Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels (NCT NCT00104247)

Last Updated: 2014-07-23

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

89 participants

Primary outcome timeframe

baseline to week 6

Results posted on

2014-07-23

Participant milestones
Measure	Sapropterin Dihydrochloride Patients administered 10 mg /kg orally once daily.	Placebo Placebo
Overall Study STARTED	42	47
Overall Study COMPLETED	41	46
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal
Measure	Sapropterin Dihydrochloride Patients administered 10 mg /kg orally once daily.	Placebo Placebo
Overall Study Withdrawal by Subject	1	0
Overall Study Noncompliant with dosing	0	1

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

Baseline characteristics by cohort
Measure	Sapropterin Dihydrochloride n=41 Participants Patients administered 10 mg /kg orally once daily.	Placebo n=47 Participants Placebo	Total n=88 Participants Total of all reporting groups
Age, Continuous	21.5 years STANDARD_DEVIATION 9.5 • n=99 Participants	19.5 years STANDARD_DEVIATION 9.8 • n=107 Participants	20.4 years STANDARD_DEVIATION 9.7 • n=206 Participants
Age, Customized 8 <= Age <= 12	6 Years n=99 Participants	11 Years n=107 Participants	17 Years n=206 Participants
Age, Customized 12 < Age	35 Years n=99 Participants	36 Years n=107 Participants	71 Years n=206 Participants
Sex: Female, Male Female	14 Participants n=99 Participants	23 Participants n=107 Participants	37 Participants n=206 Participants
Sex: Female, Male Male	27 Participants n=99 Participants	24 Participants n=107 Participants	51 Participants n=206 Participants
Race/Ethnicity, Customized Asian/Pacific Islander	1 participants n=99 Participants	0 participants n=107 Participants	1 participants n=206 Participants
Race/Ethnicity, Customized Caucasian	39 participants n=99 Participants	47 participants n=107 Participants	86 participants n=206 Participants
Race/Ethnicity, Customized Other	1 participants n=99 Participants	0 participants n=107 Participants	1 participants n=206 Participants

Timeframe: baseline to week 6

Outcome measures
Measure	Sapropterin Dihydrochloride n=41 Participants Patients administered 10 mg /kg orally once daily.	Placebo n=46 Participants Placebo
Change in Blood Phenylalanine Levels From Baseline to Week 6. Baseline Blood Phe Level	843 micromole per liter Standard Deviation 300	888 micromole per liter Standard Deviation 323
Change in Blood Phenylalanine Levels From Baseline to Week 6. Week 6 Blood Phe Level	607 micromole per liter Standard Deviation 377	891 micromole per liter Standard Deviation 348
Change in Blood Phenylalanine Levels From Baseline to Week 6. Mean Change in Blood Phe from Baseline to Week 6	-239 micromole per liter Standard Deviation 38	6 micromole per liter Standard Deviation 36

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious events: 0 serious events

Other events: 34 other events

Deaths: 0 deaths

Adverse event data not reported

Other adverse events
Measure	Sapropterin Dihydrochloride n=41 participants at risk Patients administered 10 mg /kg orally once daily.	Placebo n=47 participants at risk Placebo
Infections and infestations Upper Respiratory Tract Infection	17.1% 7/41	27.7% 13/47
Infections and infestations Pharyngitis	4.9% 2/41	2.1% 1/47
Infections and infestations Infection	4.9% 2/41	0.00% 0/47
Gastrointestinal disorders Vomiting	4.9% 2/41	8.5% 4/47
Gastrointestinal disorders Abdominal Pain	2.4% 1/41	8.5% 4/47
Gastrointestinal disorders Diarrhoea	4.9% 2/41	6.4% 3/47
Nervous system disorders Headache	9.8% 4/41	14.9% 7/47
General disorders Pyrexia	4.9% 2/41	4.3% 2/47
Musculoskeletal and connective tissue disorders Back Pain	2.4% 1/41	6.4% 3/47
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/41	6.4% 3/47
Respiratory, thoracic and mediastinal disorders Pharynholaryngeal Pain	7.3% 3/41	0.00% 0/47

BioMarin Pharmaceutical Inc.

Phone: 1-800-983-4587

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER