Treatment of TNNT1-Myopathy With L-Tyrosine.

NCT02035501 — Phase 2, Unknown, 10 participants, Hadassah Medical Organization

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

Registry	NCT02035501
Status	Unknown
Phase	Phase 2
Study type	Interventional; randomized, crossover, masking: quadruple, treatment
Enrollment	10 (Estimated)
Sponsor	Hadassah Medical Organization (Other)
Conditions	Nemaline Myopathy
Interventions	L-Tyrosine, Placebo (Drug)
Dates	start 2014-01-01, primary completion 2015-01-01 (estimated), first posted 2014-01-14, last updated 2014-01-14
Locations	1 site in Israel

-Summary

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

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+Arms and interventions

Arm	Type	Interventions	Description
L-Tyrosine	Active comparator	Drug: L-Tyrosine
Placebo	Placebo comparator	Drug: Placebo

L-Tyrosine (Drug)
Placebo (Drug)

+Outcome measures

Primary outcomes

1. goal attainment score [3 month]

+Eligibility

Sex: All, ages: any to 20 Years

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)

Facility	City	Country	Status
Hadassah Medical Organization, Jerusalem, Israel	Jerusalem	Israel

My Notes

Public

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