NCT07125066
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
Enrolling by Invitation
PHASE1All AgesInterventionalTreatmentJerry Vockley, MD, PhDInvestigator-initiated
~1 participants
Updated 2026-06-08 on ClinicalTrials.gov
What's tested:Sodium phenylbutyrate
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of treatment related adverse events
Measured over 2 years
Conditions
Where it's being run
1 sites across 1 statesPennsylvania1
Study leadership
- Gerard Vockley, MD, PhD · PRINCIPAL_INVESTIGATOR · UPMC Children's Hospital of Pittsburgh
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Single patient with Combined D,L-2-hydroxyglutaric aciduria
Parental permission must be given at the start of the protocol.
Parents willing to adhere to the protocol
Exclusion
Refusal or withdrawal of consent.
Drug Intolerance.
What this trial measures
- Number of treatment related adverse events2 years