Treatment of TNNT1-Myopathy With L-Tyrosine.

Phase 2UnknownNCT02035501

Hadassah Medical Organization10 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Nemaline Myopathy

Interventions

L-TyrosineDRUG

PlaceboDRUG

Eligibility

Sex: ALLMax age: 20 Years

Medical Language ↔ Plain English

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

Locations (1)

Hadassah Medical Organization, Jerusalem, Israel

Jerusalem, Israel

Outcomes

Primary Outcomes

1. goal attainment score

Time frame: 3 month

Data from ClinicalTrials.gov

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