A study to investigate mRNA-3927 in participants with genetically confirmed propionic acidemia

JRCT ID: jRCT2043240044

Registered date:13/06/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Propionic Acidemia
• Date of first enrollment: 13/06/2024
• Target sample size: 3
• Countries of recruitment: Canada,Japan,Spain,Japan,France,Japan,U.K.,Japan,Italy,Japan,Netherlands,Japan,U.S.A.,Japan
• Study type: Interventional
• Intervention(s): Intravenous administration once every two weeks at 0.6 mg/kg for approximately 52 weeks

Outcome(s)

• Primary Outcome: Annualized frequency of MDEs during 12 months of treatment

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Participant must be >=1 year of age at the time of consent/assent if enrolled after the first 2 participants.
- Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (PCCA and/or PCCB mutations).
- Part 2 only: At least one documented MDE in the 12-month period before consent.

Exclude criteria

- Estimated glomerular filtration rate < 30 milliliters (mL)/minute/1.73 square meter (m^2) as estimated by Schwartz formula for participants < 18 years of age or the Chronic Kidney Disease Epidemiology Collaboration creatinine based formula for participants >= 18 years of age or 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.
- COVID-19 vaccination (generally 2 doses or a booster) within 6 weeks between their last COVID-19 vaccination dose and first study drug administration.

Related Information

• Primary Sponsor: Watabe Mitsuo
• Secondary ID(s): NCT04159103,2019-003529-36