- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02035501
Treatment of TNNT1-Myopathy With L-Tyrosine.
January 13, 2014 updated by: Shimon Edverson, Hadassah Medical Organization
Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
Study Overview
Study Type
Interventional
Enrollment (Anticipated)
10
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Jerusalem, Israel
- Hadassah Medical Organization, Jerusalem, Israel
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 20 years (Child, Adult)
Accepts Healthy Volunteers
N/A
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
|
|
Active Comparator: L-Tyrosine
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
1. goal attainment score
Time Frame: 3 month
|
3 month
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2014
Primary Completion (Anticipated)
January 1, 2015
Study Registration Dates
First Submitted
July 2, 2013
First Submitted That Met QC Criteria
January 13, 2014
First Posted (Estimate)
January 14, 2014
Study Record Updates
Last Update Posted (Estimate)
January 14, 2014
Last Update Submitted That Met QC Criteria
January 13, 2014
Last Verified
January 1, 2014
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- SimonEdvardson1
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Nemaline Myopathy
-
Clinic for Special ChildrenRecruitingMyopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANMUnited States
-
Vastra Gotaland RegionEnrolling by invitationMusculoskeletal Diseases | Neuromuscular Diseases | Centronuclear Myopathy | Nemaline Myopathy | Congenital Myopathy | Myosin Storage MyopathySweden
-
Radboud University Medical CenterUMC UtrechtNot yet recruitingCentronuclear Myopathy | Nemaline Myopathy | Central Core Disease | Multi-Minicore DiseaseNetherlands
-
Boston Children's HospitalMuscular Dystrophy AssociationRecruitingCentronuclear Myopathy | Myotubular Myopathy | Nemaline Myopathy | Central Core Disease | Congenital Fiber Type Disproportion | Multiminicore Disease | Rigid Spine Muscular Dystrophy | Undefined Congenital MyopathyUnited States
-
Radboud University Medical CenterCompletedInspiratory Muscle Training | Nemaline MyopathyNetherlands
-
Radboud University Medical CenterUnknownMyotonic Dystrophy Type 2 | McArdle Disease | Nemaline Myopathy Type 6
-
Newcastle-upon-Tyne Hospitals NHS TrustRecruitingX-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-LinkedUnited Kingdom
-
Ultragenyx Pharmaceutical IncTerminatedHereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing MyopathyCanada, United States, Bulgaria
-
James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaboratorsRecruitingX Linked Myotubular MyopathyUnited Kingdom, Canada, United States
-
Ultragenyx Pharmaceutical IncTerminatedHereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing MyopathyUnited States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Clinical Trials on Placebo
-
SamA Pharmaceutical Co., LtdUnknownAcute Bronchitis | Acute Upper Respiratory Tract InfectionKorea, Republic of
-
National Institute on Drug Abuse (NIDA)CompletedCannabis UseUnited States
-
AstraZenecaParexel; Spandauer Damm 130; 14050; Berlin, GermanyCompletedMale Subjects With Type II Diabetes (T2DM)Germany
-
Heptares Therapeutics LimitedCompletedPharmacokinetics | Safety IssuesUnited Kingdom
-
GlaxoSmithKlineCompletedPulmonary Disease, Chronic ObstructiveUnited Kingdom, Netherlands
-
ItalfarmacoCompletedBecker Muscular DystrophyNetherlands, Italy
-
Shijiazhuang Yiling Pharmaceutical Co. LtdXuanwu Hospital, BeijingCompleted
-
GlaxoSmithKlineCompletedInfections, BacterialUnited States
-
West Penn Allegheny Health SystemCompletedAsthma | Allergic RhinitisUnited States