Trial · NCT01425528

Study of Kuvan Treatment in Adults With GTPCH Deficiency

NCT01425528, Phase 1, Phase 2, Completed, University of Utah; results first posted 2014-04-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Kuvan Treatment in Adults With GTPCH Deficiency. Trials, record NCT01425528. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01425528

On this page

Trial

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

Title: Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT number: NCT01425528
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 12 participants, actual
Lead sponsor: University of Utah
Conditions: GTP Cyclohydrolase Deficiency
Interventions: Sapropterin
First posted: 2011-08-30
Last updated: 2025-04-01
Results first posted: 2014-04-08

Source ClinicalTrials.gov

Registered outcomes1

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

List	Outcome	Time frame	Description
Primary	Change in BH4 Levels in Cerebral Spinal Fluid	Baseline, 8 wks, 12 wks	Identify dosing range of oral Kuvan® necessary and sufficient to normalize CSF BH4 levels in adults with GTPCH Deficiency.

Source ClinicalTrials.gov

Outcomes1

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in BH4 Levels in Cerebral Spinal Fluid	Baseline, 8 wks, 12 wks	Data from 4 of 6 participants was analyzed for Kuvan Cohort 1 and for Kuvan Cohort 2 for this outcome measure. 2 participant withdrew, 1 participant's diagnosis was reclassified and we were unable to obtain baseline CSF on 1 participant.	nmol/L	MEAN

Source ClinicalTrials.gov

Results by group4

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

Outcome	Group	Category	Value	Spread	Units
Change in BH4 Levels in Cerebral Spinal Fluid	Cohort 1	Change in BH4 from Baseline at 8 weeks	6	Standard Deviation 1.4	nmol/L
Change in BH4 Levels in Cerebral Spinal Fluid	Cohort 2	Change in BH4 from Baseline at 8 weeks	10.7	Standard Deviation 4.8	nmol/L
Change in BH4 Levels in Cerebral Spinal Fluid	Cohort 1	Change in BH4 from Baseline at 12 weeks	5.3	Standard Deviation 3.8	nmol/L
Change in BH4 Levels in Cerebral Spinal Fluid	Cohort 2	Change in BH4 from Baseline at 12 weeks	13.2	Standard Deviation 3.9	nmol/L

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Cohort 1	6
Overall Study	STARTED	Cohort 2	6
Overall Study	COMPLETED	Cohort 1	5
Overall Study	COMPLETED	Cohort 2	5
Overall Study	NOT COMPLETED	Cohort 1	1
Overall Study	NOT COMPLETED	Cohort 2	1

Source ClinicalTrials.gov

Adverse events18

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	Post LP Headache	General disorders	Cohort 1	2	6	2
other	Post LP Headache	General disorders	Cohort 2	3	6	4
other	Viral Illness	General disorders	Cohort 1	2	6	2
other	Viral Illness	General disorders	Cohort 2	3	6	3
other	GI illness	Gastrointestinal disorders	Cohort 1	0	6	0
other	GI illness	Gastrointestinal disorders	Cohort 2	1	6	1
other	Headache	General disorders	Cohort 1	2	6	3
other	Headache	General disorders	Cohort 2	1	6	1
other	root canal	General disorders	Cohort 1	1	6	1
other	root canal	General disorders	Cohort 2	0	6	0

1 to 10 of 18Source ClinicalTrials.gov

See all 18 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Cohort 1
Total, all-cause mortality	Cohort 2
Total, other adverse events	Cohort 1	6	6
Total, other adverse events	Cohort 2	6	6
Total, serious adverse events	Cohort 1	1	6
Total, serious adverse events	Cohort 2	0	6

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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 comparisons1

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

Comparison
Cohort 1 vs Cohort 2: serious adverse events

Source Trial comparisons

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/NCT01425528

GET https://conductscience.org/api/trials/v1/NCT01425528/registered

API reference For AI assistants