Open Label Study of mRNA-3927 in Patients With Propionic Acidemia

Study Purpose

This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3927 in patients 1 year of age and older with propionic acidemia (PA). The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics, and pharmacodynamics of different doses of mRNA-3927 in patients affected by PA as part of the Dose Escalation phase. During the Dose Escalation phase, three dose levels of mRNA-3927 are planned to be investigated in this study among patients with PA: low dose, mid dose, and high dose. An additional three cohorts to evaluate other dose levels and/or dosing intervals may be considered jointly by the independent SMC and the Sponsor. Upon establishment of a dose with acceptable safety and pharmacodynamic activity, additional patients will be enrolled in a Dose Expansion phase to allow for further characterization of the safety and pharmacodynamics of mRNA-3927. Patients in both phases of study will participate in a pre-dosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

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.

Interventional
Eligible Ages 1 Year and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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.
- Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association

Trial Details

Trial ID:

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.

NCT04159103
Phase

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
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

ModernaTX, Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Not yet recruiting
Countries
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Propionic Acidemia
Arms & Interventions

Arms

Experimental: Dose Escalation Phase: Dose Level 1

mRNA-3927

Experimental: Dose Escalation Phase: Dose Level 2

mRNA-3927

Experimental: Dose Escalation Phase: Dose Level 3

mRNA-3927

Experimental: Experimental: Dose Expansion Phase

mRNA-3927

Interventions

Biological: - mRNA-3927

mRNA-3927 dispersion for intravenous (IV) infusion

Contact Information

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.