Treatment of TNNT1-Myopathy With L-Tyrosine.

Hadassah Medical Center

Status and phase: Unknown; Phase 2

Conditions: Nemaline Myopathy

Treatments: Drug: Placebo; Drug: L-Tyrosine

Study type: Interventional

Funder types: Other

Identifiers

NCT02035501

SimonEdvardson1

Details and patient eligibility

About

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

Enrollment: 10 estimated patients

Sex: All

Ages: Under 20 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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10 participants in 2 patient groups, including a placebo group

L-Tyrosine

Active Comparator group

Treatment:

Drug: L-Tyrosine

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo