Status:
COMPLETED
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria
Eligibility:
All Genders
16-70 years
Phase:
PHASE2
Brief Summary
The purpose of this study is to evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU.
Detailed Description
This 16-week multi-center, open-label, Phase 2 study is designed to evaluate the safety, tolerability,and efficacy of daily SC injections of rAvPAL-PEG in subjects with PKU. Subjects who are naïve to ...
Eligibility Criteria
Inclusion
- Diagnosis of PKU with both of the following: current blood Phe concentration of ≥ 600 micromol/L at screening and average blood Phe concentration of ≥ 600 micromol/L over the past 3 years, using available data
- Evidence that the subject is a non-responder to Kuvan® treatment (ie, 4 weeks of treatment with 20 mg/kg/day of Kuvan, insufficient response per investigator determination, and treatment end date ≥ 14 days prior to Day 1 \[ie, first dose\]). Subjects who have had a previous response to Kuvan® treatment but are not currently taking Kuvan® because of noncompliance and have been off treatment for ≥ 4 months prior to screening are eligible for participation.
- Willing and able to provide written, signed informed consent, or, in the case of participants under the age of 18, provide written assent (if required) and written informed consent by a legally authorized representative, after the nature of the study has been explained, and prior to any research-related procedures.
- Willing and able to comply with all study procedures.
- Between the ages of 16 and 70 years, inclusive.
- Negative pregnancy test at screening and willing to have additional pregnancy tests performed during the study for females of childbearing potential only. Females considered not of childbearing potential are those who have been in menopause for at least 2 years or have had a tubal ligation at least 1 year prior to screening, or who have had a total hysterectomy.
- Willing to use an acceptable method of contraception while participating in the study (sexually active subjects only).
- Maintained a stable diet with no significant modifications during the 4 weeks preceding the administration of study drug.
- In generally good health as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and ECG at screening.
Exclusion
- Prior use of rAvPAL-PEG.
- Use of any investigational product 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 that is intended to treat PKU within 14 days prior to the administration of study drug.
- Use or planned use of any injectable drugs containing PEG (other than rAvPAL-PEG), including Depo-Provera, within 3 months prior to screening and during study participation.
- Known hypersensitivity to rAvPAL-PEG excipients.
- Breastfeeding at screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study.
- 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 of not completing the study.
- Alanine aminotransferase (ALT) concentration \> 2 times the upper limit of normal.
- Creatinine \> 1.5 times the upper limit of normal.
Key Trial Info
Start Date :
March 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2015
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT01212744
Start Date
March 1 2011
End Date
April 1 2015
Last Update
February 26 2019
Active Locations (9)
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1
The Children's Hospital
Aurora, Colorado, United States, 80045
2
University of Florida
Gainesville, Florida, United States, 32610
3
Weisskopf Child Evaluation Center / University of Louisville
Louisville, Kentucky, United States, 40202
4
University of Missouri
Columbia, Missouri, United States, 65212