SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan)

Phase 1

• Conditions: Phenylketonuria; MedDRA version: 14.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: PTClassification code 10034872Term: PhenylketonuriaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2009-015768-33-IT
• Lead Sponsor: MERCK SERONO SA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-28
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Diagnosis: Confirmed clinical and biochemical PKU. In addition to the above diagnosis, the inclusion criteria to be satisfied for enrolment in this study are as follows: 1. Male or female PKU infants and young children <4 years of age at the scheduled Day 1 visit of the 26-week Study Period (taking into consideration the maximum of 21 days in the Screening Period). 2. At least two previous blood Phe levels â�¥ 400 �¼mol/L obtained on 2 separate occasions. 3. Previously responded, as assessed by the Investigator, to a BH4 test, if all 3 of the following criteria are satisfied: a. The BH4 dose was 20 mg/kg/day; b. The duration of the test was at least for 24 hours; and c. Blood Phe levels decreased by at least 30%. NOTE: If a patient has not undergone a BH4 test prior to Screening, such a test must be performed during Screening, and all 3 of the above criteria must be satisfied for the subject to be eligible for entry into this study. 4. Defined level of dietary Phe tolerance consistent with the diagnosis of PKU; 5. Good adherence to dietary treatment, including prescribed dietary Phe restriction and prescribed amounts of Phe-free protein supplements and low- Phe foods. 6. Maintenance of blood Phe levels within the therapeutic target range of 120-360 �¼mol/L (defined as â�¥120 to <360 �¼mol/L) over a 1-month period prior to Screening, as assessed by the Investigator. 7. Parent(s) and/or guardian(s) willing to comply with all study procedures, maintain strict adherence to the diet, and willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study procedures.
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Use of Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin within the previous 30 days, unless for the purposes of a BH4 responsiveness test. 2. Previous exposure to Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin for >30 days. 3. Known hypersensitivity to Kuvan or its excipients. 4. Known hypersensitivity to other approved or nonapproved formulations of tetrahydrobiopterin. 5. Previous diagnosis of BH4 deficiency. 6. Current use of methotrexate, trimethoprim, or other dihydrofolate reductase inhibitors. 7. Current use of medications that are known to affect nitric oxide synthesis, metabolism or action. 8. Current use of levodopa. Current use of experimental or unregistered drugs that may affect the study outcomes. 10. Inability to comply with study procedures. 11. Inability to tolerate oral intake. 12. History of organ transplantation. 13. Concurrent disease or condition that would interfere with study participation or increase the risk for adverse events, including seizure disorders, corticosteroid administration, active malignancy, diabetes mellitus, severe congenital heart disease, renal or hepatic failure. 14. Other significant disease that in the Investigator`?s opinion would exclude the subject from the trial. 15. Any condition 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified