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6R-BH4 Pulmonary Arterial Hypertension Study

NCT00435331 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2013-06-19

No results posted yet for this study

Summary

The purpose of this study is to determine whether the addition of sapropterin dihydrochloride (6R-BH4) to existing treatment has any effect in patients with pulmonary arterial hypertension (PAH). Patients with PAH have low levels of a substance called nitric oxide (NO). Tetrahydrobiopterin (BH4) is a substance produced by the body that is an essential requirement in the formation of NO. NO is thought to be helpful in keeping blood vessels in the lung healthy. 6R-BH4 is an experimental (unproven) medicine made in the lab that is very much like the BH4 that our own body makes. The researchers are investigating whether 6R-BH4 can be added safely to current treatment for PAH and whether there is any evidence of benefit from its use. The study will take approximately one year to complete from the time recruitment begins.

The primary objective of the study is to evaluate the safety of oral 6R-BH4, administered in escalating doses in addition to standard care, in subjects with pulmonary arterial hypertension (PAH).

The secondary objective of the study is to evaluate change in biochemical markers of endothelial dysfunction and nitric oxide synthetase activity (coupled and uncoupled) in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.

The third objective of the study is to evaluate change in biomarkers of disease progression, 6-minute walk (6MW) distance, Borg dyspnea scores, and quality of life (QOL) measures in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.

Conditions

Interventions

DRUG

sapropterin dihydrochloride (6R-BH4)

2.5 mg/kg/day for two weeks, 5 mg/kg/day for two weeks, 10 mg/kg/day for four weeks, then 20 mg/kg/day for two days

Sponsors & Collaborators

  • National Institutes of Health (NIH)

    collaborator NIH

  • BioMarin Pharmaceutical

    collaborator INDUSTRY

  • Vanderbilt University

    lead OTHER

Principal Investigators

  • Ivan M. Robbins, MD · Vanderbilt University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-03-31
Primary Completion
2008-09-30
Completion
2008-10-31

Countries

  • United States

Study Locations

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00435331 on ClinicalTrials.gov