Sapropterin Expanded Access Program

This treatment has been approved for sale to the public.
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00484991
First received: June 8, 2007
Last updated: April 11, 2008
Last verified: April 2008
  Purpose

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.


Condition Intervention
Phenylketonuria
Drug: Sapropterin dihydrochloride

Study Type: Expanded Access     What is Expanded Access?
Official Title: Sapropterin Expanded Access Program

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Intervention Details:
    Drug: Sapropterin dihydrochloride
    20mg/kg/day orally, physicians may adjust the dose within a range of 5-20mg/kg/day as warranted by their clinical judgment.
    Other Names:
    • Kuvan
    • Phenoptin
Detailed Description:

The sapropterin EAP (SEAP) is an open-label, multi-center program designed to provide access to sapropterin dihydrochloride for patients diagnosed with hyperphenylalaninemia due to PKU. All patients with a confirmed diagnosis of hyperphenylalaninemia due to PKU who are not currently enrolled in a clinical study with sapropterin dihydrochloride and meet the requisite inclusion criteria and do not meet any of the exclusion criteria may be eligible to participate in this program. Eligible patients may receive sapropterin dihydrochloride and participate in the program until commercial drug is available, however, the program will not continue beyond 2 months after the product receives marketing approval from the FDA. During the program, physicians will use their clinical judgment to assess whether a patient is a responder to sapropterin dihydrochloride. Participating physicians will measure blood Phe levels at baseline prior to treatment and then at least once between Day 7 and Day 30 following initiation of treatment to determine if the patient is a responder. A responder is a patient that has a clinically significant reduction in either the absolute or percent decrease in blood phenylalanine level compared to pre-treatment levels. The recommended starting dose of sapropterin dihydrochloride is 20 mg/kg/day. Physicians treating their patients with sapropterin dihydrochloride will be able to adjust the dose within a range of 5 mg/kg/day to 20 mg/kg/day if warranted in their clinical judgment. Adverse event information will be collected in an ongoing fashion through patient reporting AEs to their physician. Physicians will ask patients for information regarding adverse events that have occurred since the last visit. An adverse event report form will be completed in these cases and sent to BioMarin.

  Eligibility

Ages Eligible for Study:   9 Years and older
Genders Eligible for Study:   Both
Criteria

Inclusion Criteria:

  1. Patient has hyperphenylalaninemia due to PKU, a rare and serious disease
  2. Patient is not participating in a sapropterin dihydrochloride clinical study
  3. Patient is older than 8 years of age
  4. 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
  5. 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
  6. Patient is willing and able to comply with program procedures
  7. Patient lives in the United States

Exclusion Criteria:

  1. 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
  2. Patient has a concurrent disease or condition that would interfere with program participation or safety
  3. Patient is 8 years old or younger
  4. Patients is eligible for enrolling in PKU-010
  5. Patient is participating in an ongoing study with sapropterin dihydrochloride
  6. Patient is pregnant, breast feeding or considering pregnancy
  7. Patient is taking levodopa
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00484991

  Show 36 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin BPPS, BioMarin Pharmaceutical Inc.
ClinicalTrials.gov Identifier: NCT00484991     History of Changes
Other Study ID Numbers: SEAP-001
Study First Received: June 8, 2007
Last Updated: April 11, 2008
Health Authority: United States: Food and Drug Administration

Keywords provided by BioMarin Pharmaceutical:
Phenylketonuria
PKU
Phenylketonurias
Hyperphenylalaninaemia
Hydroxylase Deficiency

Additional relevant MeSH terms:
Phenylketonurias
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on October 21, 2014