Patient Guide: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

We'll Help You Connect with This Trial

Quri.ai is a free clinical trial matching service. We help patients like you connect directly with research sites conducting this study.

  • ✓ Get matched to the right study location for you
  • ✓ Connect directly with study coordinators at 5 participating sites
  • ✓ Understand eligibility with our simplified screening tool
  • ✓ Free service - we're here to help you access clinical research
Trial ID: NCTNCT02716246
Status: 🟢 Enrolling Now
Condition: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Phase: PHASE2, PHASE3

Find a Study Location Near You

This study is available at 5 research sites. We'll help you connect with the location that's right for you.

Participating sites include:
  • • Columbus, Ohio
  • • Pittsburgh, Pennsylvania
  • • North Adelaide, South Australia
  • • And 2 more locations - let us help you find the closest one

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Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH - Join Clinical Trial NCTNCT02716246

How to Join This Clinical Trial - NCTNCT02716246

Learn how to participate in this PHASE2, PHASE3 trial studying an investigational therapy for MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III. This study is currently enrolling participants.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Treatment Being Tested
Investigational treatment
Study Phase
PHASE2, PHASE3 - Large-scale efficacy study
Enrollment Status
Currently enrolling participants
Study Identifier
NCTNCT02716246 - ClinicalTrials.gov Identifier
Sponsored By
Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

  • Initial screening to determine eligibility
  • Regular study visits and health assessments
  • Receiving the study treatment or placebo
  • Medical monitoring and follow-up care
  • Contributing to medical research that may help others
  • Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

  • Close medical monitoring by healthcare professionals
  • Access to potential new treatments
  • Compensation for time and travel (varies by study)
  • No-cost study-related medical care
  • The opportunity to help advance medical knowledge

Where Is This Clinical Trial Located?

This study is enrolling participants at 5 locations. Find a study site near you:

Clinical Research Site

Columbus, Ohio 43205 - United States

Status: COMPLETED

Clinical Research Site

Pittsburgh, Pennsylvania 15224 - United States

Status: COMPLETED

Clinical Research Site

North Adelaide, South Australia 5006 - Australia

Status: COMPLETED

Clinical Research Site

Barcelona, Barcelona 08035 - Spain

Status: RECRUITING

Clinical Research Site

Santiago de Compostela, 15706 - Spain

Status: RECRUITING

How to Enroll in This Study

To learn more about participating in this PHASE2, PHASE3 clinical trial for MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III:

  1. Review the eligibility criteria with your healthcare provider
  2. Contact the study team for a pre-screening interview
  3. Schedule an in-person screening visit if eligible
  4. Review and sign the informed consent form
  5. Begin participation in the clinical trial

Why Choose Quri.ai as Your Clinical Trial Matching Service

Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:

  • Find clinical trials that match your condition
  • Get matched to the right study location near you
  • Connect directly with study coordinators
  • Understand if you qualify with simplified eligibility screening
  • Navigate the enrollment process with personalized support
  • Access new treatments through clinical research participation