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Treatment of TNNT1-Myopathy With L-Tyrosine.

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2014 by Shimon Edverson, Hadassah Medical Organization.
Recruitment status was:  Enrolling by invitation
Sponsor:
ClinicalTrials.gov Identifier:
NCT02035501
First Posted: January 14, 2014
Last Update Posted: January 14, 2014
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.
Information provided by (Responsible Party):
Shimon Edverson, Hadassah Medical Organization
  Purpose
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.

Condition Intervention Phase
Nemaline Myopathy Drug: L-Tyrosine Drug: Placebo Phase 2

Study Type: Interventional
Study Design: 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.

Resource links provided by NLM:


Further study details as provided by Shimon Edverson, Hadassah Medical Organization:

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

Estimated Enrollment: 10
Study Start Date: January 2014
Estimated Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: L-Tyrosine Drug: L-Tyrosine
Placebo Comparator: Placebo Drug: Placebo

  Eligibility

Information from the National Library of Medicine

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

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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT02035501


Locations
Israel
Hadassah Medical Organization, Jerusalem, Israel
Jerusalem, Israel
Sponsors and Collaborators
Hadassah Medical Organization
  More Information

Responsible Party: Shimon Edverson, MD, Hadassah Medical Organization
ClinicalTrials.gov Identifier: NCT02035501     History of Changes
Other Study ID Numbers: SimonEdvardson1
First Submitted: July 2, 2013
First Posted: January 14, 2014
Last Update Posted: January 14, 2014
Last Verified: January 2014

Additional relevant MeSH terms:
Muscular Diseases
Myopathies, Nemaline
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Myopathies, Structural, Congenital