Trial Outcomes & Findings for Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet (NCT NCT00272792)
NCT ID: NCT00272792
Last Updated: 2015-08-13
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
45 participants
Primary outcome timeframe
at Week 10
Results posted on
2015-08-13
Participant Flow
In Part 1, subjects received Phenoptin for 8 days as part of their evaluation for Phenoptin responsiveness. Subjects who met response criteria were randomized 3:1 to Phenoptin or placebo for additional 10 weeks in Part 2, following a minimum 1-week wash-out period.
Participant milestones
| Measure |
Sapropterin Dihydrochloride
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
|---|---|---|
|
Part 1: Responsiveness
STARTED
|
90
|
0
|
|
Part 1: Responsiveness
COMPLETED
|
89
|
0
|
|
Part 1: Responsiveness
NOT COMPLETED
|
1
|
0
|
|
Part 2: Blinded, Placebo Control Period
STARTED
|
33
|
12
|
|
Part 2: Blinded, Placebo Control Period
COMPLETED
|
32
|
9
|
|
Part 2: Blinded, Placebo Control Period
NOT COMPLETED
|
1
|
3
|
Reasons for withdrawal
| Measure |
Sapropterin Dihydrochloride
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
|---|---|---|
|
Part 1: Responsiveness
Lost to Follow-up
|
1
|
0
|
|
Part 2: Blinded, Placebo Control Period
Withdrawal by Subject
|
0
|
2
|
|
Part 2: Blinded, Placebo Control Period
Lack of Efficacy
|
1
|
0
|
|
Part 2: Blinded, Placebo Control Period
Patient Uncooperative
|
0
|
1
|
Baseline Characteristics
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Baseline characteristics by cohort
| Measure |
Sapropterin Dihydrochloride
n=33 Participants
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
n=12 Participants
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Total
n=45 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.7 years
STANDARD_DEVIATION 2.8 • n=5 Participants
|
7.1 years
STANDARD_DEVIATION 2.0 • n=7 Participants
|
7.5 years
STANDARD_DEVIATION 2.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Race
Caucasian
|
33 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
|
Race
Hispanic
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: at Week 10Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=33 Participants
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
n=12 Participants
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
|---|---|---|
|
Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria
|
20.9 mg/kg/day
Standard Deviation 15.4
|
2.9 mg/kg/day
Standard Deviation 4.0
|
SECONDARY outcome
Timeframe: Baseline to Week 3Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=33 Participants
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
n=12 Participants
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
|---|---|---|
|
Change in Phenylalanine Levels From Baseline to Week 3
|
-147.4 umol/L
Standard Error 29.0
|
-90.3 umol/L
Standard Error 48.3
|
Adverse Events
Sapropterin Dihydrochloride
Serious events: 0 serious events
Other events: 26 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Sapropterin Dihydrochloride
n=33 participants at risk
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
Placebo
n=12 participants at risk
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Contusion
|
9.1%
3/33 • Number of events 3
|
8.3%
1/12 • Number of events 1
|
|
Injury, poisoning and procedural complications
Excoriation
|
6.1%
2/33 • Number of events 3
|
0.00%
0/12
|
|
Injury, poisoning and procedural complications
Thermal Burn
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Streptococcal Infection
|
6.1%
2/33 • Number of events 2
|
16.7%
2/12 • Number of events 2
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
6.1%
2/33 • Number of events 2
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Appendicitis
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Conjuctivitis Infective
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Rhinitis
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Viral Infection
|
0.00%
0/33
|
8.3%
1/12 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
21.2%
7/33 • Number of events 7
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.2%
5/33 • Number of events 7
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal Pain
|
12.1%
4/33 • Number of events 5
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
9.1%
3/33 • Number of events 4
|
0.00%
0/12
|
|
Gastrointestinal disorders
Abdominal Pain
|
9.1%
3/33 • Number of events 6
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhoea
|
12.1%
4/33 • Number of events 5
|
0.00%
0/12
|
|
Gastrointestinal disorders
Vomiting
|
12.1%
4/33 • Number of events 4
|
0.00%
0/12
|
|
Gastrointestinal disorders
Toothache
|
6.1%
2/33 • Number of events 2
|
0.00%
0/12
|
|
Gastrointestinal disorders
Aphthous Stomatitis
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Nervous system disorders
Headache
|
21.2%
7/33 • Number of events 7
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.0%
1/33 • Number of events 1
|
16.7%
2/12 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
Erythema
|
6.1%
2/33 • Number of events 2
|
0.00%
0/12
|
|
Skin and subcutaneous tissue disorders
Prurigo
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
General disorders
Pyrexia
|
9.1%
3/33 • Number of events 3
|
16.7%
2/12 • Number of events 2
|
|
General disorders
Fatigue
|
3.0%
1/33 • Number of events 1
|
16.7%
2/12 • Number of events 2
|
|
Investigations
Neutrophil Count Decreased
|
3.0%
1/33 • Number of events 1
|
8.3%
1/12 • Number of events 1
|
|
Investigations
White Blood Cell Count Decreased
|
3.0%
1/33 • Number of events 1
|
8.3%
1/12 • Number of events 1
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
6.1%
2/33 • Number of events 2
|
0.00%
0/12
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
6.1%
2/33 • Number of events 3
|
0.00%
0/12
|
|
Psychiatric disorders
Depression
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
|
Psychiatric disorders
Mood Swings
|
0.00%
0/33
|
8.3%
1/12 • Number of events 1
|
Additional Information
Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 1-800-983-4587
Email: [email protected]
Results disclosure agreements
- 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