Trials / Terminated
TerminatedNCT01977820
Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
A Multicenter, Double-Blind, Placebo-Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
- Status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 2 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- 18 Years – 29 Years
- Healthy volunteers
- Not accepted
Summary
This is a Phase 2a multicenter, double-blind, placebo-controlled, randomized, 2-arm pilot trial designed to assess the effect of sapropterin on cognitive abilities in young adults with Phenylketonuria (PKU) over a 26-week treatment period.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Sapropterin | Sapropterin tablets will be administered orally once daily at a dose of 20 milligram per kilogram (mg/kg) during both the 2-Week response test period and 24-Week study period. |
| DRUG | Placebo | Sapropterin tablets will be administered orally once daily at a dose of 20 milligram per kilogram (mg/kg) during both the 2-Week response test period and Placebo tablets matching to sapropterin will be administered orally once daily during the 24-Week study period. |
Timeline
- Start date
- 2014-02-01
- Primary completion
- 2014-11-01
- Completion
- 2014-11-01
- First posted
- 2013-11-07
- Last updated
- 2018-02-22
- Results posted
- 2016-02-12
Locations
2 sites across 2 countries: Italy, Switzerland
Source: ClinicalTrials.gov record NCT01977820. Inclusion in this directory is not an endorsement.