We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05295433
Recruitment Status : Recruiting
First Posted : March 25, 2022
Last Update Posted : December 22, 2022
Sponsor:
Information provided by (Responsible Party):
ModernaTX, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The primary objective of this study is to evaluate the long-term safety of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in Study mRNA-3705-P101 (NCT04899310).

Condition or disease Intervention/treatment Phase
Methylmalonic Acidemia Drug: mRNA-3705 Phase 1 Phase 2

Detailed Description:

Participants with isolated MMA due to MUT deficiency who were previously enrolled in the mRNA-3705-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).

Treatment Period will continue unless one of the following occurs: mRNA-3705 receives marketing approval and reimbursement in the country of origin of the participant, the participant discontinues study drug, the participant is no longer receiving clinical benefit (in the opinion of the Investigator), or Sponsor discontinues the development of mRNA-3705.

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 33 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-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study
Actual Study Start Date : March 8, 2022
Estimated Primary Completion Date : April 1, 2032
Estimated Study Completion Date : April 2, 2034

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: mRNA-3705
Participants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks [q2W], every 3 weeks [q3W], or every 4 weeks [q4W]) last received in the mRNA-3705-P101 study, unless the Sponsor recommends modification.
 Drug: mRNA-3705
A sterile liquid for injection
Other Names:
  • modified mRNA encoding human
  • methylmalonyl-coenzyme A mutase


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (AEs) [ Time Frame: Baseline up to follow-up period (up to 8 years) ]

Secondary Outcome Measures :
  1. Change in Methylmalonic Acid and 2-Methylcitric Acid (2-MC) Levels (Primary Biomarkers) From Baseline up to 8 Years [ Time Frame: Baseline, Year 8 ]
  2. Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels [ Time Frame: Baseline up to 6 years ]
  3. Number of Clinically Significant Events [ Time Frame: Baseline up to 8 years ]
    Clinically significant event is defined as a composite of the following: - Hospitalization (excluding hospitalizations for chronic diseases not related to MMA or elective hospitalizations for conditions not related to MMA); Emergency room visits; and Emergency interventions outside of healthcare settings to prevent an metabolic decompensation event (MDE) (sick-day diets and fluid resuscitation at home).

  4. Number of MDEs [ Time Frame: Baseline up to 8 years ]
  5. Number of Healthcare Resource Utilization Visits [ Time Frame: Baseline up to 8 years ]
  6. Change in Disease Impact on Missed School and Workdays From Baseline up to 8 Years [ Time Frame: Baseline, Year 8 ]
  7. Number of Anti-Polyethylene Glycol (PEG) and Anti-hMUT Antibodies [ Time Frame: Baseline up to 8 years ]
  8. Change in Health-Related Quality of Life (HRQoL) as Measured Using the Pediatric Quality of Life Inventory (PedsQL™) at Month 3 up to 8 Years [ Time Frame: Month 3, Year 8 ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completed the mRNA-3705-P101 assigned dose regimen treatment time period or is eligible for early transition to this study due to missing more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during the mRNA-3705-P101 study.
  • Completed the EOT Visit in Study mRNA-3705-P101 within 10 days of first dose of mRNA-3705 in the current study.

Exclusion Criteria:

  • Not expected to receive clinical benefit from continued mRNA-3705 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.

NOTE: Other inclusion and exclusion criteria may apply.

Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05295433


Contacts
Layout table for location contacts
Contact: Moderna Clinical Trials Support Center 1-877-777-7187 clinicaltrials@modernatx.com

Locations
Layout table for location information
Canada, Ontario
Hospital For Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
United Kingdom
Birmingham Children's Hospital NHS Foundation Trust Recruiting
Birmingham, United Kingdom, B4 6NH
Sponsors and Collaborators
ModernaTX, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: ModernaTX, Inc.
ClinicalTrials.gov Identifier: NCT05295433    
Other Study ID Numbers: mRNA-3705-P101-EXT
2021-000446-17 ( EudraCT Number )
First Posted: March 25, 2022    Key Record Dates
Last Update Posted: December 22, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by ModernaTX, Inc.:
Isolated Methylmalonic acidemia
Isolated methylmalonic aciduria
Elevated methylmalonic acid (MMA)
Metabolism, Inborn Errors
 Genetic Diseases
Moderna
mRNA
mRNA-3705