Treatment of TNNT1-Myopathy With L-Tyrosine.

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Shimon Edverson, Hadassah Medical Organization
ClinicalTrials.gov Identifier:
NCT02035501
First received: July 2, 2013
Last updated: January 13, 2014
Last verified: January 2014
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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 Hadassah Medical Organization:

Primary Outcome Measures:
  • 1. goal attainment score [ Time Frame: 3 month ] [ Designated as safety issue: No ]

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

Ages Eligible for Study:   up to 20 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT02035501

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

No publications provided

Responsible Party: Shimon Edverson, MD, Hadassah Medical Organization
ClinicalTrials.gov Identifier: NCT02035501     History of Changes
Other Study ID Numbers: SimonEdvardson1
Study First Received: July 2, 2013
Last Updated: January 13, 2014
Health Authority: Israel: Ministry of Health

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

ClinicalTrials.gov processed this record on April 17, 2014