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New treatment option for Propionic Acidemia
Official title Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
ClinicalTrials.gov ID: NCT04159103
What this study is testing
What is mRNA-3927?
mRNA-3927 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for propionic acidemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join if
- Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
- ≥ 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 :
You likely can't join if
- Participants of all ages are excluded from the study if during Screening any of the following criteria apply:
- Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical...
- Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic...
- 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.
See the full eligibility criteria
- Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
- ≥ 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...
- 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...
- 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...
- 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.
- Participants of all ages are excluded from the study if during Screening any of the following criteria apply:
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Stanford, California, United States
- Miami, Florida, United States
- Tampa, Florida, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- New York, New York, United States
- Durham, North Carolina, United States
- Cincinnati, Ohio, United States
- Cleveland, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Edmonton, Alberta, Canada
- Toronto, Ontario, Canada
- Marseille, France
- Paris, France
- Toyoake-shi, Aichi-ken, Japan
- Sendai, Miyagi, Japan
+ 11 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Stanford, California, United States; Miami, Florida, United States; Tampa, Florida, United States; Chicago, Illinois, United States; Baltimore, Maryland, United States and 25 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.