Trial · NCT00403494

A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease

NCT00403494, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2021-01-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease. Trials, record NCT00403494. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00403494

On this page

Trial

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

Title: A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT number: NCT00403494
Status: Completed
Phases: Phase 2
Enrollment: 190 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Intermittent Claudication
Interventions: Placebo, Sapropterin Dihydrochloride
First posted: 2006-11-23
Last updated: 2021-01-07
Results first posted: 2021-01-07

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Change in Peak Walking Time (PWT) From Baseline	Baseline and up to Week 24	This is to assess the effect of oral sapropterin dihydrochloride versus placebo on peak walking time (PWT) in subjects with intermittent claudication (IC) caused by peripheral arterial disease (PAD).
Primary	Number of Subjects With Adverse Events (AEs)	Up to 24-weeks	Adverse events were described and summarized with focus on treatment- emergent events (TEAEs). A TEAE was defined as any AE that presented , increased in frequency or worsened in severity following initiation of study drug administration. If the onset of an AE was missing then the AE was considered treatment emergent. Drug-related AEs are AEs classified by the investigator as possibly or probably related to study drug.
Secondary	Change in Claudication Onset Time (COT) From Baseline	Baseline and up to Week 24	Time, in minutes, when the subject first begins to experience claudication symptoms, regardless of whether this is manifested as muscle pain, ache, cramp, numbness or fatigue.
Other	Change in Peak Walking Time From Baseline With and Without Vitamin C	Baseline up to 24 weeks	The first 50% of subjects enrolled in the study were randomized to receive sapropterin dihydrochloride at 400 mg BID or oral placebo BID alone (without vitamin C). When approximately 50% enrollment was met, 1000 mg/day vitamin C was added to the dose regimen of newly enrolled subjects in both sapropterin dihydrochloride and placebo treatment groups given orally in two divided doses of 500 mg with study drug.

Source ClinicalTrials.gov

Outcomes4

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Peak Walking Time (PWT) From Baseline	Baseline and up to Week 24	Intent-To-Treat Population consisting of all subjects who were randomized, received 1 dose of study drug and had at least 1 post-baselline treadmill test.	minutes	MEAN
PRIMARY	Number of Subjects With Adverse Events (AEs)	Up to 24-weeks	Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change in Claudication Onset Time (COT) From Baseline	Baseline and up to Week 24	Intent-To-Treat Population	minutes	MEAN
OTHER_PRE_SPECIFIED	Change in Peak Walking Time From Baseline With and Without Vitamin C	Baseline up to 24 weeks	Intent-to-Treat Population	minutes	MEAN

Source ClinicalTrials.gov

Results by group34

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

Outcome	Group	Category	Value	Spread	Units
Change in Peak Walking Time (PWT) From Baseline	Sapropterin Dihydrochloride	Baseline	5.394	Standard Deviation 2.803	minutes
Change in Peak Walking Time (PWT) From Baseline	Placebo	Baseline	5.712	Standard Deviation 2.876	minutes
Change in Peak Walking Time (PWT) From Baseline	Sapropterin Dihydrochloride	Change from Baseline to Week 24	1.121	Standard Deviation 2.775	minutes
Change in Peak Walking Time (PWT) From Baseline	Placebo	Change from Baseline to Week 24	1.493	Standard Deviation 2.597	minutes
Number of Subjects With Adverse Events (AEs)	Sapropterin Dihydrochloride	Any AEs	84		Participants
Number of Subjects With Adverse Events (AEs)	Placebo	Any AEs	66		Participants
Number of Subjects With Adverse Events (AEs)	Sapropterin Dihydrochloride	Study Drug-related AEs	29		Participants
Number of Subjects With Adverse Events (AEs)	Placebo	Study Drug-related AEs	30		Participants
Number of Subjects With Adverse Events (AEs)	Sapropterin Dihydrochloride	Any SAEs	14		Participants
Number of Subjects With Adverse Events (AEs)	Placebo	Any SAEs	11		Participants

1 to 10 of 34Source ClinicalTrials.gov

See all 34 results by group

Participant flow12

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Sapropterin Dihydrochloride	96
Overall Study	STARTED	Placebo	94
Overall Study	Intent to Treat	Sapropterin Dihydrochloride	87
Overall Study	Intent to Treat	Placebo	83
Overall Study	Per-protocol	Sapropterin Dihydrochloride	69
Overall Study	Per-protocol	Placebo	69
Overall Study	Safety Population	Sapropterin Dihydrochloride	97
Overall Study	Safety Population	Placebo	91
Overall Study	COMPLETED	Sapropterin Dihydrochloride	81
Overall Study	COMPLETED	Placebo	80

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events108

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
other	Diarrhoea	Gastrointestinal disorders	Sapropterin Dihydrochloride	4	97
other	Diarrhoea	Gastrointestinal disorders	Placebo	7	91
other	Nausea	Gastrointestinal disorders	Sapropterin Dihydrochloride	5	97
other	Nausea	Gastrointestinal disorders	Placebo	4	91
other	Pharmaceutical product complaint	General disorders	Sapropterin Dihydrochloride	4	97
other	Pharmaceutical product complaint	General disorders	Placebo	7	91
other	Back pain	Musculoskeletal and connective tissue disorders	Sapropterin Dihydrochloride	9	97
other	Back pain	Musculoskeletal and connective tissue disorders	Placebo	4	91
other	Pain in extremity	Musculoskeletal and connective tissue disorders	Sapropterin Dihydrochloride	5	97
other	Pain in extremity	Musculoskeletal and connective tissue disorders	Placebo	6	91

1 to 10 of 108Source ClinicalTrials.gov

See all 108 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sapropterin Dihydrochloride	1	97
Total, all-cause mortality	Placebo	0	91
Total, other adverse events	Sapropterin Dihydrochloride	84	97
Total, other adverse events	Placebo	66	91
Total, serious adverse events	Sapropterin Dihydrochloride	14	97
Total, serious adverse events	Placebo	11	91

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 comparisons2

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

Comparison	Source
Sapropterin Dihydrochloride vs Placebo: deaths from any cause	Trial comparisons
Sapropterin Dihydrochloride vs Placebo: serious adverse events	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/NCT00403494

GET https://conductscience.org/api/trials/v1/NCT00403494/results?page=2

API reference For AI assistants