Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

This study has been completed.

Study NCT00104247 Information provided by BioMarin Pharmaceutical

First Received on February 24, 2005. Last Updated on September 24, 2009 History of Changes

Results First Received: January 29, 2009

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double-Blind; Primary Purpose: Treatment
Condition:	Phenylketonurias
Intervention:	Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Sapropterin Dihydrochloride	Patients administered 10 mg /kg orally once daily.
Placebo	Placebo

Participant Flow: Overall Study

	Sapropterin Dihydrochloride	Placebo
STARTED	42	47
COMPLETED	41 ^[1]	46 ^[2]
NOT COMPLETED	1	1
Withdrawal by Subject	1	0
Noncompliant with dosing	0	1

[1]	Subject withdrew prior to dosing
[2]	One subject withdrew at Week 4, but is included in the data analysis

Baseline Characteristics

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Reporting Groups

	Description
Sapropterin Dihydrochloride	Patients administered 10 mg /kg orally once daily.
Placebo	Placebo

Baseline Measures

	Sapropterin Dihydrochloride	Placebo	Total
Number of Participants [units: participants]	41	47	88
Age [units: years] Mean ± Standard Deviation	21.5 ± 9.5	19.5 ± 9.8	20.4 ± 9.7
Age, Customized [units: Years]
8 <= Age <= 12	6	11	17
12 < Age	35	36	71
Gender [units: participants]
Female	14	23	37
Male	27	24	51
Race/Ethnicity, Customized [units: participants]
Asian/Pacific Islander	1	0	1
Caucasian	39	47	86
Other	1	0	1

Outcome Measures

1. Primary:

Change in Blood Phenylalanine Levels From Baseline to Week 6. [ Time Frame: baseline to week 6 ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The first publication of the results shall be made by Sponsor in a joint publication. If such a multi-center publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from their site individually, subject however to comply with the other terms of the agreement.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Not Applicable

Results Point of Contact:

Name/Title: Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
phone: 1-800-983-4587
e-mail: medinfo@bmrn.com

No publications provided by BioMarin Pharmaceutical

Publications automatically indexed to this study:

Levy HL, Milanowski A, Chakrapani A, Cleary M, Lee P, Trefz FK, Whitley CB, Feillet F, Feigenbaum AS, Bebchuk JD, Christ-Schmidt H, Dorenbaum A; Sapropterin Research Group. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. Lancet. 2007 Aug 11;370(9586):504-10.

ClinicalTrials.gov Identifier:	NCT00104247 History of Changes
Other Study ID Numbers:	PKU-003
Study First Received:	February 24, 2005
Results First Received:	January 29, 2009
Last Updated:	September 24, 2009
Health Authority:	United States: Food and Drug Administration