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Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome - Healing Genes
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Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome

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Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA


Phase 1 / 2

DESCRIPTION:

Doctors at the Nationwide Children’s Hospital in Columbus, OH, seek patients with Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome to trial an investigatory gene therapy, a virus engineered to transfer a functional SGSH gene to the patient, so that their bodies can process heparan sulfate normally.

Participants will undergo a single IV infusion of the investigatory gene therapy, along with a course of steroids, then be assessed at 6, 12, 18, and 24 months.


PATIENT MUST:

  • Be 6 months of age or older
  • Have confirmed diagnosis of MPS IIIA
  • Meet specific genetic screening criteria regarding their type of MPS mutation

THE STUDY INVOLVES:

  1. Screening to confirm eligibility.
  2. Participants receive a single infusion of the investigatory gene therapy, as well as a steroid prescription.
  3. Lab and physical assessments will continue 24 months at 6-month intervals.

LOCATIONS AND CONTACTS:

The study site is at the Nationwide Children’s Hospital in Columbus, OH. Map.

Contacts:
Juan Ruiz, MD, PhD  |  +34 685895069 (international)   |  [email protected]

SPONSOR INFORMATION:
Abeona Therapeutics, Inc

Or go online:
https://clinicaltrials.gov/ct2/show/NCT02716246

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