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

Sponsor

ModernaTX, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05130437

Collaborator

(none)

Enrollment

Locations

Arm

120.8

Anticipated Duration (Months)

7.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief 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).

Condition or Disease	Intervention/Treatment	Phase
Propionic Acidemia	Biological: mRNA-3927	Phase 1/Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

36 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 9, 2021

Anticipated Primary Completion Date :

Dec 4, 2029

Anticipated Study Completion Date :

Dec 4, 2031

Arms and Interventions

Arm	Intervention/Treatment
Experimental: mRNA-3927 Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.	Biological: mRNA-3927 mRNA-3927 dispersion for IV infusion

Arm

Intervention/Treatment

Experimental: mRNA-3927

Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.

Biological: mRNA-3927

mRNA-3927 dispersion for IV infusion

Outcome Measures

Primary Outcome Measures

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [Baseline up to End of Study Visit (up to 8 years)]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Michigan	Ann Arbor	Michigan	United States	48109
2	Duke University Medical Center	Durham	North Carolina	United States	27710
3	Hospital For Sick Children	Toronto	Ontario	Canada	M5G 1X8
4	Birmingham Children's Hospital	Birmingham		United Kingdom	B4 6NH
5	Great Ormond Street Hospital (GOSH)	London		United Kingdom	WC1N 3JH