Purpose

The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

Conditions

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Patient has confirmed diagnosis of PKU with hyperphenylalaninemia documented by a Phenylalanine level of greater than or equal to 360 umol/L (6 mg/dL)
  • Patient has previously received Kuvan
  • Patient is currently receiving Kuvan
  • Patient intends to receive Kuvan therapy within 90 days of enrollment into the registry
  • The Patient is being followed at a PKUDOS participating center
  • Willing and able to provide written authorization or, if under the age of 18 years, provide written assent (if required) and written patient authorization by a parent or legal guardian
  • Willing to provide personal health information

Exclusion Criteria

  • Patients are not eligible to participate in PKUDOS if they are participating in a BioMarin-sponsored clinical study of Kuvan
  • Patients not previously treated with Kuvan and patients that are unwilling to begin Kuvan therapy within 90 days of entry into the registry

PKU MOMS Subregistry

Inclusion Criteria:

- Willing to enroll in (or are already enrolled in) PKUDOS

- Agree to follow the standard of care for pregnant women with PKU in the United States (NIH, 200, NIH Consensus Statement)

- Agree to be followed by a hospital or PKU clinic offering the standard of care for maternal PKU

- Are within 10 weeks of their last menstrual period

Exclusion Criteria:

- Patients who have not adhered to the standard of care for pregnant women with PKU in the United States are not eligible to participate in PKU MOMS

Study Design

Phase
Study Type
Observational
Observational Model
Case-Only
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
1. PKUDOS Registry Patients with a confirmed diagnosis of Phenylketonuria (PKU) with hyperphenylalaninemia who have either received Kuvan therapy, or currently receive Kuvan therapy, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.
2. PKU MOMS Subregistry Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
  • Drug: Kuvan
    After the baby is born, follow-up assessments will be requested for six months. If Kuvan is taken by the mother during and/or after pregnancy, blood samples will be requested from the mother and the baby. If the mother is lactating and taking Kuvan, breast milk samples will be requested.
    Other names:
    • sapropterin dihydrochloride

Recruiting Locations

Children's National and nearby locations

Children's National Medical Center
Washington, District of Columbia 20010

More Details

NCT ID
NCT00778206
Status
Recruiting
Sponsor
BioMarin Pharmaceutical

Study Contact

Caroline Moore
(651) 523-0310
MEDINFO@bmrn.com

Detailed Description

The PKUDOS program is a voluntary, multicenter, strictly observational program for patients with PKU who have either received Kuvan therapy, or currently receive Kuvan, or intends to begin receiving Kuvan therapy within 90 days of entering the registry.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.