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NCT05095727RECRUITINGIRB Ready

A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

Sponsored by ModernaTX, Inc.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 45 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.

Who Can Participate

Inclusion Criteria

Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-ฮฑ (G6PC) mutations by genetic testing.
Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening

Exclusion Criteria

Solid organ transplant
Received gene therapy for GSD1a
Presence of liver adenoma \>5 centimeters (cm) in size
Diagnosis of type 1 or type 2 diabetes mellitus
Presence of liver adenoma with growth of \>2 cm or \>5 newly diagnosed liver adenomas, in the previous 2 years Note: Additional inclusion/exclusion criteria may apply, per protocol.

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

45 participants

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Gender

ALL

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Sponsor

ModernaTX, Inc.

Study Locations

Choose your preferred location or select flexible during enrollment

Unknown

Farmington

Connecticut

Location available
View Farmington location page
Unknown

Boston

Massachusetts

Location available
View Boston location page
Unknown

Durham

North Carolina

Location available
View Durham location page
Unknown

Cincinnati

Ohio

Location available
View Cincinnati location page
Unknown

Philadelphia

Pennsylvania

Location available
View Philadelphia location page
Unknown

Houston

Texas

Location available
View Houston location page
Unknown

Houston

Texas

Location available
View Houston location page
Unknown

Salt Lake City

Utah

Location available
View Salt Lake City location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Glycogen Storage Disease Treatment in Farmington?

Join others in Connecticut exploring innovative treatment options through clinical research

Glycogen Storage Disease Treatment Options in Farmington, Connecticut

If you're searching for Glycogen Storage Disease treatment in Farmington, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Farmington, Boston, Durham and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Glycogen Storage Disease. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Connecticut
Now Enrolling
Up to 45 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Glycogen Storage Disease?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Glycogen Storage Disease

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Glycogen Storage Disease Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05095727. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.