Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria

• Sponsor: Klaus Parhofer (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT04210206
• Collaborator: (none)
• Enrollment: 36
• Location: 1
• Arms: 2
• Anticipated Duration (Months): 36
• Patients Per Site Per Month: 1

Study Details

Study Description

Brief Summary

The goal of this study is to evaluate if in adult patients with phenylketonuria (PKU) without strict metabolic control during the last 12 months, strict metabolic control for 8 weeks results in an improvement of cognitive function.

Condition or Disease	Intervention/Treatment	Phase
Phenylketonurias	Other: PHE-diet	N/A

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 36 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Kurzfristiger Einfluss Einer Guten Stoffwechseleinstellung Auf Kognitive Funktion, Wohlbefinden Und Stoffwechselparameter Bei Erwachsenen Patienten Mit Phenylketonurie (Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria)
• Actual Study Start Date: Dec 1, 2019
• Anticipated Primary Completion Date: Dec 1, 2022
• Anticipated Study Completion Date: Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Diet	Other: PHE-diet; phenylalanine-restricted diet with phenylalanine free supplements for 8 weeks
No Intervention: Control

Outcome Measures

Primary Outcome Measures

1. Spatial working memory strategy index [8 weeks]

   cognitive function; scores range from 4 to 28, with lower scores representing better outcome

Secondary Outcome Measures

1. Reaction time [8 weeks]

   cognitive function; milliseconds, with faster times representing better outcome

2. Paired Associates Learning [8 weeks]

   cognitive function; scores range from 0 to 70, with lower scores indicating better outcome

3. Multitasking Test [8 weeks]

   cognitive function; scores range from 0 to 279, with lower scores indicating better outcome

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 59 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Men and women with classical PKU

• Age ≥ 18 years

• Good metabolic control during childhood (at least until 12 years of age)

• Plasma Phe levels ≥ 15 mg/dl for ≥ 1 year

• Written informed consent prior to study participation

Exclusion Criteria:

• Reduced cognitive function (unability to perform test battery)

• Drug and or alcohol abuse

• Treatment with BH4

• Treatment with drugs known to affect cognitive function

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University Munich	Munich		Germany	81377

Sponsors and Collaborators

• Klaus Parhofer

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Klaus Parhofer, Professor of Endocrinology and Metabolism, Ludwig-Maximilians - University of Munich
• ClinicalTrials.gov Identifier: NCT04210206
• Other Study ID Numbers: CoFu-PKU
• First Posted: Dec 24, 2019
• Last Update Posted: Apr 8, 2022
• Last Verified: Apr 1, 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Klaus Parhofer, Professor of Endocrinology and Metabolism, Ludwig-Maximilians - University of Munich: cognitive function adults
• Additional relevant MeSH terms: Phenylketonurias

Study Results

No Results Posted as of Apr 8, 2022