Treatment of TNNT1-Myopathy With L-Tyrosine.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

See also
Status	Clinical Trial	Phase
Enrolling by invitation	`NCT05099107` - Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment	N/A
Completed	`NCT03728803` - Inspiratory Muscle Training in Nemaline Myopathy	N/A
Not yet recruiting	`NCT06157268` - The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.
Recruiting	`NCT00272883` - Molecular and Genetic Studies of Congenital Myopathies

See also

Status

Clinical Trial

Phase

Enrolling by invitation

NCT05099107 - Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment

N/A

Completed

NCT03728803 - Inspiratory Muscle Training in Nemaline Myopathy

N/A

Not yet recruiting

NCT06157268 - The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.

Recruiting

NCT00272883 - Molecular and Genetic Studies of Congenital Myopathies

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT02035501
Study type	Interventional
Source	Hadassah Medical Organization
Contact
Status	Enrolling by invitation
Phase	Phase 2
Start date	January 2014

Treatment of TNNT1-Myopathy With L-Tyrosine.

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms