Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01732471
• Date of registration: 19/11/2012
• Prospective Registration: No
• Primary sponsor: Merck KGaA
• Public title: Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
• Scientific title: A Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine Levels
• Date of first enrolment: November 2012
• Target sample size: 90
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01732471
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Germany

Key inclusion & exclusion criteria

Inclusion Criteria:

- Willing and able to provide written informed consent (for children under 18 years old
the parent[s]/guardians give informed consent, subjects 14-17 years old give
additionally their own written informed consent)

- Age of 4 - 18 years, inclusive

- Confirmed clinical and biochemical hyperphenylalaninemia due to phenylketonuria
documented by past medical history with at least 2 blood phenylalanine level greater
than or equal to 400 micromole per liter obtained in 2 separate occasions

- Blood phenylalanine level at screening greater than or equal to 450 micromole per
liter (mean of two measurements)

- For women of childbearing potential, a negative urine pregnancy test is required at
screening and willingness to use a highly effective method of contraception is
required while participating in the study

- Subject and/or the parent/guardian willing and able to comply with study procedures

- Subject and/or the parent/guardian willing to continue current diet unchanged during
the 8 days response test and to adapt the diet according to phenylalanine therapeutic
target range during the 6 week treatment period

Exclusion Criteria:

- Subject already assessed for responsiveness to sapropterin dihydrochloride or other
tetrahydrobiopterin (BH4)

- Used any investigational agent other than Kuvan® (sapropterin dihydrochloride) within
30 days of screening, or required any investigational agent or vaccine prior to
completion of all scheduled study assessments

- Pregnant or breastfeeding, or considering pregnancy

- Concurrent disease or conditions that would interfere with study participation or
safety (for example, seizure disorder, asthma or other condition requiring oral or
parenteral corticosteroid administration, insulin-dependent diabetes, or organ
transplantation recipient)

- Concurrent use of required concomitant treatment with any drug known to inhibit folate
synthesis (for example, methotrexate), levodopa, phosphodiesterase type-5 (PDE-5)
inhibitors (such as, sildenafil, vardenafil or tadalafil), medications that are known
to affect nitric oxide synthesis metabolism or action

- Any conditions, that, in the view of the Principal Investigator renders the subject at
high risk for failure to comply with treatment or to complete the study

- Clinical diagnosis of primary BH4 deficiency

- Known hypersensitivity to Kuvan® (sapropterin dihydrochloride) or its excipients or to
other approved or non-approved formulation of tetrabiopterin



Age minimum: 4 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Drug: Kuvan®

Primary Outcome(s)

Percentage of Participants With Response to Kuvan® (Sapropterin Dihydrochloride) Treatment [Time Frame: Day 8]

Secondary Outcome(s)

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population [Time Frame: Baseline up to Week 11]

Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders [Time Frame: Baseline, Day 8]

Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population [Time Frame: Baseline, Day 8]

Secondary ID(s)

EMR 700773_510

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 28/07/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01732471