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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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12 December 2020 |
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Main ID: |
NCT01212744 |
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Date of registration:
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29/09/2010 |
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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, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
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Scientific title:
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A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria |
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Date of first enrolment:
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March 2011 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01212744 |
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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 2
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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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Ari Gershman, 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:
- 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 Criteria:
- 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.
Age minimum:
16 Years
Age maximum:
70 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria
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Intervention(s)
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Drug: rAvPAL-PEG
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Primary Outcome(s)
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Blood Phenylalanine Concentration
[Time Frame: Baseline, Week 16]
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Secondary Outcome(s)
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Percentage of Participants With PEG-IgG Antibody Positivity
[Time Frame: Baseline, Week 16]
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Percentage of Participants With Neutralizing Antibody Positivity
[Time Frame: Baseline, Week 16]
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Study Drug Related Adverse Events
[Time Frame: Weekly]
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Percentage of Participants With PAL IgG Antibody Percentage of Participants With Positive PAL IgG
[Time Frame: Baseline, Week 16]
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Percentage of Participants With PAL-IgE Antibody Positivity
[Time Frame: Baseline, Week 16]
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Percentage of Participants With PAL-IgM Antibody Positivity
[Time Frame: Baseline, Week 16]
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Percentage of Participants With PAL-PEG-IgE Antibody Positivity
[Time Frame: Baseline, Week 16]
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Percentage of Participants With PEG-IgM Antibody Positivity
[Time Frame: Baseline, Week 16]
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Plasma Concentrations of rAvPAL-PEG (BMN 165)
[Time Frame: Baseline, Week 8, Week 13]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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