Top
Mucopolysaccharidosis Type IIIA - Healing Genes
1302
post-template-default,single,single-post,postid-1302,single-format-standard,mkdf-bmi-calculator-1.0,mkd-core-1.1.1,tribe-no-js,wellspring-ver-1.8,mkdf-smooth-page-transitions,mkdf-ajax,mkdf-grid-1300,mkdf-blog-installed,mkdf-header-standard,mkdf-no-behavior,mkdf-default-mobile-header,mkdf-sticky-up-mobile-header,mkdf-dropdown-slide-from-bottom,mkdf-full-width-wide-menu,mkdf-search-dropdown,elementor-default,elementor-template-full-width,elementor-kit-3486,elementor-page-2540

Mucopolysaccharidosis Type IIIA

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA


Phase 2/3

DESCRIPTION:
Researchers in TX, NY. MN, and CA seek patients with Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome to trial an investigatory gene therapy, LYS-SAF302, 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 intracerebral (into the brain, guided by MRI) infusion of the investigatory gene therapy, 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
  • Not have past use of SGSH enzyme replacement therapy for a period exceeding 3 months. If so, a period of at least 2 months without its use is required prior to screening.

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 sites include:
CHOC Children’s in Orange, CA. Map.
University of Minnesota in Minneapolis, MA. Map.
Weill Cornell Medical College in New York, NY. Map.
Baylor College of Medicine / Texas Children’s Hospital in Houston, TX. Map.
 
Contacts:
Lysogene trial inquiries  |  +33 (0) 1 41 43 03 90 (international)  |  [email protected]
 
SPONSOR INFORMATION:
Lysogene
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT03612869

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader