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New treatment option for Propionic Acidemia
Official title AAVrh10-PCCA Gene Therapy for Propionic Acidemia
ClinicalTrials.gov ID: NCT07643844
What this study is testing
What is AAVrh10-PCCA low dose?
AAVrh10-PCCA low dose is an investigational medicine, being studied as a potential treatment for propionic acidemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It results from loss of function of the mitochondrial enzyme propionyl-CoA carboxylase and can be due to disease-causing variants in the PCCA gene, leading to accumulation of propionyl-CoA and its toxic metabolites.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 2
You may be able to join if
- Age six months to 2 years of age at day of vector infusion. For those \<1 year of age they must have been ≥37 weeks gestational age at the time of...
- Confirmed diagnosis of propionic acidemia with biallelic PCCA gene mutations based on molecular genetic testing.
- Study participants must have a diagnosis of neonatal-onset propionic acidemia with a documented episode of decompensation that can include any of the...
- Parents or legal guardians of study participants must agree to comply in good faith with the conditions of the study, including attending all of the...
You likely can't join if
- Hemoglobin \<10 g/dl
- Platelet count \< 100,000 per mm3
- Liver Enzyme ALT/AST \>2.5 ULN
- Direct Bilirubin \> 1.5
- Active viral infection (includes HIV or serology positive for hepatitis B or C).
- Previous liver transplant
See the full eligibility criteria
- Age six months to 2 years of age at day of vector infusion. For those \<1 year of age they must have been ≥37 weeks gestational age at the time of birth and without other conditions/comorbidities that in the opinion of...
- Confirmed diagnosis of propionic acidemia with biallelic PCCA gene mutations based on molecular genetic testing.
- Study participants must have a diagnosis of neonatal-onset propionic acidemia with a documented episode of decompensation that can include any of the following findings: lethargy, poor feeding, irritability, vomiting...
- Parents or legal guardians of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and parents or legal...
- Hemoglobin \<10 g/dl
- Platelet count \< 100,000 per mm3
- Liver Enzyme ALT/AST \>2.5 ULN
- Direct Bilirubin \> 1.5
- Active viral infection (includes HIV or serology positive for hepatitis B or C).
- Previous liver transplant
- people with active decompensation as demonstrated by a pH \ 75 mcmol/L, lactate \> 2.5 mmol/L, urine ketones
- Previously received gene therapy or messenger ribonucleic acid (mRNA) treatments for PA.
- Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
- Family does not want to disclose patient's study participation with primary care physician and other medical providers.
The study team makes the final eligibility decision.
Where it's taking place
- Rochester, Minnesota, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months to 2 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Rochester, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.