Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00006823

Trial Description

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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

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Trial Acronym

SIGNAL

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URL of the Trial

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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.

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Brief Summary in Scientific Language

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Organizational Data

  •   DRKS00006823
  •   2014/09/26
  •   2013/10/31
  •   no
  •   [---]*
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Secondary IDs

  •   2010-024311-13 
  •   NCT01977820  (ClinicalTrials.gov)
  •   EMR 700773-004  (Merck KGaA)
  •   2010-024311-13 
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Health Condition or Problem studied

  •   Phenylketonuria
  •   E70.0 -  Classical phenylketonuria
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Interventions/Observational Groups

  •   Drug: Sapropterin
  •   Drug: Placebo
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Characteristics

  •   Interventional
  •   [---]*
  •   Randomized controlled trial
  •   Blinded
  •   patient/subject, investigator/therapist
  •   Placebo
  •   Treatment
  •   Parallel
  •   II
  •   [---]*
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Primary Outcome

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

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Secondary Outcome

[---]*

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Countries of Recruitment

  •   Belgium
  •   Germany
  •   Italy
  •   Netherlands
  •   Spain
  •   Switzerland
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Locations of Recruitment

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Recruitment

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  •   2014/02/27
  •   30
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   29   Years
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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

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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

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Addresses

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    • Merck KGaA
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    • Merck KGaA Communication Center 
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    • Merck KGaA Communication Center 
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Sources of Monetary or Material Support

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    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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    •   [---]*
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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov 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.