,
hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic,
neurological, oncologic, or psychiatric disease)
- Any condition that, in the view of the investigator, places the subject at high risk
of poor treatment compliance or terminating early from the study
Age minimum:
18 Years
Age maximum:
70 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria (PKU)
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Intervention(s)
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Drug: Pegvaliase
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Primary Outcome(s)
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Change in Blood Phe Concentration
[Time Frame: The Outcome Measure Data Table below uses the 'analysis visit' as defined by the mapping rule in the SAP. The last Phe measurement was mapped to 'analysis visit' week 121. The actual date of the last Phe measurement was day 836 (approximately Week 119).]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
[Time Frame: Up to Day 741 (approximately Week 106)]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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