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Limb-Girdle Muscular Dystrophy (LGMD2E) (Beta-sarcoglycan Deficiency) - Healing Genes
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Limb-Girdle Muscular Dystrophy (LGMD2E) (Beta-sarcoglycan Deficiency)

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Gene Transfer Clinical Trial for LGMD2E (Beta-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB

Phase I/IIa Gene Transfer Clinical Trial for LGMD2E (β-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB Administered by Systemic Perfusion


Phase 1 / 2

DESCRIPTION:

Doctors at Nationwide Children’s Hospital in Columbus, OH are recruiting patients with Limb-Girdle Muscular Dystrophy to trial therapeutic gene therapy. The therapy uses a harmless virus to deliver a deliver a functional copy of the β-sarcoglycan gene to the central nervous system, to correct its deficiency in LGMD2E patients. The researchers hope this treatment will improve physical abilities and muscle tissue strengthening.

Study participants will receive the investigatory treatment via a single IV infusion, with a section of patients receiving a placebo. Follow up will continue for approximately 3 years.


PATIENT MUST:

  • Be 4 to 15 years of age
  • Have confirmed biallelic SGCB gene mutation
  • Not be pregnant or test positive for HIV, Hep C, or Hep B

THE STUDY INVOLVES:

  1. Screening to confirm acceptance to the study.
  2. Intravenous injection of the therapy, under observation.
  3. Lab and physical assessment follow up will continue at 1 month, then yearly for 3 years.

LOCATIONS & CONTACTS:

The trial will take place at the Nationwide Children’s Hospital in Columbus, OH. Map.

Contact:
Amanda Nicholl, RN  |  614-355-2765  |  [email protected]

SPONSORS:
Jerry R. Mendell
Myonexus Therapeutics

 

Or go online:
https://clinicaltrials.gov/ct2/show/NCT03652259

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