Use of Tyrosine in Nemaline Myopathy

Title:

Recruitment Status:

Terminated

Status Last Updated:

January 2014

Clinical Phenotype(s):

Nemaline Myopathy

Study Purpose:

The aim of this study is to gather information on the use of tyrosine in individuals with muscle problems.

Phase:

Unknown

Study Description:

Tyrosine is commonly used in individuals with nemaline myopathy. Information about side effects and benefits of this medication is limited. We wish to gather information about tyrosine from parents of children with nemaline myopathy and from individuals with nemaline myopathy.

Study Type:

Observational

Official Title:

Tyrosine in Nemaline Myopathy

Study Start Date:

January 2014

Study Completion Date:

Unknown

Primary Objective(s):

See above

Secondary Objective(s):

See above

Eligibility:

1. Diagnosis of nemaline myopathy by muscle biopsy or genetic testing in affected child (age <18 years) or in affected individual (age >18 years).

2. Consent must be provided by parent/caregiver (affected individual’s age less than 18 years or for those individuals greater than 18 years with learning disabilities or inability to physically access consent) or affected individual (age greater than 18 years)

Inclusion Criteria:

See above

Exclusion Criteria:

See above

Study Site(s)/Location(s):

Institute for Neuroscience and Muscle Research

Children’s Hospital at Westmead, Sydney, Australia

Sponsors & Collaborators:

Institute for Neuroscience and Muscle Research

Children’s Hospital at Westmead

Sydney, Australia

Principal Investigator(s):

Dr. Sarah Sandaradura

ClinicalTrials.gov ID:

N/A