Trial document

This study has been imported from without additional data checks.
drksid header


Trial Description

start of 1:1-Block title


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

end of 1:1-Block title
start of 1:1-Block acronym

Trial Acronym


end of 1:1-Block acronym
start of 1:1-Block url

URL of the Trial


end of 1:1-Block url
start of 1:1-Block public summary

Brief Summary in Lay Language

This is a Phase IIa 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. This pilot trial is conducted to
detect trends and generate hypotheses, relating to variability and the possible effect size
on executive function, attention and processing speed. As this trial is exploratory in
nature, no statistical endpoints were defined as primary or secondary.

end of 1:1-Block public summary
start of 1:1-Block scientific synopsis

Brief Summary in Scientific Language


end of 1:1-Block scientific synopsis
start of 1:1-Block organizational data

Organizational Data

  •   DRKS00006823
  •   2014/09/26
  •   2013/10/31
  •   no
  •   [---]*
  •   [---]*
end of 1:1-Block organizational data
start of 1:n-Block secondary IDs

Secondary IDs

  •   2010-024311-13 
  •   NCT01977820  (
  •   EMR 700773-004  (Merck KGaA)
  •   2010-024311-13 
end of 1:n-Block secondary IDs
start of 1:N-Block indications

Health Condition or Problem studied

  •   Phenylketonuria
  •   E70.0 -  Classical phenylketonuria
end of 1:N-Block indications
start of 1:N-Block interventions

Interventions/Observational Groups

  •   Drug: Sapropterin
  •   Drug: Placebo
end of 1:N-Block interventions
start of 1:1-Block design


  •   Interventional
  •   [---]*
  •   Randomized controlled trial
  •   Blinded
  •   patient/subject, investigator/therapist
  •   Placebo
  •   Treatment
  •   Parallel
  •   II
  •   [---]*
end of 1:1-Block design
start of 1:1-Block primary endpoint

Primary Outcome

- Number of subjects with adverse event; time frame: baseline up to 30 weeks

end of 1:1-Block primary endpoint
start of 1:1-Block secondary endpoint

Secondary Outcome


end of 1:1-Block secondary endpoint
start of 1:n-Block recruitment countries

Countries of Recruitment

  •   Belgium
  •   Germany
  •   Italy
  •   Netherlands
  •   Spain
  •   Switzerland
end of 1:n-Block recruitment countries
start of 1:n-Block recruitment locations

Locations of Recruitment

end of 1:n-Block recruitment locations
start of 1:1-Block recruitment


  •   [---]*
  •   2014/02/27
  •   30
  •   Multicenter trial
  •   International
end of 1:1-Block recruitment
start of 1:1-Block inclusion criteria

Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   29   Years
end of 1:1-Block inclusion criteria
start of 1:1-Block inclusion criteria add

Additional Inclusion Criteria

- Written informed consent given before any trial-related activities are carried out

- Women or men with documented PKU diagnosed by at least two phenylalanine levels equal
or greater than 600 micromole per liter (mcmol/L)

- For women of childbearing potential: a negative urine pregnancy test is required at
screening and willingness to use a highly effective method of contraception is
required during the study and follow-up periods

- Aged greater than or equal to 18 to 29 years, inclusive

- Mean blood phenylalanine levels 600 to 1000 mcmol/L during 12 months preceding
inclusion in the study. The mean should be calculated from at least 3 blood
phenylalanine values over the last 12 months. Screening blood phenylalanine level can
be one of these values. There should be at least one value dated between Month -12
and -6 before Screening and at least one value dated between Month -6 and Screening

- An intelligence quotient (IQ) score greater than or equal to 85, assessed a maximum
of 2 years before screening with an age-appropriate Wechsler scale. If no IQ test
result is available, IQ testing must be performed as part of Screening using an
age-appropriate Wechsler scale before the subject can be included

- Subjects willing to comply with all study procedures, including willingness to
continue current dietary recommendations during the whole trial duration

end of 1:1-Block inclusion criteria add
start of 1:1-Block exclusion criteria

Exclusion Criteria

- Subjects with tetrahydrobiopterin (BH4) deficiency

- Previous exposure to sapropterin or BH4 for greater than 30 days (or exposure to
sapropterin or BH4 for less than or equal to 30 days but within the previous 6 months
prior to Screening visit)

- Subjects who, according to the Investigator, will not be able to comply with study
procedures and computerized neuropsychological testing

- Any significant illness which, according to the Investigator, might preclude
participation in the study (including neurological disease, cardio-vascular disease,
history of seizure, predisposition to convulsions, renal or hepatic insufficiency,
and active malignancy)

- Any significant illness, medication or substance abuse which, according to the
Investigator, might affect cognitive function and cognitive testing (for example,
significant visual or motor impairment, history of major head trauma, history of
stroke, alcoholism, drug dependency, psychological disorder requiring chronic use of
psychotropic medications such as anxiolytics, antidepressants, antipsychotic
medication, mood stabilizers, and hypnotics)

- Concomitant forbidden medication as described in the Kuvan® Summary of Product
Characteristics, namely, inhibitors of dihydrofolate reductase (for example,
methotrexate, trimethoprim), medications that are known to affect nitric oxide
synthesis (for example, glyceryl trinitrate, isosorbide dinitrate, sodium
nitroprusside, molsidomin, phosphodiesterase type 5 inhibitors, and minoxidil), and
levodopa, as it may cause increased excitability and irritability

- Known hypersensitivity to sapropterin or any ingredients in the product's
formulation, or to other approved or non-approved formulations of BH4

- Subjects who have undergone cognitive neuropsychological testing similar to that to
be performed as part of this trial with the following time limits: tasks with limited
practice effect performed in the last 6 months, and tasks with important practice
effect (such as tasks involving development of strategies) performed in the year
preceding inclusion in the trial. Whether a task falls into one or the other category
is left to Investigator judgment

- Female subjects who are pregnant or in the lactation period

- Subjects currently participating to another clinical trial or who participated in a
previous clinical trial within 30 days prior to screening

- Legal incapacity or limited legal capacity

end of 1:1-Block exclusion criteria
start of 1:n-Block addresses


  • start of 1:1-Block address primary-sponsor
    • Merck KGaA
    end of 1:1-Block address primary-sponsor
    start of 1:1-Block address contact primary-sponsor
    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
    end of 1:1-Block address contact primary-sponsor
  • start of 1:1-Block address scientific-contact
    • Merck KGaA Communication Center 
    end of 1:1-Block address scientific-contact
    start of 1:1-Block address contact scientific-contact
    end of 1:1-Block address contact scientific-contact
  • start of 1:1-Block address public-contact
    • Merck KGaA Communication Center 
    end of 1:1-Block address public-contact
    start of 1:1-Block address contact public-contact
    end of 1:1-Block address contact public-contact
end of 1:n-Block addresses
start of 1:n-Block material support

Sources of Monetary or Material Support

  • start of 1:1-Block address materialSupport
    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
    end of 1:1-Block address materialSupport
    start of 1:1-Block address contact materialSupport
    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
    end of 1:1-Block address contact materialSupport
end of 1:n-Block material support
start of 1:1-Block state


  •   Recruiting ongoing
  •   [---]*
end of 1:1-Block state
start of 1:n-Block publications

Trial Publications, Results and other Documents

  • [---]*
end of 1:n-Block publications
The parameters in and DRKS are not identical. Therefore the data import from required adjustments. For full details please see the DRKS FAQs .
  •   2
  •   2014/09/25
* This entry means the parameter is not applicable or has not been set.