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A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927

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: NCT05130437
Recruitment Status : Recruiting
First Posted : November 23, 2021
Last Update Posted : March 16, 2022
Sponsor:
Information provided by (Responsible Party):
ModernaTX, Inc.

Brief Summary:
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).

Condition or disease Intervention/treatment Phase
Propionic Acidemia Biological: mRNA-3927 Phase 1 Phase 2

Detailed Description:

The study will assess long-term safety and clinical activity of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT) Visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.

The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (up to 2 years after the last dose of study drug). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
Actual Study Start Date : November 9, 2021
Estimated Primary Completion Date : December 4, 2029
Estimated Study Completion Date : December 4, 2031


Arm Intervention/treatment
Experimental: mRNA-3927
Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.
Biological: mRNA-3927
mRNA-3927 dispersion for IV infusion




Primary Outcome Measures :
  1. Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Baseline up to End of Study Visit (up to 8 years) ]

Secondary Outcome Measures :
  1. Change From Baseline in Plasma 2-Methylcitrate (2-MC) Levels [ Time Frame: Baseline, End of Study (up to 8 years) ]
  2. Change From Baseline in Plasma 3-Hydroxypropionic Acid (3-HP) Levels [ Time Frame: Baseline, End of Study (up to 8 years) ]
  3. Pharmacokinetic (PK): Propionyl-CoA Carboxylase (PCC) Subunit α (PCCA) and Propionyl-CoA Carboxylase Subunit β (PCCB) mRNA (Serum) and SM-86 (Plasma) Levels [ Time Frame: Predose and End of Infusion up to 8 years postdose ]
  4. Number of Clinically Significant Events [ Time Frame: Baseline up to 8 years ]
    Clinically significant events include hospitalization, emergency room visits, emergency interventions outside of healthcare settings, and complications of cardiomyopathy treated outside of hospital.

  5. Number of Metabolic Decompensation Events (MDEs) [ Time Frame: Baseline up to 8 years ]
  6. Number of Healthcare Resource Utilization Visits [ Time Frame: Baseline up to 8 years ]
  7. Number of Days Participants Missed School and Workdays [ Time Frame: Baseline up to 8 years ]
  8. Number of Anti-Polyethylene Glycol Antibodies [ Time Frame: Month 6 up to 8 years ]
  9. Number of Anti-PCC Antibodies [ Time Frame: Month 6 up to 8 years ]
  10. Change From Baseline in Pediatric Quality-of-Life Inventory (PedsQL) [ Time Frame: Baseline, End of Study (up to 8 years) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participated in Study mRNA-3927-P101.
  • Completed the EOT Visit in Study mRNA-3927-P101 within 10 days of first dose of mRNA-3927 in current study.

Exclusion Criteria:

  • Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
  • Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
  • History of liver and/or kidney transplant.

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): NCT05130437


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, Michigan
Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Canada, Ontario
Hospital For Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
United Kingdom
Birmingham Children's Hospital Recruiting
Birmingham, United Kingdom, B4 6NH
Great Ormond Street Hospital (GOSH) Recruiting
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
ModernaTX, Inc.
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Responsible Party: ModernaTX, Inc.
ClinicalTrials.gov Identifier: NCT05130437    
Other Study ID Numbers: mRNA-3927-P101-EXT
First Posted: November 23, 2021    Key Record Dates
Last Update Posted: March 16, 2022
Last Verified: March 2022

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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.:
Moderna
mRNA-3927
Propionic Aciduria
Metabolism, Inborn Errors
Genetic Diseases
Inborn Amino Acid Metabolism, Inborn Errors
Acidosis
Acid-Base Imbalance
Metabolic Diseases
Organic Acidemias
mRNA
Additional relevant MeSH terms:
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Propionic Acidemia
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases