A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.

Registry ID

EUCTR2010-024311-13

Source registry

EUCTR

Status

Prematurely Ended

Phase

PHASE2

Sponsor

Merck KGaA

Start date

2013-11-22

Last update

2026-08-16

Conditions

• Phenylketonuria

Summary

The purpose of this pilot trial is to assess the effect of sapropterin on the cognitive abilities of young adults with phenylketonuria (PKU).

Locations

• , , Italy
• , , Belgium
• , , Netherlands
• , , Spain
• , , Germany

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