Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia.
Husain Attarwala, Matthew Lumley, Min Liang...
https://pubmed.ncbi.nlm.nih.gov/36577040Actively Recruiting
Phase 1
Phase 2
All Genders
ID04159103
Open-Label Phase 1/2 Study to Evaluate Safety and Pharmacodynamics of mRNA-3927 in Participants With Propionic Acidemia
Led by ModernaTX, Inc. · Updated on 2026-01-22
77
Participants Needed
35
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying mRNA-3927, a biological treatment, in people with propionic acidemia (PA), a rare genetic condition. This Phase 1/2 open-label study aims to evaluate the safety, tolerability, and how the drug works in the body by measuring biomarkers. The study is divided into three parts: Part 1 focuses on finding the right dose for participants aged 1 year and older, Part 2 assesses the treatment's effects in participants aged 1 year and older, and Part 3 studies infants under 1 year old.
CONDITIONS
Brief Title
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Who Can Participate
All Genders
Eligibility Criteria
You may qualify if you...
- Participants aged 1 year or older with a confirmed diagnosis of propionic acidemia by genetic testing
- Participants aged 8 years or older if among the first two enrolled in Part 1
- Participants aged 1 year or older enrolling after the first two participants in Part 1
- For Part 2, at least one metabolic decompensation event documented in the past 12 months
- Infants under 1 year identified by newborn screening or suspected PA with metabolic symptoms or family history
- Infants in NICU must be at least 37 weeks gestational age without interfering conditions
- Infants must weigh at least 3 kilograms at screening
- At least one documented PA-related event before screening such as metabolic deterioration signs or lab abnormalities
You will not qualify if you...
- Any clinically significant laboratory abnormalities that could affect study participation
- Estimated glomerular filtration rate less than 30 mL/min/1.73 m² for those on chronic dialysis
- History or planned organ transplantation during the study
- Corrected QT interval greater than 480 milliseconds
- Grade 3 or 4 heart failure by accepted classifications
- Pregnancy or breastfeeding
- Other significant medical conditions that could affect safety or study results in the investigator's opinion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Predosing Observational Period
Duration - Up to 12 months
Participants are observed before starting treatment to collect baseline data.
Visits as needed for baseline assessments
Dose Optimization Treatment
Duration - Up to approximately 20 to 30 weeks depending on dosing interval
Participants aged 1 year or older receive single doses of mRNA-3927 by intravenous infusion every 2 or 3 weeks for up to 10 doses to determine the optimal dose and dosing interval.
Up to 10 IV infusion visits every 2 or 3 weeks
Pivotal Treatment
Duration - Up to approximately 12 months
Participants aged 1 year or older receive the identified optimal dose of mRNA-3927 by intravenous infusion every 2 weeks for up to 26 doses or approximately 12 months to evaluate safety and efficacy.
Up to 26 IV infusion visits every 2 weeks
Infant Treatment
Duration - Up to approximately 12 months
Infants under 1 year receive the identified optimal dose of mRNA-3927 by intravenous infusion every 2 weeks for up to 26 doses or approximately 12 months to evaluate safety and efficacy.
Up to 26 IV infusion visits every 2 weeks
Follow-up
Duration - Up to 37 weeks after last dose
Participants are monitored after treatment ends to assess safety and long-term outcomes.
Visits as scheduled for safety and outcome assessments
Trial Site Locations
Total: 35 locations
1
UCSD Altman Clinical and Transalational Research Institute Building
Los Angeles, California, United States, 90027
Not Yet Recruiting
2
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
Actively Recruiting
3
Lucile Packard Children's Hospital Stanford
Stanford, California, United States, 94304
Actively Recruiting
4
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Not Yet Recruiting
5
University of South Florida - 12901 Bruce B Downs
Tampa, Florida, United States, 33606-3603
Not Yet Recruiting
6
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit
Baltimore, Maryland, United States, 21287
Completed
8
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Completed
9
University of Michigan Hospitals
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
10
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, New York, United States, 10029
Actively Recruiting
11
Duke University Medical System (Duke Health)
Durham, North Carolina, United States, 27710
Actively Recruiting
12
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Completed
13
University Hospitals Cleveland Medical Center - 11100 Euclid Ave
Cleveland, Ohio, United States, 44106-2624
Active, Not Recruiting
14
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
15
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
16
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada, T6G 2R7
Actively Recruiting
17
Hospital For Sick Children
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
18
CHU de Marseille - Hôpital de la Timone
Marseille, France, 13005
Active, Not Recruiting
19
Hôpital Necker - Enfants Malades
Paris, France, 75019
Actively Recruiting
20
Fujita Health University Hospital
Toyoake-shi, Aichi-ken, Japan, 470-1192
Actively Recruiting
21
Tohoku University Hospital
Sendai, Miyagi, Japan, 980-8574
Actively Recruiting
22
National Center for Child Health and Development
Tokyo, Japan, 113-8519
Active, Not Recruiting
23
Erasmus MC
Rotterdam, South Holland, Netherlands, 3015 GD
Actively Recruiting
24
Universitair Medisch Centrum Utrecht - PPDS
Utrecht, Netherlands, 3584 CX
Active, Not Recruiting
25
King Faisal Specialist Hospital & Research Center - Riyadh
Riyadh, Ar Riya, Saudi Arabia, 11211
Active, Not Recruiting
26
King Fahad Medical City
Riyadh, Ar Riya, Saudi Arabia, 11564
Not Yet Recruiting
27
King Abdullah Children's Specialist Hospital
Riyadh, Ar Riya, Saudi Arabia, 14611
Not Yet Recruiting
28
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona, Spain, 8950
Active, Not Recruiting
29
Hospital Universitario Cruces
Barakaldo, Biscay, Spain, 48903
Actively Recruiting
30
Hospital Universitario 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
31
Hospital Universitario Virgen del Rocio - PPDS
Seville, Spain, 41013
Active, Not Recruiting
32
University Hospital Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2TH
Actively Recruiting
33
Birmingham Children's Hospital
Birmingham, United Kingdom, B4 6NH
Completed
34
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
35
Willink Biochemical Genetics Unit - PPDS
Manchester, United Kingdom, M13 9WL
Actively Recruiting
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Research Team
M
Moderna WeCare Team
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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