NCT02035501

Brief Summary

The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 2, 2013

Completed
6 months until next milestone

Study Start

First participant enrolled

January 1, 2014

Completed
13 days until next milestone

First Posted

Study publicly available on registry

January 14, 2014

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2015

Completed
Last Updated

January 14, 2014

Status Verified

January 1, 2014

Enrollment Period

1 year

First QC Date

July 2, 2013

Last Update Submit

January 13, 2014

Conditions

Nemaline Myopathy

Outcome Measures

Primary Outcomes (1)

  • 1. goal attainment score

    3 month

Study Arms (2)

L-Tyrosine

ACTIVE COMPARATOR
Drug: L-Tyrosine

Placebo

PLACEBO COMPARATOR
Drug: Placebo

Interventions

L-TyrosineDRUG
L-Tyrosine
PlaceboDRUG
Placebo

Eligibility Criteria

AgeUp to 20 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Male or female patients
  • any age with TNNT1-related myopathy

You may not qualify if:

  • Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hadassah Medical Organization, Jerusalem, Israel

Jerusalem, Israel

Location

MeSH Terms

Conditions

Myopathies, Nemaline

Interventions

Tyrosine

Condition Hierarchy (Ancestors)

Myopathies, Structural, CongenitalMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

Amino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and Proteins

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

July 2, 2013

First Posted

January 14, 2014

Study Start

January 1, 2014

Primary Completion

January 1, 2015

Last Updated

January 14, 2014

Record last verified: 2014-01

Locations

IL(1)