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New treatment option for Pyruvate Dehydrogenase Complex Deficiency
Official title Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
ClinicalTrials.gov ID: NCT06340685
What this study is testing
What is Triheptanoin?
Triheptanoin is an investigational medicine, being studied as a potential treatment for pyruvate dehydrogenase complex deficiency.
Also referred to as Dojolvi.
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
- This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency. The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders.
- 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 1 to 17
You may be able to join if
- Age 1 year to \<18 years of age
- people with PDCD would need to have a metabolic physician following their clinical care needs prior to their enrollment in the study
- Diagnosis of PDCD by molecular genetic confirmation of PDHA1, PDHB, DLAT, PDHX, or PDP1 mutation
- Not pregnant or lactating
- Parental permission and assent of minor and willingness to comply with study procedures
You likely can't join if
- Diagnosis of medium-chain acyl-CoA dehydrogenase (MCAD)
- Use of alcohol or drugs of abuse
- Evidence of liver disease as defined by elevations of AST or ALT \>2x ULN in the past 6 months
- Pregnant, breastfeeding, or lactating females
- On any investigational product research study (and not completed the required 30-day washout period prior to Intake \& Dosing) or recipient of gene...
See the full eligibility criteria
- Age 1 year to \<18 years of age
- people with PDCD would need to have a metabolic physician following their clinical care needs prior to their enrollment in the study
- Diagnosis of PDCD by molecular genetic confirmation of PDHA1, PDHB, DLAT, PDHX, or PDP1 mutation
- Not pregnant or lactating
- Parental permission and assent of minor and willingness to comply with study procedures
- Not participating in any treatment treatment clinical trials
- Not a recipient of gene therapy, organ transplant, or bone-marrow transplantation
- If currently on any investigational drugs or therapies, must complete a 30-day washout period prior to Intake \& Dosing (Day 1).
- Negative pregnancy test for all female patients of childbearing age. Individuals of childbearing potential must agree to use a highly effective method of contraception, and males must agree not to father a child or...
- people are following some form or type of ketogenic diet at the time of the screening visit.
- Diagnosis of medium-chain acyl-CoA dehydrogenase (MCAD)
- Use of alcohol or drugs of abuse
- Evidence of liver disease as defined by elevations of AST or ALT \>2x ULN in the past 6 months
- Pregnant, breastfeeding, or lactating females
- On any investigational product research study (and not completed the required 30-day washout period prior to Intake \& Dosing) or recipient of gene therapy or organ or bone-marrow transplantation
The study team makes the final eligibility decision.
Where it's taking place
- Pittsburgh, Pennsylvania, United States
Compensation & support
Some study costs may be covered.
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 listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Pittsburgh, Pennsylvania, 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.