RECRUITING

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

Conditions

Propionic Acidemia mRNA-3927 Propionic Aciduria Metabolism, Inborn Errors Genetic Diseases Inborn Amino Acid Metabolism, Inborn Errors Acidosis Acid-Base Imbalance Metabolic Diseases Organic Acidemias Moderna mRNA

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).

Official Title

A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia

Quick Facts

Study Start:2021-04-15

Study Completion:2026-01-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04159103

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. * 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.	* 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.

Inclusion Criteria

Exclusion 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.
* 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.

* 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.

Contacts and Locations

Study Contact

Moderna WeCare Team

CONTACT

1-866-663-3762

WeCareClinicalTrials@modernatx.com

Study Locations (Sites)

UCSD Altman Clinical and Transalational Research Institute Building

Los Angeles, California, 90027

United States

Ronald Reagan UCLA Medical Center

Los Angeles, California, 90095

United States

Lucile Packard Children's Hospital Stanford

Stanford, California, 94304

United States

Nicklaus Children's Hospital

Miami, Florida, 33155

United States

University of South Florida - 12901 Bruce B Downs

Tampa, Florida, 33606-3603

United States

Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit

Baltimore, Maryland, 21287

United States

Boston Children's Hospital

Boston, Massachusetts, 02115

United States

University of Michigan Hospitals

Ann Arbor, Michigan, 48109

United States

Icahn School of Medicine at Mount Sinai - Clinical Research Unit

New York, New York, 10029

United States

Duke University Medical System (Duke Health)

Durham, North Carolina, 27710

United States

Ann and Robert H Lurie Childrens Hospital of Chicago

Cincinnati, Ohio, 45229-3026

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

United States

University Hospitals Cleveland Medical Center - 11100 Euclid Ave

Cleveland, Ohio, 44106-2624

United States

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104

United States

Texas Children's Hospital

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: ModernaTX, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-04-15

Study Completion Date2026-01-31

Study Record Updates

Study Start Date2021-04-15

Study Completion Date2026-01-31

Terms related to this study

Keywords Provided by Researchers

mRNA-3927
Propionic Aciduria
Metabolism, Inborn Errors
Genetic Diseases
Inborn Amino Acid Metabolism, Inborn Errors
Acidosis
Acid-Base Imbalance
Metabolic Diseases
Organic Acidemias
Moderna
mRNA

Additional Relevant MeSH Terms

Propionic Acidemia