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New mRNA therapy aims to control blood sugar in rare genetic disease

NCT ID NCT05095727

Disease control Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests an mRNA drug called mRNA-3745 in 15 adults and children with glycogen storage disease type 1a (GSD1a), a rare genetic condition that causes dangerously low blood sugar. The drug is given by IV infusion and aims to help the body produce a missing enzyme. The main goal is to check safety and tolerability, with a secondary focus on whether it prevents hypoglycemia during fasting challenges.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mRNA-3745 (a messenger RNA drug given by IV infusion)
What this could lead to
If successful, this could lead to a new treatment that helps control blood sugar levels and reduces the risk of dangerously low blood sugar in people with GSD1a.
What could go wrong
This is an early-phase trial with only 15 participants, so results may not apply to everyone. The treatment is new and may cause side effects or not work as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

15 people

The number who actually took part.

Started

Jun 2022

Expected to finish

Nov 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (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.

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Conditions

The condition(s) this trial relates to.

disorder of glycogen metabolism glucose metabolism disease glycogen storage disease due to glucose-6-phosphatase deficiency type IA glycogen storage disease I hereditary disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Glycogen storage disease

Contacts and locations

Locations

  • AP-HP - Hôpital Antoine Béclère

    Clamart, 92140, France

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHRU Tours - Hopital Clocheville

    Tours, 37000, France

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27713, United States

  • Hospital Regional Universitario de Malaga

    Málaga, 29011, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28026, Spain

  • Instytut Pomnik Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • Stollery Children's Hospital University of Alberta

    Edmonton, Alberta, T6G 2R7, Canada

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030-1501, United States

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030-0001, United States

  • University of Utah

    Salt Lake City, Utah, 84132-0001, United States

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