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
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, see Learn About Clinical Studies.

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
Myopathies, Structural, Congenital
Nervous System Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on October 29, 2014