Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
NCT ID: NCT04159103
Last Updated: 2025-10-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
77 participants
INTERVENTIONAL
2021-04-15
2026-01-31
Brief Summary
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Detailed Description
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Upon establishment of a dose with an acceptable safety and PD activity in Part 1 (participants ≥1 year of age), additional participants will be enrolled into the study in Part 2 (participants ≥1 year of age) to allow for determination of the safety, efficacy, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response in infants (\<1 year of age).
Participants in all the phases will participate in a predosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment.
Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Part 1 (Dose Optimization), Part 2 (Pivotal Study), and Part 3 (Infants)
Part 1 (Dose Optimization): Participants (≥1 year of age) will receive single dose of mRNA-3927 by intravenous (IV) infusion every 2 weeks (Q2W) or every 3 weeks (Q3W) for up to 10 doses.
Part 2 (Pivotal Study): Participants (≥1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months. Part 3: Participants (\<1 year of age) will receive single dose of mRNA-3927 (identified during Dose Optimization Phase) by IV infusion Q2W for up to 26 doses or approximately 12 months.
mRNA-3927
mRNA-3927 dispersion for IV infusion
Interventions
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mRNA-3927
mRNA-3927 dispersion for IV infusion
Eligibility Criteria
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Inclusion Criteria
* ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
* ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1.
* Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations).
* Part 2 only: At least one documented MDE in the 12-month period before consent.
Participants \<1 Year of Age :
* Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed.
* For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.
* Body weight ≥3 kilograms (kg) at Screening.
* At least 1 documented PA-related event prior to Screening defined as the following criteria:
* Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR
* Meeting the criteria of MDE definition, OR
* Evidence of laboratory abnormalities as evidenced by at least one of the following:
* Metabolic acidosis with elevated anion gap.
* Acute hyperammonemia.
* Neutropenia or thrombocytopenia.
Exclusion Criteria
* Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study.
* Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis.
* History of organ transplantation or planned organ transplantation during the period of study participation.
* Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction.
* Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
* Pregnant or breastfeeding.
* Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.
ALL
No
Sponsors
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ModernaTX, Inc.
INDUSTRY
Responsible Party
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Locations
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UCSD Altman Clinical and Transalational Research Institute Building
Los Angeles, California, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
Lucile Packard Children's Hospital Stanford
Stanford, California, United States
Nicklaus Children's Hospital
Miami, Florida, United States
University of South Florida - 12901 Bruce B Downs
Tampa, Florida, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan Hospitals
Ann Arbor, Michigan, United States
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, New York, United States
Duke University Medical System (Duke Health)
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center - 11100 Euclid Ave
Cleveland, Ohio, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
Hospital For Sick Children
Toronto, Ontario, Canada
CHU de Marseille - Hôpital de la Timone
Marseille, , France
Hôpital Necker - Enfants Malades
Paris, , France
Fujita Health University Hospital
Toyoake-shi, Aichi-ken, Japan
Tohoku University Hospital
Sendai, Miyagi, Japan
National Center for Child Health and Development
Tokyo, , Japan
Erasmus MC
Rotterdam, South Holland, Netherlands
Universitair Medisch Centrum Utrecht - PPDS
Utrecht, , Netherlands
King Faisal Specialist Hospital & Research Center - Riyadh
Riyadh, Ar Riya, Saudi Arabia
King Fahad Medical City
Riyadh, Ar Riya, Saudi Arabia
King Abdullah Children's Specialist Hospital
Riyadh, Ar Riya, Saudi Arabia
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Cruces
Barakaldo, Biscay, Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, , Spain
University Hospital Birmingham NHS Foundation Trust
Birmingham, , United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Willink Biochemical Genetics Unit - PPDS
Manchester, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Role: primary
Role: primary
Role: primary
References
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Attarwala H, Lumley M, Liang M, Ivaturi V, Senn J. Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia. Nucleic Acid Ther. 2023 Apr;33(2):141-147. doi: 10.1089/nat.2022.0036. Epub 2022 Dec 27.
Other Identifiers
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2022-502910-10-00
Identifier Type: OTHER
Identifier Source: secondary_id
mRNA-3927-P101
Identifier Type: -
Identifier Source: org_study_id
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