A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
Who is this study for? Patients with isolated methylmalonic acidemia due to methylmalonyl-CoA mutase deficiency
What treatments are being studied? mRNA-3705
Status: Recruiting
Location: See all (17) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, pharmacokinetics, and pharmacodynamics of mRNA-3705.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Healthy Volunteers: f
View:
• Participant has a body weight of ≥11.0 kilograms (kg) at the Screening Visit.
• Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed by molecular genetic testing.
• Participant has a blood vitamin B12 level equal to or above the lower limit of normal (based on laboratory reference range) confirmed in the Screening Period. For those participants found to have an elevated blood vitamin B12 level, the participant may enter if, in the opinion of the Investigator, the cause of the elevation is secondary to B12 supplementation.
• Participant or their legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulations and is willing and able to comply with study-related assessments.
• Sexually active females of childbearing potential and sexually active males of reproductive potential agree to use a highly-effective method of contraception during the study and for 3 months after the last administration of study drug.
• (Part 2 only) At least 1 documented MDE in the 12-month period before consent.
Locations
United States
California
UCLA Medical Center
RECRUITING
Los Angeles
Lucile Packard Children's Hospital at Stanford
RECRUITING
Palo Alto
Pennsylvania
The Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Other Locations
Australia
Royal Children's Hospital Melbourne
WITHDRAWN
Parkville
Children's Hospital at Westmead
WITHDRAWN
Westmead
Canada
Stollery Children's Hospital University of Alberta
RECRUITING
Edmonton
Hospital For Sick Children
RECRUITING
Toronto
France
Hôpital Necker - Enfants Malades
RECRUITING
Paris
Netherlands
Erasmus MC
RECRUITING
Rotterdam
Universitair Medisch Centrum Utrecht
RECRUITING
Utrecht
Spain
Hospital Universitario Cruces
RECRUITING
Barakaldo
Hospital Sant Joan de Deu - PIN
RECRUITING
Esplugues De Llobregat
Hospital Universitario 12 de Octubre
RECRUITING
Madrid
CHUS - H. Clinico U. de Santiago
RECRUITING
Santiago De Compostela
Hospital Universitario Virgen del Rocio - PPDS
RECRUITING
Seville
United Kingdom
Birmingham Children's Hospital NHS Foundation Trust
TERMINATED
Birmingham
Royal Manchester Childrens Hospital
RECRUITING
Manchester
Contact Information
Primary
Moderna WeCare Team
WeCareClinicalTrials@modernatx.com
1-866-663-3762
Time Frame
Start Date:2021-08-06
Estimated Completion Date:2028-08-01
Participants
Target number of participants:63
Treatments
Experimental: mRNA-3705
Participants in Part 1 will receive a weight based dose of mRNA-3705, administered intravenously (IV), once every 2 weeks (Q2W) or once every 3 weeks (Q3W) for up to 10 doses over approximately 40 weeks. Participants in Part 2 will receive mRNA 3705 at the selected dose level and frequency for up to 12 months.