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Register:	EUCTR
Last refreshed on:	30 June 2019
Main ID:	EUCTR2004-002071-16-IE
Date of registration:	13/10/2004
Prospective Registration:	Yes
Primary sponsor:	BioMarin Pharmaceutical Inc.
Public title:	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA
Scientific title:	A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA
Date of first enrolment:
Target sample size:	400
Recruitment status:	Not Recruiting
URL:	https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-002071-16
Study type:	Interventional clinical trial of medicinal product
Study design:	Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
Phase:	Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment
Germany	Ireland	Italy	United Kingdom

Contacts

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Key inclusion & exclusion criteria

Inclusion criteria:
1. Age = or > 8 years·
2. Blood Phe level = or > 600 µmol/L at screening· Clinical diagnosis of PKU with hyperphenylalaninemia documented by past medical history of at least one blood Phe measurement = or > 360 µmol/L (6 mg/dL) ·
3. Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained·
4. Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)· Currently using acceptable birth control measures, as determined by the investigator, and willing to continue to use acceptable birth control measures while participating in the study (if sexually active and non-sterile)·
5. Willing and able to comply with study procedures·
6. Willing to continue current diet unchanged while participating in the study
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Use of any investigational agent within 30 days prior to screening, or requirement for any investigational agent or vaccine prior to completion of all scheduled study assessments·
2. Pregnant or breastfeeding, or considering pregnancy·
3. ALT > 5 times the upper limit of normal (i.e., Grade 3 or higher based on WHO Toxicity Criteria) at screening·
4. Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid–dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes, or organ transplantation)· Serious neuropsychiatric illness (e.g., major depression) not currently under medical control·
5. Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)· Concurrent use of levodopa·
6. Clinical diagnosis of primary BH4 deficiency

Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Phenylketonuria (PKU)
MedDRA version: 6.1 Classification code 10034872

Intervention(s)

Product Name: Phenoptin
Product Code: T1401
Pharmaceutical Form: Tablet
INN or Proposed INN: Sapropterin, sapropterin hydrochloride
CAS Number: 69056-38-8
Concentration unit: mg milligram(s)
Concentration type: not less then
Concentration number: 100 mg-

Primary Outcome(s)

Primary end point(s): The percentage of subjects who experience a response to Phenoptin on Day 8 will be calculated, with response defined as a reduction in blood Phe level of = or > 30% compared with baseline in subjects with a baseline Phe of < 1200 mmol/L or a reduction of = or > 50% in subjects with a baseline Phe of = or >1200 mmol/L. In addition, the mean change in blood Phe levels at Day 8 will be estimated in the two baseline strata (<1200 mmol/L and = or > 1200 mmol/L).

Main Objective: The primary objective of this study is to evaluate the degree and frequency of response to Phenoptin treatment, as demonstrated by a reduction in blood phenylalanine (Phe) level, among subjects with phenylketonuria (PKU) who have elevated blood Phe levels.

Secondary Objective: The secondary objectives of this study are as follows:

To evaluate the safety of Phenoptin treatment in this subject population·

To identify individuals in this subject population who respond to Phenoptin treatment with a reduction in blood Phe level

Secondary Outcome(s)

Secondary ID(s)

PKU-001

Source(s) of Monetary Support

Secondary Sponsor(s)

Ethics review

Status:
Approval date:
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Results

Results available:
Date Posted:
Date Completed:
URL:

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