Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||1 Year and Over|
- - Participant must be ≥ 1 year of age at the time of consent/assent (Inclusion of the
first three participants entering the study at the Dose Escalation stage will be
restricted to individuals age ≥ 8 years)
- Confirmed diagnosis of propionic acidemia based on diagnosis by molecular genetic
testing (PCCA and/or PCCB mutations)
Exclusion Criteria:- Estimated glomerular filtration rate <30 mL/min/1.73 m2, as estimated by Schwartz formula (Schwartz et al 2012); or participants who receive chronic dialysis.
- - History of organ transplantation or planned organ transplantation during the period of study participation.
- - QTc >480 ms using Bazett's correction.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
|Phase 1/Phase 2|
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Principal Investigator Affiliation||N/A|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Overall Status||Not yet recruiting|
The disease, disorder, syndrome, illness, or injury that is being studied.
Experimental: Dose Escalation Phase: Dose Level 1
Experimental: Dose Escalation Phase: Dose Level 2
Experimental: Dose Escalation Phase: Dose Level 3
Experimental: Experimental: Dose Expansion Phase
Biological: - mRNA-3927
mRNA-3927 dispersion for intravenous (IV) infusion
This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:
For additional contact information, you can also visit the trial on clinicaltrials.gov.