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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 52

Inclusion Criteria

= 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1;, = 1 year of age at the time of consent/assent if enrolled after the first2 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., (Part 2 only) At least one documented MDE in the 12-month period before consent.

Exclusion Criteria

Any individual with laboratory abnormalities achieving thresholds defined in Table 16;, 3. QTc > 480 ms using Bazett's correction;, In female participants of reproductive potential, a positive pregnancy test;, 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-/H2 receptor blockers;, History of hypersensitivity to any component of the study drug;, 17. Previously received gene therapy for PA., 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;, COVID-19 vaccination (generally 2 doses or a booster) within 6 weeks between their last COVID-19 vaccination dose and first study drug administration., 16. (Part 2 only) History of hepatitis B (known positive HBsAg, HCV, or HIV (positive HIV-1/HIV-2 antibodies). Participants with a past or resolved hepatitis virus B infection (defined as the presence of hepatitis B core antibody and absence of HBsAg are eligible. Participants with history of positive results for HCV antibody are eligible only if polymerase chain reaction is negative for HCV RNA., Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, as estimated by Schwartz formula for participants < 18 years of age (Schwartz et al 2012) or by the Chronic Kidney Disease Epidemiology Collaboration creatinine-based formula for participants = 18 years of age or for participants of all ages receiving chronic dialysis;

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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