A clinical study to evaluate the medicinal product in Patients with Propionic Acidemia

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 36

Inclusion Criteria

= 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants;= 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); Participant and/or legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulations and 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 study treatment and for 3 months following the last administration of study drug.
Are the trial subjects under 18? yes
Number of subjects for this age range: 36
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Any individual with laboratory abnormalities achieving thresholds;
Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, as estimated by Schwartz formula (Schwartz et al 2012); or participants who receive chronic dialysis;
Corrected QT interval (QTc) > 480 ms using Bazett’s correction;
In female participants of reproductive potential: a positive pregnancy test at the Screening Visit;
Pregnant or breastfeeding;
Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification;
History of organ transplantation or planned organ transplantation during the period ofstudy participation;
Hypersensitivity to acetaminophen/paracetamol and/or ibuprofen or H1-receptor blockers;
History of hypersensitivity to any component of the study drug;
History of anaphylaxis/anaphylactoid reaction or severe hypersensitivity with infusions;
Participation in another clinical study of another investigational agent within 30 days before study entry or within 5 elimination half-lives of the investigational agent,whichever is longer;
Major surgical procedure within 30 days before the Screening Visit (excludes central line, port, or feeding tube placement);
Enrollment in the study is not deemed to be of clinical benefit, in the opinion of the Investigator;
Other condition that in the Investigator’s opinion could interfere with interpretation of study results or limit the participant’s participation in the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluate the safety and tolerability of mRNA-3927 in participants with PA;Secondary Objective: Characterize the PD responses of mRNA-3927 as determined by changes in plasma 2-MC and 3-HP after single and repeated administrations of mRNA-3927;Characterize the single-dose and repeated-dose PK of mRNA-3927;Assess for the presence of anti-PEG antibodies.;Primary end point(s): Incidence and severity of AEs (including study drug-related and not related AEs), SAEs, and AEs leading to treatment discontinuation;Timepoint(s) of evaluation of this end point: Primary endpoints will be evaluated over the entire period of the trial

Secondary Outcome Measures

Name	Time	Method

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