Treatment of TNNT1-Myopathy With L-Tyrosine.

Nemaline Myopathy Clinical Trial

Official title:

Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02035501
• Other study ID #: SimonEdvardson1
• Status: Enrolling by invitation
• Phase: Phase 2
• First received: July 2, 2013
• Last updated: January 13, 2014
• Start date: January 2014

Study information

• Verified date: January 2014
• Source: Hadassah Medical Organization
• Contact: n/a
• Is FDA regulated: No
• Health authority: Israel: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 10
• Est. primary completion date: January 2015
• Gender: Both
• Age group: N/A to 20 Years

Eligibility

Inclusion Criteria:

• Male or female patients

• any age with TNNT1-related myopathy

Exclusion Criteria:

• Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Muscular Diseases
• Nemaline Myopathy

Intervention

Drug:
• L-Tyrosine

• Placebo

Locations

Country	Name	City	State
Israel	Hadassah Medical Organization, Jerusalem, Israel	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Hadassah Medical Organization

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	1. goal attainment score		3 month	No