Phase 2
N=190
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
Intermittent Claudication
Bottom Line
View on ClinicalTrials.gov: NCT00403494 ↗Enrolled (actual)
190
Serious AEs
13.3%
Results posted
Jan 2021
Primary outcome: Primary: Change in Peak Walking Time (PWT) From Baseline — 5.394; 5.712; 1.121; 1.493 minutes — p=0.727
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo (Other); Sapropterin Dihydrochloride (Drug)
- Age
- Adult, Older Adult · 40+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Nov 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Peak Walking Time (PWT) From Baseline |
5.394; 5.712; 1.121; 1.493 | 0.727 |
| PRIMARY Number of Subjects With Adverse Events (AEs) |
84; 66; 29; 30; 14; 11 | — |
| SECONDARY Change in Claudication Onset Time (COT) From Baseline |
2.159; 2.313; 0.903; 1.015 | — |
Summary
The purpose of this study is to evaluate whether sapropterin dihydrochloride is safe and effective in the treatment of intermittent claudication (IC) caused by peripheral arterial disease (PAD).
Eligibility Criteria
Inclusion Criteria
- At least 40 years and no more than 80 years old
- A 6-month (or longer) history of walking limitation because of IC, severity of which has not changed in the past 3 months
- Diagnosis of PAD secondary to atherosclerosis, with PAD documented at Screening by one of the following criteria:
- Resting ankle-brachial index (ABI) 1.3 (indicating non-compressible vessels), vascular etiology documented by toe-brachial index (TBI) 40 (gross obesity)
- Hypertension at Screening, defined as seated mean resting BP value of > 160 mmHg systolic, > 110 mmHg diastolic, or both
- Hypotension at Screening, defined as seated mean resting BP values of 2.0 mg/dL or hepatic enzyme levels more than 2 times the upper limit of normal
- Concomitant treatment with levodopa
- Requirement for concomitant treatment with any drug known to inhibit folate metabolism (eg, methotrexate)
- Use of any investigational product or device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
- Pregnant or breastfeeding at Screening or planning to become pregnant (subject or partner) at any time during the study
Data sourced from ClinicalTrials.gov (NCT00403494). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.