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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05095727
Recruitment Status : Recruiting
First Posted : October 27, 2021
Last Update Posted : March 2, 2023
Sponsor:
Information provided by (Responsible Party):
ModernaTX, Inc.

Study Description
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Brief Summary:
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in participants with GSD1a.

Condition or disease Intervention/treatment Phase
Glycogen Storage Disease Drug: mRNA-3745 Phase 1

Detailed Description:
This is a dose escalation study in adult participants with GSD1a to determine the safety and tolerability of mRNA-3745 and characterize the pharmacokinetic (PK) and pharmacodynamic (PD) response following IV administration of mRNA-3745.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a)
Actual Study Start Date : June 1, 2022
Estimated Primary Completion Date : January 19, 2024
Estimated Study Completion Date : January 19, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: mRNA-3745
Participants will receive a single IV dose of mRNA-3745 on Day 1. Participants that are/have been enrolled in the study and receive an administration of mRNA-3745 may receive one or more doses in subsequent cohorts (intrapatient dosing). The next dose must occur at least 21 days after the previous one.
 Drug: mRNA-3745
Sterile frozen liquid dispersion for injection


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 52 ]

Secondary Outcome Measures :
  1. Number of Participants With No Hypoglycemic Events for up to 12 Hours During Fasting Challenges [ Time Frame: Baseline through Week 52 ]
    Hypoglycemia is defined as blood glucose <60 milligrams (mg)/deciliter (dL) (3.3 millimoles [mmol]/liter [L]).

  2. Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges [ Time Frame: Baseline through Week 52 ]
  3. Change From Baseline in Time to Hypoglycemia During Fasting Challenges [ Time Frame: Baseline through Week 52 ]
  4. Change From Baseline in Maximum Effect (Emax) During Fasting Challenges [ Time Frame: Baseline through Week 52 ]
  5. Maximum Observed Serum Concentration (Cmax) of Messenger Ribonucleic Acid (mRNA) and Lipid Nanoparticle (LNP) [ Time Frame: Pre-infusion, at mid-infusion, and at the end of infusion (EOI) on Day 1 up to Week 52 ]
  6. Time to Reach Cmax (Tmax) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  7. Area Under the Serum Concentration-Time Curve From Time 0 to the Time of the Last Measurable Serum Concentration (AUC0-t) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  8. Terminal Elimination Half-Life (t1/2) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  9. Clearance (CL) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  10. Volume of Distribution at Steady State (Vss) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  11. Change From Baseline in Metabolic Biomarkers [ Time Frame: Baseline through Week 52 ]
  12. Number of Participants With No Hypoglycemic Events for up to 8 Hours During Fasting Challenges [ Time Frame: Baseline through Week 52 ]
    Hypoglycemia is defined as blood glucose <60 milligrams (mg)/deciliter (dL) (3.3 millimoles [mmol]/liter [L]).

  13. Maximum Observed Serum Concentration (Cmax) of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at the end of infusion (EOI) on Day 1 up to Week 52 ]
  14. Time to Cmax of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]
  15. Volume of Distribution at Steady State of mRNA and LNP [ Time Frame: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Documented GSD1a with confirmation by genetic testing
  • Documented history of ?1 hypoglycemic event with blood glucose <60 milligrams/deciliter (mg/dL) (<3.3 millimoles/liter [mmol/L]) and symptoms of hypoglycemia in the absence of acute illness, with at least one such event in the 4 weeks before signing the Informed Consent.

Exclusion Criteria:

  • Liver transplant, including hepatocyte cell therapy/transplant
  • Received gene therapy for GSD1a
  • Presence of liver adenoma >5 centimeters (cm) in size
  • Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenoma, in the previous 2 years.

Note: Additional inclusion/exclusion criteria may apply, per protocol.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05095727


Contacts
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Contact: Moderna Clinical Trials Support Center 1-877-777-7187 clinicaltrials@modernatx.com

Locations
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United States, Connecticut
University of Connecticut Health Center Recruiting
Farmington, Connecticut, United States, 06030-0001
Contact    860-679-6584    shking@uchc.edu   
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032-3822
Contact    212-305-5508    jc688@cumc.columbia.edu   
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27713
Contact    919-681-4026    gretchen.nichting@duke.edu   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact    513-636-4507    laurie.bailey@cchmc.org   
United States, Texas
The University of Texas Health Science Center at Houston Recruiting
Houston, Texas, United States, 77030-1501
Contact    713-500-7098    heather.saavedra@uth.tmc.edu   
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132-0001
Sponsors and Collaborators
ModernaTX, Inc.
More Information
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Publications:

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Responsible Party: ModernaTX, Inc.
ClinicalTrials.gov Identifier: NCT05095727    
Other Study ID Numbers: mRNA-3745-P102
First Posted: October 27, 2021    Key Record Dates
Last Update Posted: March 2, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by ModernaTX, Inc.:
Glycogen storage disease type 1a
GSD1a
Von Gierke disease
Glucose metabolism disorder
 Genetic disorder
Autosomal recessive disorder
messenger RNA
mRNA
Additional relevant MeSH terms:
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Glycogen Storage Disease
Metabolic Diseases
Disease
Pathologic Processes
 Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn