Patient Guide: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

We'll Help You Connect with This Trial

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  • ✓ Get matched to the right study location for you
  • ✓ Connect directly with study coordinators at 20 participating sites
  • ✓ Understand eligibility with our simplified screening tool
  • ✓ Free service - we're here to help you access clinical research
Trial ID: NCTNCT05139316
Status: ACTIVE_NOT_RECRUITING
Condition: Glycogen Storage Disease Type IA
Phase: PHASE3

Find a Study Location Near You

This study is available at 20 research sites. We'll help you connect with the location that's right for you.

Participating sites include:
  • • Orange, California
  • • Aurora, Colorado
  • • Farmington, Connecticut
  • • And 17 more locations - let us help you find the closest one

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A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) - Join Clinical Trial NCTNCT05139316

How to Join This Clinical Trial - NCTNCT05139316

Learn how to participate in this PHASE3 trial studying an investigational therapy for Glycogen Storage Disease Type IA. Current status: ACTIVE_NOT_RECRUITING.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Glycogen Storage Disease Type IA. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied
Glycogen Storage Disease Type IA
Treatment Being Tested
Investigational treatment
Study Phase
PHASE3 - Large-scale efficacy study
Enrollment Status
ACTIVE_NOT_RECRUITING
Study Identifier
NCTNCT05139316 - 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 20 locations. Find a study site near you:

Clinical Research Site

Orange, California 92868 - United States

Status: Contact for availability

Clinical Research Site

Aurora, Colorado 80045 - United States

Status: Contact for availability

Clinical Research Site

Farmington, Connecticut 06030 - United States

Status: Contact for availability

Clinical Research Site

The Bronx, New York 10467 - United States

Status: Contact for availability

Clinical Research Site

Durham, North Carolina 27710 - United States

Status: Contact for availability

Clinical Research Site

Cleveland, Ohio 44195 - United States

Status: Contact for availability

Clinical Research Site

Philadelphia, Pennsylvania 19104 - United States

Status: Contact for availability

Clinical Research Site

Houston, Texas 77030 - United States

Status: Contact for availability

Clinical Research Site

Salt Lake City, Utah 84132 - United States

Status: Contact for availability

Clinical Research Site

Porto Alegre, Rio Grande do Sul 90035-903 - Brazil

Status: Contact for availability

And 10 more locations available. Contact us to find the nearest participating site.

How to Enroll in This Study

To learn more about participating in this PHASE3 clinical trial for Glycogen Storage Disease Type IA:

  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