Treatment of TNNT1-Myopathy With L-Tyrosine.

Phase 2

• Conditions: Nemaline Myopathy
• Interventions: Drug: L-Tyrosine; Drug: Placebo

• Registration Number: NCT02035501
• Lead Sponsor: Hadassah Medical Organization

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-07-02
• Status Verified: 2014-01
• Study Start: 2014-01
• Study First Submitted QC: 2014-01-13
• Last Update Submitted: 2014-01-13
• Study First Posted: 2014-01-14
• Last Update Posted: 2014-01-14
• Primary Completion: 2015-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
L-Tyrosine	L-Tyrosine	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
1. goal attainment score	3 month

Trial Locations

Locations (1)

Hadassah Medical Organization, Jerusalem, Israel; 🇮🇱 Jerusalem, Israel