Other Clinical Trial - Gene Delivery Clinical Trial of SRP-9003

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Limb-Girdle Muscular Dystrophy, Type 2E Clinical Trial

Official title:
A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

See also
	Status	Clinical Trial	Phase
	Recruiting	`NCT03492346` - Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
	Recruiting	`NCT03488784` - Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

See also

Status

Clinical Trial

Phase

Recruiting

NCT03492346 - Limb Girdle Muscular Dystrophy Type 2E Recruitment Study

Recruiting

NCT03488784 - Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

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	NCT03652259
Study type	Interventional
Source	Sarepta Therapeutics, Inc.
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	October 27, 2018
Completion date	February 14, 2027

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms