Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle

Clinical Trial Description

n/a

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Gamma-sarcoglycanopathy
Limb Girdle Muscular Dystrophy Type 2C
Muscular Dystrophies

See also
	Status	Clinical Trial	Phase
	Not yet recruiting	`NCT06210672` - Natural History Study in Patients With LGMDR5/2c

See also

Status

Clinical Trial

Phase

Not yet recruiting

NCT06210672 - Natural History Study in Patients With LGMDR5/2c

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	NCT01344798
Study type	Interventional
Source	Genethon
Contact
Status	Completed
Phase	Phase 1
Start date	November 2006
Completion date	June 2010

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms