APPROVED_FOR_MARKETINGNot Applicable

Sapropterin Expanded Access Program

United States

Plain-language summary

The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded access program (EAP) until commercial product is available.

Who can participate

Age range9 Years

SexALL

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Inclusion criteria

✓. Patient has hyperphenylalaninemia due to PKU, a rare and serious disease
✓. Patient is not participating in a sapropterin dihydrochloride clinical study
✓. Patient is older than 8 years of age
✓. Patient is willing and able to provide written informed consent or, in the case of under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian
✓. If female and of child bearing potential, the patient has a negative urine pregnancy test within 24 hours prior to enrollment (females of child-bearing potential only) and will be using adequate contraceptive methods to avoid pregnancy while participating in the program
✓. Patient is willing and able to comply with program procedures
✓. Patient lives in the United States

Exclusion criteria

✕. Patient is perceived to be unreliable or unwilling to comply with program participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unwilling to comply with program participation
✕. Patient has a concurrent disease or condition that would interfere with program participation or safety
✕. Patient is 8 years old or younger
✕. Patients is eligible for enrolling in PKU-010
✕. Patient is participating in an ongoing study with sapropterin dihydrochloride
✕. Patient is pregnant, breast feeding or considering pregnancy
✕. Patient is taking levodopa

Trial details

NCT IDNCT00484991

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Phenylketonuria trials