Trial · NCT00688844

Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

NCT00688844, Completed, Emory University; results first posted 2014-08-20

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Nutritional and Neurotransmitter Changes in PKU Subjects on BH4. Trials, record NCT00688844. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00688844

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT number: NCT00688844
Status: Completed
Enrollment: 58 participants, actual
Lead sponsor: Emory University
Conditions: Phenylketonuria
Interventions: KuvanTM Therapy
First posted: 2008-06-03
Last updated: 2015-06-08
Results first posted: 2014-08-20

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in BMI at 12 Months	Baseline and 12 months	Change in Body mass index (BMI) from baseline of Kuvan study to 12 months post-baseline
Primary	Change From Baseline in Bone Mineral Density (BMD) at 12 Months	Baseline and 12 months	Change in bone mineral density (BMD) from baseline of Kuvan study to 12 months post-baseline
Primary	Change From Baseline in Percent (%) Lean Mass at 12 Months	Baseline and 12 months	% lean mass was measured via dual energy x-ray absorptiometry (DXA)
Primary	Change From Baseline in Percent (%) Fat Mass at 12 Months	Baseline and 12 months	Percent fat mass measured via dual energy x-ray absorptiometry (DXA)
Primary	Change From Baseline in Plasma Phenylalanine at 12 Months	Baseline and 12 months	Full amino acid panel, including phenylalanine, analyzed in fasting plasma samples.
Primary	Change From Baseline in Total Dietary Protein Intake at 12 Months	Baseline and 12 months	Total dietary protein intake assessed through 3-day food records - calculated as average protein intake (grams/day) in the 3 days recorded
Primary	Change From Baseline in Phenylalanine Intake at 12 Months	Baseline and 12 months	Total dietary phenylalanine assessed through 3-day food records - calculated as average phenylalanine intake (grams/day) in the 3 days recorded
Other	Change From Baseline in Serotonin at 12 Months	Baseline and 12 months	Objective: 1 year prospective cohort of PKU patients introduced to sapropterin (Kuvan)to evaluate peripheral neurotransmitter changes across time.

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in BMI at 12 Months	Baseline and 12 months	Data missing for 2 participants	kg/m^2	MEAN
PRIMARY	Change From Baseline in Bone Mineral Density (BMD) at 12 Months	Baseline and 12 months	Data missing for 3 participants	grams/centimeter^2	MEAN
PRIMARY	Change From Baseline in Percent (%) Lean Mass at 12 Months	Baseline and 12 months	Data missing for 3 participants	Percent Lean Mass	MEAN
PRIMARY	Change From Baseline in Percent (%) Fat Mass at 12 Months	Baseline and 12 months	Data missing for 3 participants	Percent Fat Mass	MEAN
PRIMARY	Change From Baseline in Plasma Phenylalanine at 12 Months	Baseline and 12 months		Micromoles per liter	MEAN
PRIMARY	Change From Baseline in Total Dietary Protein Intake at 12 Months	Baseline and 12 months	Dietary intake not submitted by 11 participants at 12 months.	grams per day	MEAN
PRIMARY	Change From Baseline in Phenylalanine Intake at 12 Months	Baseline and 12 months	Dietary intake not submitted by 11 participants at 12 months.	grams per day	MEAN
OTHER_PRE_SPECIFIED	Change From Baseline in Serotonin at 12 Months	Baseline and 12 months

Source ClinicalTrials.gov

Results by group7

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in BMI at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	-0.65574	Standard Deviation 8.060658	kg/m^2
Change From Baseline in Bone Mineral Density (BMD) at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	0.005401	Standard Deviation 0.0243	grams/centimeter^2
Change From Baseline in Percent (%) Lean Mass at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	2.419224	Standard Deviation 3.4666	Percent Lean Mass
Change From Baseline in Percent (%) Fat Mass at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	1.208889	Standard Deviation 3.519867	Percent Fat Mass
Change From Baseline in Plasma Phenylalanine at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	-67.5187	Standard Deviation 362.9412	Micromoles per liter
Change From Baseline in Total Dietary Protein Intake at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	-10.9549	Standard Deviation 20.10814	grams per day
Change From Baseline in Phenylalanine Intake at 12 Months	Subjects With Phenylketonuria Starting Sapropterin Therapy	0.131676	Standard Deviation 0.797247	grams per day

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Subjects With Phenylketonuria Starting Sapropterin Therapy	58
Overall Study	COMPLETED	Subjects With Phenylketonuria Starting Sapropterin Therapy	48
Overall Study	NOT COMPLETED	Subjects With Phenylketonuria Starting Sapropterin Therapy	10

Source ClinicalTrials.gov

Adverse events6

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Sinus and general respiratory infections	Infections and infestations	Subjects With Phenylketonuria Starting Sapropterin Therapy	11	58	13
other	Headache	Nervous system disorders	Subjects With Phenylketonuria Starting Sapropterin Therapy	8	58	9
other	Gastrointestinal upset (not flu)	Gastrointestinal disorders	Subjects With Phenylketonuria Starting Sapropterin Therapy	3	58	3
other	Urinary tract infection	Renal and urinary disorders	Subjects With Phenylketonuria Starting Sapropterin Therapy	3	58	3
other	Flu and flu-type sickness	Infections and infestations	Subjects With Phenylketonuria Starting Sapropterin Therapy	7	58	8
other	Sore throat	Respiratory, thoracic and mediastinal disorders	Subjects With Phenylketonuria Starting Sapropterin Therapy	4	58	6

Source ClinicalTrials.gov

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Subjects With Phenylketonuria Starting Sapropterin Therapy
Total, other adverse events	Subjects With Phenylketonuria Starting Sapropterin Therapy	20	58
Total, serious adverse events	Subjects With Phenylketonuria Starting Sapropterin Therapy	0	58

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
28465125	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00688844

GET https://conductscience.org/api/trials/v1/NCT00688844/registered

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