NCT02847975

Brief Summary

Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD). If tadalafil restores functional sympatholysis in BMD via the NO-cyclic guanosine monophosphate pathway, then functional sympatholysis should also be restored by sodium nitrite- which is an indirect nitric oxide donor.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Oct 2013

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2013

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

April 30, 2014

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
1.7 years until next milestone

First Posted

Study publicly available on registry

July 28, 2016

Completed
Last Updated

August 6, 2018

Status Verified

August 1, 2018

Enrollment Period

1.2 years

First QC Date

April 30, 2014

Last Update Submit

August 2, 2018

Conditions

Keywords

Beckerdystrophysympatholysis

Outcome Measures

Primary Outcomes (1)

  • change in muscle tissue oxygenation

    The pre-specified primary outcome is the pre vs. post treatment change in functional sympatholysis measured by muscle oxygenation.

    change from baseline to post treatment (3-4 hours)

Study Arms (1)

Sodium nitrate

EXPERIMENTAL

Sodium nitrate will be administered orally and functional sympatholysis assessed before and after treatment

Dietary Supplement: Sodium nitrate

Interventions

Sodium nitrateDIETARY_SUPPLEMENT

Sodium nitrate will be ingested orally

Sodium nitrate

Eligibility Criteria

Age15 Years - 55 Years
Sexmale
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Becker muscular dystrophy
  • age 15-55 years of age
  • ambulatory

You may not qualify if:

  • hypertension, diabetes, or heart failure by standard clinical criteria
  • elevated brain natriuretic peptide level (\>100 pg/ml)
  • Left ventricular ejection fraction \< 50%
  • cardiac rhythm disorder, specifically: rhythm other than sinus, supraventricular tachycardia, atrial fibrillation, ventricular tachycardia, heart block
  • continuous ventilatory support
  • liver disease
  • renal impairment
  • history of asthma or bronchospasm
  • use of any medications other than common supplements
  • unable to perform handgrip exercise

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Cedars-Sinai Heart Institute

Los Angeles, California, 90048, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Related Publications (1)

  • Martin EA, Barresi R, Byrne BJ, Tsimerinov EI, Scott BL, Walker AE, Gurudevan SV, Anene F, Elashoff RM, Thomas GD, Victor RG. Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med. 2012 Nov 28;4(162):162ra155. doi: 10.1126/scitranslmed.3004327.

    PMID: 23197572BACKGROUND

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

sodium nitrate

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Ronald G Victor, MD

    Cedars-Sinai Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Medicine

Study Record Dates

First Submitted

April 30, 2014

First Posted

July 28, 2016

Study Start

October 1, 2013

Primary Completion

December 1, 2014

Study Completion

December 1, 2014

Last Updated

August 6, 2018

Record last verified: 2018-08

Data Sharing

IPD Sharing
Will not share

Published http://www.ncbi.nlm.nih.gov/pubmed/26437761

Locations