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FK506 (Tacrolimus) in Pulmonary Arterial Hypertension

NCT01647945 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2016-10-05

Study results available
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Summary

Mutations in bone morphogenetic protein receptor 2 (BMPR2) are present in \>80% of familial and \~20% of sporadic pulmonary arterial hypertension (PAH) patients. Furthermore dysfunctional BMP signaling is a general feature of pulmonary hypertension even in non-familial PAH.

We therefore hypothesized that increasing BMP signaling might prevent and reverse the disease. We screened \> 3500 FDA approved drugs for their propensity to increase BMP signaling and found FK506 (Tacrolimus) to be a strong activator of BMP signaling. Tacrolimus restored normal function of pulmonary artery endothelial cells, prevented and reversed experimental PAH in mice and rats.

Given that Tacrolimus is already FDA approved with a known side-effect profile, it is an ideal candidate drug to use in patients with pulmonary arterial hypertension.

The aims of our trial are:

1. Establish the Safety of FK506 in patients with PAH.
2. Evaluate the Efficacy of FK506 in PAH
3. Identify ideal candidates for future FK506 phase III clinical trial.

Conditions

Interventions

DRUG

Placebo

placebo pill

DRUG

FK506 level < 2 ng/ml

FK506 goal trough blood level \< 2 ng/ml

DRUG

FK506 level 2-3 ng/ml

FK506 goal trough blood level 2-3 ng/ml

DRUG

FK506 level 3-5 ng/ml

FK506 goal trough blood level 3-5 ng/ml

Sponsors & Collaborators

Principal Investigators

  • Edda Spiekerkoetter, MD · Stanford University

  • Roham Zamanian, MD · Stanford University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-07-31
Primary Completion
2014-05-31
Completion
2014-08-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

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 NCT01647945 on ClinicalTrials.gov