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Treatment of TNNT1-Myopathy With L-Tyrosine.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02035501
Recruitment Status : Unknown
Verified January 2014 by Shimon Edverson, Hadassah Medical Organization.
Recruitment status was:  Enrolling by invitation
First Posted : January 14, 2014
Last Update Posted : January 14, 2014
Information provided by (Responsible Party):
Shimon Edverson, 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.

Condition or disease Intervention/treatment Phase
Nemaline Myopathy Drug: L-Tyrosine Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
Study Start Date : January 2014
Estimated Primary Completion Date : January 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders
Drug Information available for: Tyrosine

Arm Intervention/treatment
Active Comparator: L-Tyrosine Drug: L-Tyrosine
Placebo Comparator: Placebo Drug: Placebo

Primary Outcome Measures :
  1. 1. goal attainment score [ Time Frame: 3 month ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02035501

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Hadassah Medical Organization, Jerusalem, Israel
Jerusalem, Israel
Sponsors and Collaborators
Hadassah Medical Organization
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Responsible Party: Shimon Edverson, MD, Hadassah Medical Organization Identifier: NCT02035501    
Other Study ID Numbers: SimonEdvardson1
First Posted: January 14, 2014    Key Record Dates
Last Update Posted: January 14, 2014
Last Verified: January 2014
Additional relevant MeSH terms:
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Muscular Diseases
Myopathies, Nemaline
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Myopathies, Structural, Congenital