A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927

Propionic Acidemia Clinical Trial

Official title:

A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05130437
• Other study ID #: mRNA-3927-P101-EXT
• Secondary ID: 2020-005009-14
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: November 9, 2021
• Est. completion date: December 4, 2031

Study information

• Verified date: February 2024
• Source: ModernaTX, Inc.
• Contact: Moderna Clinical Trials Support Center
• Phone: 1-877-777-7187
• Email: clinicaltrials[@]modernatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).

Description:

The study will assess long-term safety and clinical activity of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT) Visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.

The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (up to 2 years after the last dose of study drug). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. completion date: December 4, 2031
• Est. primary completion date: December 4, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Inclusion Criteria:

• Participated in Study mRNA-3927-P101.

• Completed the EOT Visit in Study mRNA-3927-P101 within 10 days of first dose of mRNA-3927 in current study.

Exclusion Criteria:

• Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.

• Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.

• History of liver and/or kidney transplant.

Related Conditions & MeSH terms

• Propionic Acidemia

Intervention

Biological:
• mRNA-3927
mRNA-3927 dispersion for IV infusion

Locations

Country	Name	City	State
Canada	Hospital For Sick Children	Toronto	Ontario
United Kingdom	University Hospital Birmingham NHS Foundation Trust	Birmingham
United Kingdom	Great Ormond Street Hospital (GOSH)	London
United States	Michigan	Ann Arbor	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
ModernaTX, Inc.

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Treatment-Emergent Adverse Events (TEAEs)		Baseline up to End of Study Visit (up to 8 years)
Secondary	Change From Baseline in Plasma 2-Methylcitrate (2-MC) Levels		Baseline, End of Study (up to 8 years)
Secondary	Change From Baseline in Plasma 3-Hydroxypropionic Acid (3-HP) Levels		Baseline, End of Study (up to 8 years)
Secondary	Pharmacokinetic (PK): Propionyl-CoA Carboxylase (PCC) Subunit a (PCCA) and Propionyl-CoA Carboxylase Subunit ß (PCCB) mRNA (Serum) and SM-86 (Plasma) Levels		Predose and End of Infusion up to 8 years postdose
Secondary	Number of Clinically Significant Events	Clinically significant events include hospitalization, emergency room visits, emergency interventions outside of healthcare settings, and complications of cardiomyopathy treated outside of hospital.	Baseline up to 8 years
Secondary	Number of Metabolic Decompensation Events (MDEs)		Baseline up to 8 years
Secondary	Number of Healthcare Resource Utilization Visits		Baseline up to 8 years
Secondary	Number of Days Participants Missed School and Workdays		Baseline up to 8 years
Secondary	Number of Anti-Polyethylene Glycol Antibodies		Month 6 up to 8 years
Secondary	Number of Anti-PCC Antibodies		Month 6 up to 8 years
Secondary	Change From Baseline in Pediatric Quality-of-Life Inventory (PedsQL)		Baseline, End of Study (up to 8 years)