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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT03076814
Other study ID # PRO27634
Secondary ID
Status Withdrawn
Phase N/A
First received September 23, 2014
Last updated October 15, 2017
Start date March 2012
Est. completion date December 2015

Study information

Verified date October 2017
Source Cedars-Sinai Medical Center
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

the Investigator will test the hypothesis that short term PDE5A inhibition (with tadalafil) will reduce post-exercise edema by MRI in males with Becker Muscular Dystrophy.


Description:

Becker muscular dystrophy (BMD) is a rare, progressive and fatal muscle disease. Tadalafil is approved by the FDA for the treatment of erectile dysfunction and pulmonary hypertension. This class of medication improves muscle blood flow in a mouse model of muscular dystrophy, prevents post-exercise muscle edema, and alleviates post-exercise muscle fatigue. To begin to translate this work from the mouse into actual patients with muscular dystrophy, the Investigator group recently demonstrated that a single dose of tadalafil can improve blood flow regulation in boys with Duchenne muscular dystrophy and men with BMD. the Investigator now wish to test the hypothesis that acute treatment with Tadalafil can prevent post-exercise muscle edema in men with BMD.

the Investigator will recruit men with BMD, and healthy age-matched controls, ages 15-55y who are ambulatory and without heart failure.

Visit 1: Participants will undergo informed consent, physical exam and medical history screening, electrocardiogram, blood pressure monitoring, blood chemistry testing, echocardiogram (ejection fraction measurement), and evaluation of skeletal muscle blood flow. Patients, but not controls, will also undergo a muscle biopsy.

Visit 2 - 4: Participants will undergo baseline magnetic resonance imaging of the forearm muscle, followed by rhythmic forearm muscle exercise (handgrip). MRI will then be repeated 2-, 4-, 24-, and 48 hours following the rhythmic handgrip exercise.

Patients with BMD will then be randomized in a 3:1 fashion to tadalafil vs. placebo.

Visit 5-7: Using a standard double-blind cross-over design, patients will be treated with either tadalafil or placebo. Magnetic resonance imaging of the forearm muscle will be performed to establish baseline measurements. Patients will then repeat the rhythmic handgrip exercise protocol performed on Visit 2, followed by MRI 2-, 4-, 24-, and 48 hours following exercise.


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date December 2015
Est. primary completion date December 2015
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Male
Age group 15 Years to 55 Years
Eligibility Patient with BMD -

Inclusion Criteria:

- diagnosis of BMD confirmed by muscle biopsy or DNA analysis

- age 15-55 years of age

- ambulatory

- no clinical evidence of heart failure

Exclusion Criteria:

- hypertension, diabetes, or heart failure by standard clinical criteria

- elevated BNP (brain natriuretic peptide) level (>100 pg/ml)

- left ventricular ejection fraction < 50%

- wheelchair bound

- cardiac rhythm disorder, specifically: rhythm other than sinus, sinoventricular tachycardia, atrial fibrillation, ventricular tachycardia

- continuous ventilatory support

- liver disease

- renal impairment

- contraindications to tadalafil (use of nitrates, alpha-blockers, Cytochrome P450 (CYP3A )inhibitors, amlodipine, or other phosphodiesterase 5A inhibitors)

Healthy Control Participants -

Inclusion Criteria:

- age 15-55 years of age

- ambulatory

- no clinical evidence of heart failure

Exclusion Criteria:

- hypertension, diabetes, or heart failure by standard clinical criteria

- elevated BNP (brain natriuretic peptide) level (>100 pg/ml)

- left ventricular ejection fraction < 50%

- cardiac rhythm disorder, specifically: rhythm other than sinus, sinoventricular tachycardia, atrial fibrillation, ventricular tachycardia

- liver disease

- renal impairment

- unsatisfactory completion of the sympatholysis protocol

- BMI < 35

Study Design


Intervention

Drug:
Tadalafil

Other:
Placebo


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Cedars-Sinai Medical Center

Outcome

Type Measure Description Time frame Safety issue
Primary Change in post-exercise muscle edema by MRI. Pre vs. 48 hours post treatment change
See also
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