Back to resultsTreatment of TNNT1-Myopathy With L-Tyrosine.
Primary Purpose
Nemaline Myopathy
Status
Unknown status
Phase
Phase 2
Locations
Israel
Study Type
Interventional
Intervention
L-Tyrosine
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Nemaline Myopathy
Eligibility 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
Sites / Locations
- Hadassah Medical Organization, Jerusalem, Israel
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
L-Tyrosine
Placebo
Arm Description
Outcomes
Primary Outcome Measures
1. goal attainment score
Secondary Outcome Measures
Full Information
NCT ID
NCT02035501
First Posted
July 2, 2013
Last Updated
January 13, 2014
Sponsor
Hadassah Medical Organization
1. Study Identification
Unique Protocol Identification Number
NCT02035501
Brief Title
Treatment of TNNT1-Myopathy With L-Tyrosine.
Official Title
Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
Study Type
Interventional
2. Study Status
Record Verification Date
January 2014
Overall Recruitment Status
Unknown status
Study Start Date
January 2014 (undefined)
Primary Completion Date
January 2015 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hadassah Medical Organization
4. Oversight
5. Study Description
Brief Summary
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Nemaline Myopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
L-Tyrosine
Arm Type
Active Comparator
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
L-Tyrosine
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
1. goal attainment score
Time Frame
3 month
10. Eligibility
Sex
All
Maximum Age & Unit of Time
20 Years
Eligibility 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
Facility Information:
Facility Name
Hadassah Medical Organization, Jerusalem, Israel
City
Jerusalem
Country
Israel
12. IPD Sharing Statement
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Treatment of TNNT1-Myopathy With L-Tyrosine.
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