Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01114737
• Date of registration: 27/04/2010
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical
• Public title: Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
• Scientific title: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria
• Date of first enrolment: August 2010
• Target sample size: 206
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01114737
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Canada, United States

Contacts

• Name: Suyash Prasad, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Inclusion Criteria:

- = 8 years of age

- Confirmed diagnosis of PKU

- Willing to continue current diet (typical diet for the 3 months prior to study entry)
unchanged while participating in the study

- Willing and able to provide written, signed informed consent or in the case of
subjects 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

- Sexually active subjects must be willing to use an acceptable method of contraception
while participating in the study and for at least 30 days following the last dose of
sapropterin dihydrochloride

- Females of childbearing potential must have a negative pregnancy test at screening and
be willing to have additional pregnancy tests during the study. Females considered not
of childbearing potential include those who have been in menopause at least 2 years,
or had tubal ligation at least 1 year prior to screening, or have had total
hysterectomy.

- Willing and able to comply with all study procedure

Exclusion Criteria:

- Has known hypersensitivity to sapropterin dihydrochloride or its excipients

- Subject breastfeeding at screening or planning to become pregnant (subject or partner)
at any time during the study

- Use of any investigational product or investigational medical device within 30 days
prior to screening, or requirement for any investigational agent prior to the
completion of all scheduled study assessments

- Received sapropterin dihydrochloride within 16 weeks of randomization

- Have initiated or adjusted medication for treatment of ADHD, depression, or anxiety =
8 weeks prior to randomization

- Taking medication known to inhibit folate synthesis (eg, methotrexate)

- Any condition requiring treatment with levodopa or any PDE-5 inhibitor

- Concurrent disease or condition that would interfere with study participation,
compliance or safety as determined by the Investigator

- 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



Age minimum: 8 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Drug: Placebo

Drug: Sapropterin dihydrochloride

Primary Outcome(s)

Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Number of Participants With a Score of 1 or 2 in Global Function Evaluation (CGI-I) From Baseline to Week 13. [Time Frame: 13 weeks]

Secondary Outcome(s)

Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Hamilton Depression Rating Scale (HAM-D) Score From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Change in Hamilton Rating Scale For Depression (HAM-D) Score From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Change in Hamilton Depression Rating Scale (HAM-D) Score From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 13 [Time Frame: Baseline to Week 13]

Change in Clinical Global Impression-Severity (CGI-S) From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 26 [Time Frame: Baseline to Week 26]

Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Week 13 to Week 26 [Time Frame: Week 13 to Week 26]

Secondary ID(s)

PKU Ascend

PKU-016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/02/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01114737