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Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

NCT01977820 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2018-02-22

Study results available
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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

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.

Sponsors & Collaborators

Principal Investigators

  • Medical Responsible · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
29 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-28
Primary Completion
2014-11-30
Completion
2014-11-30

Countries

  • Italy
  • Switzerland

Study Locations

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Entities

Drugs
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Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01977820 on ClinicalTrials.gov